Thalassemia Patients and Friends

Discussion Forums => Working Towards a Cure => Topic started by: Andy Battaglia on June 16, 2014, 03:10:11 PM



Title: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 16, 2014, 03:10:11 PM
Bluebird today released the full data on their first two patients in their current gene therapy trials. Both are responding well and have not had a transfusion since the second week of the trials. The full pdf is attached to this post. If you have questions, please ask them, as I will be able to talk to Bluebird again in the coming weeks to ask any follow up questions you may have.

This is a very exciting time. The early results are quite good and show good expression of the HbB-A-T87Q, which is the adult hemoglobin being produced by their process.

I want to thank Bluebird Bio for being kind enough to spend some time talking to me and answering and explaining this process which is very important to all of you. Those attending the CAF conference this weekend will see presentations from Bluebird and also from Sloan Kettering where a separate trial is taking place.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: JV on June 17, 2014, 07:17:41 AM
This is great Andy, thank you for all your efforts


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Bostonian_04 on June 17, 2014, 04:13:36 PM
This is great news. Thank you Andy for the attached presentation. They saw positive outcome for β0/βE -thalassemia major...Will this be seen in β0/β0 or other mutations as well? Will the treatment is less harsh than BMT?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on June 18, 2014, 12:12:04 AM
Thanks Andy...for sharing the great news..hope soon it will available to all....


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on June 18, 2014, 12:55:01 AM
This is really amazing news, thank you Andy for sharing the updates!! Awaiting results from Dr. Sadelain on their trails...


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Rajiv on June 18, 2014, 08:51:13 AM
Dear Andy,
Thank you so much for your tireless work to get details of this trial.  Also thanks for your kind efforts for supporting and helping thal kids and the families. 
This is really an amazing and long awaited good news.  Hope this therapy will provide best and permanent cure to the thal patients with
no chances of side effects, I pray to almighty for those suffering with this disease.
best regards


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 18, 2014, 09:26:58 AM
Patients with beta zero thal will be included in trials and the expectation is that they will have the same type of beta globin production. These new results are very encouraging because an increase of >6 will be needed to free beta zero majors from transfusion.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sushil on June 18, 2014, 10:38:57 AM
Hi Andy,

Please take two question from my side also.

1) by when it will be available for all Thal Major
2) I see that most of trial are being done in Europe/US whereas i believe that most patient for Thal major lies in Asian region. is there any chance to start
these test in Asia also like in India, Pakistan etc.
3) most important how much cost we expect to have for this treatment.

Regards
Sushil Thakur


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 18, 2014, 06:45:51 PM
Sushil,

It is premature to guess when this will be available to the public. It will take years of observation before it can be presented to the FDA and then the FDA will take quite a bit of time reviewing this before it can ever be approved.

Trials are currently slated for Australia and Thailand, also. I keep reminding them that India has a huge amount of thal majors.

Again, no idea on cost, but early estimates were that it would be more than it costs for BMT.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on June 19, 2014, 03:16:58 PM
I just can't help but follow the money trail. One has to be very careful with public limited companies, since the very early announcement of gene therapy, the shares of Bluebird has jumped 65% in last four days (amazing). The share is in the oversold territory with RSI of approx 84 times. Now, I am a little worried about the timing of Blue bird announecement, since they recently have two earnings downgrades by the analysts.

The share was going to fall on earth, untill the recent announcement. This is just another perspective.

We all know what happened to some other experimental companies (who claimed to have find the jackpot process), but in the end are now trading over the counter (they are not listed anymore).

From the Thal point of view (I hope the best to BlueBird).


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: EugenioLaMesa on June 20, 2014, 03:40:20 AM
Trials are currently slated for Australia and Thailand, also. I keep reminding them that India has a huge amount of thal majors.

About 130,000 thal majors in India, with 10,000 new born every year.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Rajiv on June 20, 2014, 11:07:14 AM
I think that cure thalassemia should put all required efforts to make available gene therapy treatment in India.  They do BMT across world and in Italy and in India, therefore,
to make the treatment more safe and GVHD and infection free and also to make treatment more successful they can opt for gene therapy.  They don't
have to find a donor for this treatment.   Hope with the commencing of gene therapy treatment, cure thalassemia will work in this field.
thanks and best regards


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: EugenioLaMesa on June 20, 2014, 06:49:59 PM
I think that cure thalassemia should put all required efforts to make available gene therapy treatment in India.  They do BMT across world and in Italy and in India, therefore,
to make the treatment more safe and GVHD and infection free and also to make treatment more successful they can opt for gene therapy.  They don't
have to find a donor for this treatment.   Hope with the commencing of gene therapy treatment, cure thalassemia will work in this field.
thanks and best regards

We do BMT now because it exists since 30 years and there is lot of data and experience available. But we also know that BMT is only for young children, for adults it is not an option, the risk is too high.

Regarding Gene Therapy we are monitoring it, as you know it is still a trial and if and when it will be available we will carefully evaluate its results, side effects, cost, etc to see if we can make it available in India. It will probably takes some/many years.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 21, 2014, 10:32:19 AM
Canadian family,

I have had the same thoughts about the stock price. I saw it coming. When Bluebird finally contacted me, I knew something was up. I considered buying the stock, but felt it could compromise my objectivity, so I decided against it, in spite of knowing it was going to explode. I could have made a bundle too, at a time when I could really use the money. I just couldn't do it. I have to remain objective if I am going to serve thals. It's not the first sacrifice I've made and won't be the last. I have to stay true to myself and to those I hope to help.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on June 22, 2014, 05:35:34 AM
Andy, Truly appreciate your self-less efforts for the good and betterment for all the thals across the word!!


Take a Bow :thankyou


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on June 22, 2014, 09:18:06 PM
Andy,

Thank you for your constant service to thalassemia patients. 



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on June 23, 2014, 08:44:53 AM
Hi Andy,

You did the right thing. Great achievements comes with great sacrifices.....



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Rajiv on June 23, 2014, 10:09:32 AM
Dear Andy
We are grateful for your constant help and guidance which is greater than any wealth in the world.
Only rare person can make that.  We find you stand besides us all the time.  Whenever we have any issue
we take your help first and then the doctors.   We have great respect for your dedication and sacrifice.
Thanks and regards


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: EugenioLaMesa on June 23, 2014, 02:43:20 PM
Dear Andy,

I admire and respect you, and you're a great source of inspiration for me.

Best
Eugenio


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sara76 on June 23, 2014, 09:11:36 PM
I echo the above sentiments! 
The whole world would benefit from more people like Andy!

 :thankyou
Suelou


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Bostonian_04 on June 24, 2014, 06:06:06 PM
Andy, i am not surprised by your action. this is what you are...a truly selfless man helping everyone who asks for help. Thank you for being there for us.....The world is certainly a better place for all of us Thalpals because you are here.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on October 15, 2014, 05:57:51 AM
Bluebird has started its first trails for Sickle Cell Disease with its LentiGlobin vector


CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, today announced that the first subject with severe sickle cell disease has undergone infusion with bluebird bio’s LentiGlobin BB305 drug product in an autologous hematopoietic stem cell transplantation. This patient is enrolled in the HGB-205 Study being conducted in Paris, France. bluebird has also opened a separate US-based trial (HGB-206) in the United States for the treatment of up to 8 severe sickle cell disease patients with the company’s LentiGlobin BB305 drug product

“We are treating a sickle cell patient for the first time with gene therapy,” stated Marina Cavazzana, MD, PhD, Professor of Medicine at Paris Descartes University and Research Director at the Centre for Clinical Research in Biotherapy, Necker Hospital, and at the Institute of Genetic Diseases, Imagine, Paris France. “Sickle cell disease is a devastating disease that affects hundreds of thousands of people in the US and Europe and millions around the world. The therapeutic options for patients with sickle cell disease are currently limited, so the opportunity to bring a one-time, potentially curative treatment to these patients by modification of autologous hematopoietic stem cells would represent a great advance for patients with sickle cell disease and for the field.”

“Sickle cell disease shortens life expectancy by decades even in developed countries, so it is exciting to contemplate that LentiGlobin may offer the curative potential of allogeneic stem cell transplantation by using a patient’s own cells,” stated David Davidson, MD, bluebird bio’s Chief Medical Officer. “In June 2014, we reported preliminary results from the HGB-205 Study demonstrating that treatment with LentiGlobin drug product led to high-level production of beta-T87Q-globin and rapid transfusion independence in two beta-thalassemia major patients. Given the anti-sickling property of the amino acid substitution engineered into beta-T87Q-globin, we are optimistic about the potential for LentiGlobin to mitigate the signs and symptoms of sickle cell disease. We anticipate providing initial clinical data on LentiGlobin in sickle cell disease patients in 2015.”

Reference: http://www.businesswire.com/news/home/20141014005311/en/bluebird-bio-Announces-Patient-Sickle-Cell-Disease


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on October 15, 2014, 09:18:02 AM
This will be the true test of the process, as sickle cell requires higher dosing and more output from the beta globin gene if it is to be cured by gene therapy.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on October 15, 2014, 09:39:00 PM
This is great news!  My best to the sickle cell patients - they truly go through so much. 

Furthermore, if there is success in sickle cell - and there are far more sickle cell patients, chances are blue bird will get more attention and hopefully more funding to help sickle cell patients, thalassemia patients and patients with other hemoglobinopathies which are less known.


Great news for everyone - I wish them the best!


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on October 16, 2014, 12:18:41 AM
All the very best....hope for the best only..

Regards,
Ashutosh


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: bomboi on December 03, 2014, 10:24:26 PM
When is the next update on the study , i read somewhere that it should be coming soon


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 03, 2014, 10:27:51 PM
I believe it is going to be updated at the ASH conference this month.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on December 04, 2014, 01:57:47 PM
Lot of insiders selling the stock recently. Does it mean anything, probably not or perhaps yes.....


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on December 04, 2014, 09:34:09 PM
I'm wondering though, if a blackout period wouldn't be imposed on insiders if something significant were happening?  but my understanding of business if very poor. 

I sure hope things are going well...


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 04, 2014, 10:25:43 PM
If you look at the past two months, there's been dumping of the stock when it gets to 42-43. The timing right now just before the ASH conference may just be coincidence. I'm more concerned about what is happening at Sloan Kettering, which seems to be very little. Look for announcements from ASH next week.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on December 05, 2014, 12:51:42 AM
I am hopeful that the news will be good.  I am also worried about Sloan Kettering.  I had really been hoping that their myelosuppression method would be more successful.


I wonder if the greater success bluebird is enjoying is due to the myeloablation or if they are using a superior method of gene transfer?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: JV on December 05, 2014, 06:33:05 AM
Andy, could you elaborate on Sloan a little more because I feel the same way. Delays, not much news, poor results, etc...


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on December 05, 2014, 12:02:33 PM
You are right on Andy. Yes, when a stock reaches 42-43, insiders seems to be dumping the stock. Now the question is if external analyst have a target price of stock at 50-57, why would one just dump the stock at 42-43. Please see few examples, there are number of other examples.

CEO Nick Leschly sold 25,000 shares of bluebird bio stock in a transaction that occurred on Monday, November 3rd. The stock was sold at an average price of $41.68, for a total transaction of $1,042,000.00

bluebird bio (NASDAQ:BLUE) Insider Mitchell H. Finer sold 18,000 shares of bluebird bio stock on the open market in a transaction dated Monday, December 1st. The stock was sold at an average price of $39.70, for a total value of $714,600.00....

Lets see what happens at ASH presentation.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 05, 2014, 01:11:45 PM
The timing is interesting, but I don't want to read anything into it until we hear what is released at ASH.

JV, all I know is what Pat G said and it sounded like they are not currently beginning new patients in trials. My concern is the money side and what may be attempts to stifle the competition. I have very little faith in the top management at SK. I think they can be bought.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: JV on December 07, 2014, 10:20:27 AM
Thanks Andy, ya I agree with you. I wonder how much of Cooleys Funding goes into that research...


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on December 07, 2014, 02:28:22 PM
That is all very concerning.  Things are not moving at SK at all.  Most of our cards are on BB at this time.  With the momentum these two groups started off last year - it is very surprising that more exciting news is not coming out and there is no sign of new patients being treated by either group.  

The last BB update in November stated that the first two patients continue to be transfusion free, expressing high rates of the new hemoglobin.  

Praying for the best news from both of these groups.  

Very disappointing that what is a life line for some of us - is a money making scheme for some :(


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: JV on December 07, 2014, 07:12:08 PM
Sharmin, that was very well put. We have been all in since day one and for a major organization like SK to not be more proactive is extremely disappointing. Hoping for Bluebird to run with it now...


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Bostonian_04 on December 08, 2014, 07:17:51 PM
Latest from Bluebird....

Bluebird posts more promising results for its rare disease therapy
December 8, 2014

http://www.fiercebiotech.com/press-releases/bluebird-bio-announces-data-demonstrating-first-four-patients-thalassemia-m


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 08, 2014, 08:22:21 PM
These results include the first that Bluebird has done using thalassemia major patients. The early results are quite impressive. We will be eagerly waiting for the results on the one sickle cell patient that has started.

It is still quite early on, and hopefully there will be no serious issues develop down the road, but we now know with certainty that gene therapy does work. I will not hesitate to tell patients to take part in trials if eligible. It may be the only way many patients will ever be able to afford it.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on December 08, 2014, 08:45:36 PM
Bostonian,

Thank you soooo much for posting this!  Such great news!  Praying that it provides a solution for everyone suffering from thalassemia or sickle cell anemia!

Sharmin


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 08, 2014, 09:01:34 PM
And for those keeping track, Bluebird's stock rose more than $4/share today.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on December 08, 2014, 11:48:58 PM
Bluebird Bio Announces Data Demonstrating First Four Patients with β-Thalassemia Major Treated with LentiGlobin™ are Transfusion-Free

http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=1995913


Regards,
Ashutosh


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on December 09, 2014, 11:31:37 AM
This news is beyond incredible Andy.  For Beta zero patients - I am assuming we would be looking at patient 1106 and 1107 for the next few months.  It is impressive that their hemoglobins are 9.6 are 3 months and 1 month.   Even if the hemoglobin were to stay at this level it is remarkable success. 

Hoping this works for everyone!

It would be nice to see follow up data regarding any possible deficits due to bisulfan - such as fertility.  But that is a future consideration.  for the time being - this is life changing!

Sharmin


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 09, 2014, 12:10:07 PM
Bluebird's stock value has doubled today.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 09, 2014, 12:35:46 PM
I should be having another phone conference with Bluebird later this week. If there are any questions you would like asked, please reply by Thursday morning at the latest.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: JV on December 09, 2014, 01:54:48 PM
As Sharmin said... Fertility issues and post transplant medication/care.

Thanks Andy


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Bostonian_04 on December 09, 2014, 11:17:33 PM
Andy,
Thanks for talking to Bluebird....Few questions in my mind is...

1. which hospitals in US are they partnering with for these trials.
2. what are their main challenges and setbacks so far....not asking about financials.. from patient safety.
3. when can we expect it to be a mainstream cure...covered by insurance.

there are so many questions...but answering these few wiil do :) I am sure other members and you have additional questions.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on December 10, 2014, 12:19:31 AM
Andy,
can we also understand the reason for variance in hb (beta-T87Q) patient to patient post administration of Lenti-globin? How they are trying to achieve a similar hb increase for different patients.
And obviously safety concerns is another must ask.

thanks Andy!





Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on December 10, 2014, 09:14:18 PM
I'm not sure if this is valid question - but I wonder about the interim period - while waiting for the new treated marrow to engraft, how the patient is supported.  I am guessing transfusions of red bloods cells and ivig?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on December 11, 2014, 04:48:07 AM
I don't know if I put by question currently: my question was why there is difference in hb increase post gene drug administration between patients

is it because of varying amounts of vector being administered??


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 11, 2014, 09:25:50 AM
There are many variables, including the fact that each patient is unique. However, dosing is still in the testing stage, so the dosing has not been the same for each patient. Time and experience will refine the process.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 12, 2014, 07:32:22 PM
I think the events of the past week have made it look like it was poor timing for people at Bluebird to not wait another week and make a fortune before selling stock. What I have learned is that it actually is impossible for anyone involved in the company to sell their stock on their own. Each one has a program that has set prices at which X amount of shares will be automatically sold. That is the only way that their own stock can be sold, so they cannot even react to events after they've been made public, so this means they can't do anything even though the stock more than doubled in the past week. I really like their arrangement. The only way they will make big money is by producing a successful product. Even if their only motivation was money, this would be enough incentive to do it right. But I have met people from Bluebird and spoken with others on the phone and there is more to it than money. They really do want for find cures for people. It's about money, but it's also about doing good for others. That's something I can really respect.

The importance to us is that we won't learn anything new from watching their financial picture. The news will come in these periodic announcements at conferences. What is most exciting is that the news has been quite good. Let's hope the trials continue to show success without any unforeseen obstacles. I would describe the people at Bluebird as cautiously optimistic. We're watching the world change right in front of us. After so many years of seeking a road to cures for so many disorders, we have finally found that road and begun our journey.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 13, 2014, 12:24:22 PM
I had a two hour talk with Faraz from Bluebird. I have to say that the more I talk to him, the better feeling I get about Bluebird. It's not often that I hear the Golden Rule brought up in conversation, so I do take note that there is more to Bluebird than money, which is refreshing.

I received answers for most questions that were posed.

Chemotherapy concerns: Chemo brings the same risk of side effects that are present when used with BMT. Loss of fertility is a possibility, but is not predictable, so it is advised the sperm or eggs be collected in advance. Short term side effects from the chemo are a known factor. Reminder: There is no risk of graft vs host disease, removing the biggest concern about BMT. There is nothing to reject, as the patient's own bone marrow is collected and used.

Long term costs: We don't know yet. Patients must continue chelation afterwards until their iron burden is normal, but aside from that, nothing long term is currently known. In theory, there should be no long term issues, but one of the reasons trials are run is to observe over longer periods. As far as long term maintenance, no immunosuppression drugs or other drugs are required.

Are transfusions required after the process? Patients are transfused to a high Hb before the process begins. Only a couple have needed any blood at all after the procedure, and that involved transfusing only small amounts of blood one time. After that, they remain transfusion-free.

Will patients lives eventually become normal? That is the hope and nothing currently known suggests otherwise.

Have there been any setbacks or unforeseen issues? Not so far, and they learn more from each new patient.

Have things changed a lot since the first patient was treated 7 years ago in France? The process continues to become more refined and with each new patient, more is learned. The vector used today is improved and more effective, but there haven't been any major changes. The first patient continues transfusion-free, as have all subsequent patients.

Recruitment for trials: See http://www.northstarstudy.com/resources.html and begin the screening process if interested. The current trials are almost full, but a few more patients will make it into these ongoing trials which are taking place in the US, France, Thailand and Australia. Current patient origins are from France, the US, Pakistan, Australia, Thailand and Syria. More trials will be coming and the age limit will be lifted, allowing younger and older patients to apply. There has been no trouble attracting patients for trials, and with this recent report, we can expect interest to increase, so it is highly recommended that interested patients begin the screening process ASAP, so they can get in the queue and have a chance of being selected for trials. I have seen nothing in any report that should cause any hesitation applying for participation in trials. Bluebird takes care of the cost for the trials, including transportation costs, so do not be deterred by your location.

Re: Sickle Cell: The first patient has been started but it is too early for any reports. With sickle cell, stem cell mobilization has to be carefully done, as the drugs used can mobilize the production of more sickle cells, as well so achieving proper dosing and use of mobilization drugs will take some time and experience. We will eagerly await further reports. It's exciting that the trials have expanded beyond thalassemia.

I have never had any doubts about the eventual success of gene therapy. The waiting has been agonizing, but we are now well on the road to a new era of medicine. We are living in miraculous times and we need to seize the moment and not allow research to be deterred by political buffoons who constantly threaten to de-fund research, as we in the US saw last year with the government shutdown. As world citizens, we should demand better from our leaders. Let's demand that our tax dollars go to helping solve the real problems humanity faces, rather than fund more exploitation of humanity through conflict and wars. There are so many avenues of research taking place to find treatments and cures for a multitude of disorders and diseases, and in most cases, the only thing holding them back is lack of funding for research. We have seen this over the past 10 years regarding gene therapy. We are a couple years behind in the US, due to earlier lack of funding. At every conference, you can find researchers presenting their work, and so much shows real promise, but always the same stumbling block is present and that is lack of support for research. Let's make a conscience decision to support saving humanity, rather than destroying it.

I have attached the latest report as a pdf attachment to this post.



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 13, 2014, 01:13:50 PM
I think I left out a little bit of information that everyone wants to know. We are hopefully looking at a time frame of 5-10 years before we see FDA approval. Of course, we've all seen how slow things move in the real world, but let's be optimistic about this.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on December 13, 2014, 02:05:46 PM
Andy,

Thank you so much for all of this information.  What an incredible source and advocate you are for us.



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on December 15, 2014, 12:37:37 AM
Incredible, Incredible, Incredible..

Thanks Andy for sharing the information.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on December 15, 2014, 05:58:19 AM
Thanks Andy for all the update, it is indeed great & lucky to have you along side :)


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on December 15, 2014, 08:44:57 AM
I am joining the party little late, busy with work...

Great news from Bluebird, Andy thanks for clarifying lot of issues. With respect to shares, its good to see financial strength, I like the way 'insiders' dispose their shares (learned something new here).

I continue to hope that Bluebird make progress, however, I still believe MSKCC can bring a lot of value to the table.

PS. I do not hold stock position in Bluebird :)



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on December 15, 2014, 12:37:11 PM
Andy,

I agree with your statement about seeing the humanity in this group and the research they are doing.  I have seen a few videos and reviewed some of the companies policies and to be honest I do so a lot of good will on the part of bluebird bio.  

I also see goodwill in Michael Sadalaine, the initial errant gene group and our good friend Pat Girondi.  Let's pray that their research continues and yields strong results in the near future.

Sharmin


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Bostonian_04 on December 16, 2014, 03:21:35 PM
Andy, Thanks you so much for the detail sharing. Can't even have enough words to Thank you for all the things you do for this group. Your statement about Bluebird bio's management makes me feel very hopeful that it is not only money...but they do care for the patients. Praying for continued success of Bluebird....


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: jay on December 29, 2014, 09:43:51 AM
Hi Andy,
A question for Bluebird.
Will they release subject's full genome sequence public before and after gene therapy?
Is it the part of their terms and conditions with subjects?
I tried to search if putting such condition in trial agreement is allowed by US law but not successful.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: jay on January 02, 2015, 06:48:15 PM
So many questions to Andy, please answer anyone,
In addition to my previous post, after seeing David Levy's interview by Josephine, another question to Bluebird.
What is the exact mutation they cure? or do they replace entire HBB?
There are multiple Thalassemia major mutations on HBB.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on January 03, 2015, 01:29:42 PM
Jay,

I have many urgent matters to deal with on a daily basis, so I must prioritize. Addressing situations that require immediate attention will always take precedence over questions that have no urgency. I hope you do realize that my efforts are volunteer and that I give an inordinate amount of time to thals. I have no appreciation for impatience. Excuse me if my time has been spent on helping people with urgent issues that should have been addressed by their own doctors long ago. I really will put the mother of a 36 year old woman whose life is not worth living above random questions that have absolutely no urgency. In addition to my public work here, I run three Facebook thal groups and am constantly inundated with emails and personal messages. It becomes quite frustrating when adults show a lack of patience. And I say all this while suffering for over two weeks from the flu, yet I have missed no work and have attended to some very urgent cases, in spite of feeling terrible.

To answer the questions, they are doing DNA testing of patients. This is seen in each patient being labeled HbE beta thal +, beta zero zero, beta + zero, etc. They can only know this through testing. The reason for doing this is to show that it works on all patients, regardless of the severity of their thalassemia. The original condition has no relevance in terms of the cure, though. Good beta globin genes are inserted into the body and what is already there has no relevance beyond its initial ability to produce hemoglobin, which in the end will be part of the whole. What trials will show is what has to be done in terms of preparation in terms of chemotherapy, and how dosing has to be adjusted for individual cases. The original genotype has no effect on this process. Unlike with BMT, you are not changing the patients genetically. It is simply insertion of working genes. Their function substitutes for what is already in the body and does not work properly. The old genes are still there and 'working" but a newly introduced beta globin gene will do the work. You can almost look at it as a recipe that needs something. Ah, salt! It's incomplete until salt is added and the blood making ability of thals is incomplete until you add the salt of a working gene and now the recipe works.



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on January 03, 2015, 01:45:16 PM
And Jay, if I have missed any questions with my responses, please point to the posts and I will address therm. I try to catch up on weekends when time permits, and try to find everything I have missed. but sometimes overlook posts.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: jay on January 03, 2015, 02:49:56 PM
Hi Andy,
I must apologize for sounding urgent, I am not native English, and actually I wanted someone else to answer it because I do understand the extra efforts you put and I am aware of it from long time. This forum has given immense knowledge to me and I couldn't have done better it without this forum. I feel so many questions are directed to you because everybody believes in you.  :imsorry


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on January 03, 2015, 03:28:27 PM
No problem. Just realize that I am an incredibly busy person even before I get to thal work, so I do have to often put things off until I have more time. My activity on thalpal does normally increase on weekends when I am not at work. Lately, my efforts have been reduced by illness. Hopefully, this lingering flu will soon clear up. I hate to respond to many posts when my mind is not as clear as I wish. If I feel I cannot adequately process thought, I will wait until my head clears up.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: jay on January 05, 2015, 04:31:44 AM
How gene therapy works? TEDx by a Bluebird guy

https://www.youtube.com/watch?v=Ez560GnkSrE


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: jay on January 05, 2015, 05:42:25 AM
I have been studied biology till age of 19 before moving on to technology, I can do some basic DNA tests, I can do electrophoresis, and I think I understood the explanation in previous post. (These all 'I can's where unnecessary). But again I stuck, pity on me, do this process cause additional mutation in the particular cells? Do this process cause extra addition in genome other that the mutated part? Are there other parts of vector grow along with the cells?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on January 05, 2015, 10:33:04 AM
No, no mutation is caused. Working beta globin genes are inserted into the patient where they can multiply. The gene vectors are produced in a lab and are not from a donor, so no reactions. To put it simply, something is added that is not already there. It's a normally working gene. The method varies slightly between companies, as only the trial at Sloan Kettering uses the beta globin gene exactly as is found in the human body. The Northstar trial uses a mutated gene, but this causes no mutation in the body, anymore than the mutated gene already present in thals does not cause any mutation of anything else in the body. This is all confined to the beta globin gene. So, instead of relying on a mutation that does not work, they use a mutation that does produce beta globin. This new gene's output can thus be easily monitored and we see the results in the trial reports, as the new hemoglobin being produced is listed as part of the whole in the charts that have been posted. The new hemoglobin produced by the new gene is labeled as HbA t87Q in the charts.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on January 05, 2015, 07:11:22 PM
Andy,

Thank you kindly for synthesizing this information for us.  I hope that you are starting to feel better!  I was wondering if you had heard anything from Sloan Kettering.

Sharmin


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on January 07, 2015, 07:15:08 PM
Thanks Andy for making it so easy to understand, we can easily lost on the technicalities.

Hope you have started to feel better!


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: jay on January 13, 2015, 05:29:10 PM
one more, What causes infertility? does it? Why it is suggested to save reproductive cells?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on April 15, 2015, 04:02:17 PM
Bluebird bio is due to present this weekend.  The stock price has skyrocketed.  This success is so so great to see.  We're are blessed that thalassemia is the disease being cured by this therapy.  So many other ailments wait.  I hope that this will be a huge success and such treatments can be extended to help others.

Jay, the chemotherapy which is used to suppress and ablate the marrow (which is done both to suppress the immune system so that the corrected cells will be accepted and to make room for the corrected marrow to settle in to the patient's body) can also cause damage to reproductive cells.  There is a risk of infertility with this dose of busulfan (the chemotherapy drug utlitized for this procedure).  Storing reproductive cells prior to the treatment can ensure that the patient can have children if the patient becomes infertile after treatment. 

I hope my answer is helpful,

Sharmin


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on April 15, 2015, 10:06:56 PM
I do have a call coming up with Bluebird in the first half of May. We're getting far along enough in the trials where we should have a very good idea of what to expect in terms of both results and quality of life post trial. The next report should also have a follow up on the sickle cell patient. I'm optimistic.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on April 16, 2015, 02:57:25 AM
Thanks Andy for sharing the details.

I'm too optimistic.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on April 16, 2015, 12:23:17 PM
Thank you Andy, that is wonderful to hear.  I hope that the patients are all doing well. 


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Emby on April 17, 2015, 09:46:01 PM
Hiya Andy,

Please rest, rest, rest.
May God shower you with His special blessings.
Thank you for everything and I hope you get well soon.

Emby



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: nanuaru73 on April 18, 2015, 07:30:20 AM
 :rolleyes :rolleyes wondering when my cutiepie would be cured of this curse. there is querry can anyone help? do thal major have body temp bit higher than normal kids. My cutiepie is having fever for last 8 days and after giving course of 8 days antibiotics and negative blood and urine culture and -ve widal for typhoid and malaria antigen her temp is varying from 98.7f to 99.5f. what should i do


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on May 10, 2015, 12:25:25 AM
nanuaru73,

How is your child's temperature now? 

Gene therapy is in the trial phases at this time.  Presently adults and possibly some teens are being treated.  Once safety is better understood the trials will open to younger patients.  Focus on keeping a good hemoglobin level, low iron levels and nutritious food for now.  Gene therapy is showing great promise - it will hopefully cure all our children one day.



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: nanuaru73 on May 11, 2015, 04:15:04 AM
thanks for ur reply. she is much better now. at once stage she was investigated for t.b. also but by god grace every thing came out normal. no dignose was made and it was thought to be long case of viral fever. Is it benfi ial for her to go for swi.ming


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on June 02, 2015, 12:17:42 AM
Bluebird Bio Releases Data on Current Gene Therapy Trials - Jefferies 2015 Global Healthcare Conference

http://investor.bluebirdbio.com/phoenix.zhtml?p=irol-eventDetails&c=251820&eventID=5195088


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: JV on June 02, 2015, 07:45:49 AM
Thank you Parin for sharing...very exciting


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: JV on June 10, 2015, 07:24:46 AM
http://www.thestreet.com/story/13181244/1/bluebird-ceo-gene-therapy-study-plans-on-track-despite-request-to-go-slow-with-kids.html

Andy, what are your thoughts on this?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 10, 2015, 09:28:50 AM
JV,

I will comment after my next call with Bluebird. I could speculate, but would rather be sure.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on June 10, 2015, 03:16:57 PM
I think this is great news for anyone concerned about fertility following bmt or gene therapy:

http://www.iflscience.com/health-and-medicine/first-child-born-woman-who-had-ovary-tissue-removed-and-frozen-during-childhood



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: JV on June 10, 2015, 04:37:50 PM
Sharmin...you bring up an excellent point...


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 10, 2015, 04:52:49 PM
I have received further explanation about the RAC recommendation.

Quote
"The RAC brought up challenges intrinsic to developing novel therapeutics in the pediatric population across therapeutic areas and modalities - not unique to gene therapy trials. Ultimately, evaluating the risks and benefits for this population are decisions that are really made on an individual basis, by individual families, investigators and sites.
 
And, as our CEO told Adam Feuerstein from The Street, “We respect RAC and the process. RAC took a reasonable position when it comes to dealing with kids. We disagree and believe the decision [to proceed with a gene therapy pediatric study] should be placed in the hands of the institutional review boards, the physicians of patients and their families.
 
(My Question to Bluebird). Is Bluebird bound by RAC or can they proceed on their own?

"The outcome of the RAC review is a series of recommendations and advice, and it does not constitute a formal approval of a proposed protocol. The RAC review is specific to the U.S. and their comments are provided to the IRBs at U.S. trial sites, who individually decide on whether to follow the recommendations.
 
We do appreciate the recommendations from the RAC members and will take the RAC feedback on the timing of initiating HGB-208 under advisement. Of course, we will continue to work closely with the regulatory authorities and our clinical study sites to pursue appropriate accelerated regulatory approval pathways in the U.S. and the EU."


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: JV on June 11, 2015, 07:54:46 AM
Thank you for the follow up Andy...doesn't sound like it will be a problem but further evaluation in children cant hurt either.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 11, 2015, 10:50:00 AM
Bluebird feels that it is their decision to make and that their scientists are in the best position to know if trials with children should commence. The RAC has made a rather general recommendation covering all research using children in trials. I feel that specific therapies must stand on their own merits.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on June 11, 2015, 02:32:22 PM
Good News.

Andy,

Thanks for sharing Blue thoughts.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 13, 2015, 10:50:16 AM
The latest update from Bluebird has been released.
Very good news that gene therapy is working for the sickle cell patient, as well.

http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2058989
Quote
bluebird bio Reports New Beta-thalassemia Major and Severe Sickle Cell Disease Data from HGB-205 Study at EHA
Patient with Severe Sickle Cell Disease Producing 45% Anti-sickling Hemoglobin at Six Months and Has Been Free of Transfusions for More Than Three Months
Patients with Beta-thalassemia Major Remain Transfusion-Independent at 16 and 14 Months, Respectively
First Patient with Severe Sickle Cell Disease Infused in HGB-206 Study


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on June 13, 2015, 11:39:00 AM
Such great news Andy!  Thank you for sharing!!


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on June 14, 2015, 08:44:37 AM
This is great news! Bluebird continues to impress with its results....

Andy,
Did you get a chance to have a direct discussion with BB on their trails and near term plans?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: nanuaru73 on June 14, 2015, 12:05:01 PM
In this dark night you have shown us the light of hope. god willing this becomes the reality for masses soon. my prayers are are for them. may one day imay once again become beliver from athesist. :hugfriend


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 14, 2015, 01:38:13 PM
There has been some concern raised that Bluebird only mentioned two HbE beta thal patients in their latest report. I asked Bluebird for clarification and received this reply. I would say we shouldn't worry about any results that we have not seen.

Quote
bluebird bio is running two trials with beta-thalassemia major patients – 204 and 205. This weekend at EHA, bluebird presented 205 because that is the European trial and EHA is a European meeting. All the patients that have been treated in 205 were presented at EHA. We expect to update on 204, our US trial, at a US meeting.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on June 15, 2015, 12:00:57 AM
Andy you are rocking....

Thanks for sharing updates....

Regards,
Ashutosh


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on June 15, 2015, 10:23:52 AM
Thank you Andy,

You are extremely resourceful and thorough - thank you again for advocating and researching on our behalf.

Sharmin


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 15, 2015, 10:49:28 AM
The new rep from Bluebird is making it easy for me. Her replies are quick and informative every time.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: DrRajpal on October 02, 2015, 12:56:29 AM
Canadian family,

I have had the same thoughts about the stock price. I saw it coming. When Bluebird finally contacted me, I knew something was up. I considered buying the stock, but felt it could compromise my objectivity, so I decided against it, in spite of knowing it was going to explode. I could have made a bundle too, at a time when I could really use the money. I just couldn't do it. I have to remain objective if I am going to serve thals. It's not the first sacrifice I've made and won't be the last. I have to stay true to myself and to those I hope to help.
you are amazzing andy sir.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on October 20, 2015, 04:15:57 AM
Bluebird Shares Plunge on Waning Effect of Old Gene Therapy

http://www.thestreet.com/story/13329068/1/bluebird-shares-plunge-on-waning-effect-of-old-gene-therapy.html?puc=yahoo&cm_ven=YAHOO


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on October 20, 2015, 08:18:02 PM
The first patient Bluebird treated has seen a waning of the effects as time has passed. Bluebird is confident that their new vector is much more effective and they have confidence it will continue to work in the long term. Only time will tell.

Bluebird released this update. 

Quote
bluebird bio Announces Presentation at Cooley’s Anemia Foundation Symposium
Professor Marina Cavazzana presents overview on experience with investigational gene therapy in beta-thalassemia
CAMBRIDGE, Mass. – October 19, 2015 – bluebird bio, Inc. (Nasdaq: BLUE) announced that Marina Cavazzana, M.D., Ph.D., professor of hematology and director of the Department of Biotherapy at Hospital Necker, University Paris Descartes and lead investigator for the HGB-205 clinical study, presented a review of the clinical study experience with lentiviral-based gene therapies for beta-thalassemia at the 10th Annual Cooley’s Anemia Foundation Symposium in Chicago today.
Professor Cavazzana summarized results from HGB-205, an ongoing clinical study using LentiGlobin® BB305 for the treatment of beta-thalassemia major and severe sickle cell disease, which were presented earlier this year at the Annual Congress of the European Hematology Association. She also provided an update on subject 1003, a patient with beta-thalassemia major treated by Professor Cavazzana in 2007 in the LG001 study using the first-generation HPV569 lentiviral vector. Following approximately seven years of transfusion independence, subject 1003 has recently required two blood transfusions after experiencing clinical symptoms of anemia. Importantly, both the expression of HbAT87Q and the vector copy number in peripheral blood leukocytes, a measure of the persistence of the gene therapy, have remained largely unchanged. The safety profile for both vectors is consistent with autologous transplantation, with no gene-therapy related serious adverse events.
Development of bluebird bio’s approach to treating hemoglobinopathies was guided by the data collected from the LG001 study, a pioneering clinical study that served to highlight both the potential for gene therapy as an intervention in beta-thalassemia as well as the need for an improved lentiviral vector. To this end, Professor Cavazzana also discussed the advances in the design of lentiviral vectors for hemoglobinopathy gene therapies -- specifically the evolution from the first-generation HPV569 lentiviral vector to bluebird bio’s current lentiviral vector, BB305, which results in substantially improved vector copy number and HbAT87Q expression. “The early clinical experience with HPV569 represented a crucial proof of concept for the potential of gene therapy to bring life-changing treatment to patients in need,” said David Davidson, M.D., chief medical officer, bluebird bio. “The data from the LG001 study were invaluable to our efforts over the last five years to optimize our gene therapy approach with improvements to the potency, robustness and manufacturing for the next-generation lentiviral vector, BB305. LentiGlobin BB305 drug product is being evaluated in three ongoing clinical trials for the treatment of patients with beta-thalassemia major and severe sickle cell disease, and three abstracts related to these ongoing clinical trials have been accepted for presentation at this year’s American Society of Hematology’s Annual Meeting in December.”


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on October 21, 2015, 02:40:22 PM
A bit more follow up on this first case.

Quote
This patient was treated using an old version of the gene therapy, and tests show that the amount of hemoglobin being produced by the gene-therapy-modified stem cells remained stable. This suggests that the persistence of the gene therapy, even though it’s an older version, did not wear off. However, of course, this is only 8 years after the treatment.
 
No one at the company has spoken with the patient (obviously!), and all treatment decisions are made by the patient and their physician. Regardless of a patient’s hemoglobin level, we recognize that the patient may feel more tired or have other anemia symptoms simply because of other factors, and the decision to transfuse is always be made by the patient and his or her care team.
 
We’ll have more data on the patients being treated with our current version of the gene therapy treatment at ASH later this year. I will email you as soon as any data become publicly available.

Nothing has changed. Panic in the stock market is unwarranted.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on October 22, 2015, 11:55:26 AM
Andy,

Being in stock markets for the last 15 years, I can say that stocks are speculative in nature (99% of the time). Remember Bluebird was just trading $17 last year, after the announcement of human trials the stock shooted to $181 in few months. The answer is speculation, Bluebird has not made any money from its technology, it is on trails basis but stock is trading $81 these days. It came down quickly because news about competition (SKMCC).

News, speculation, big whales at Bay street and Wall street manipulates money and gets bigger.

Note to others: I do not own Bluebird but watch the stock movement as we need this company to succeed.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on October 27, 2015, 03:01:55 PM
That is very interesting Canadian Family, like you we are invested in bluebird in a far more important way.  They may hold the key to curing our children. 

Sharmin


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on October 28, 2015, 01:36:00 PM
Agreed. Our hopes and prayers are our investment in Bluebird.

Kindly,


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on November 08, 2015, 09:29:27 AM
The recent news regarding Bluebird Bio's trials has been very positive for Beta + but not as great for Beta 0s.  All patients have experienced improvement but 2 of the beta 0s have required one transfusion each and one is still dependent.  I wonder however if more time is required to see the benefits for these patients.  Beta 0s require a greater level of expression than Beta +.  I'm glad there is improvement in all of the patients - I hope the team continues to fine tune the treatment until everyone can be cured.   We are closer than we have ever been. 

As an aside, I personally believe SKs vectors with Bluebird's preparation and diligence would be superior.  Hopefully we will see carefully planned trials from SK soon.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on November 08, 2015, 09:53:53 AM
Thank you for sharing. I echo your comments on SK as being superior.

After so many years of wait, no one wants a cure with side effects of treatment impacting the quality of life of patients. Although, at this point there is no perfect cure in progress but patient well being should be supreme.



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on November 08, 2015, 12:21:10 PM
I hope and pray that these companies continue to fine tune these procedures and bring them forward in the best interest of patients. 

I'm looking forward to hearing Bluebird's results at the end of this year. 

I hope that SK allows the cure that they are sitting on to get to the patients who need it. 


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on January 12, 2016, 10:50:09 PM
J.P. Morgan Healthcare Conference

http://phx.corporate-ir.net/External.File?t=1&item=VHlwZT0yfFBhcmVudElEPTUyMTM4MDF8Q2hpbGRJRD02MDY1NDI=

if you unable to get attachement above click below link

http://investor.bluebirdbio.com/phoenix.zhtml?p=irol-eventDetails&c=251820&eventID=5213801


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on January 13, 2016, 03:58:48 PM
Thank you for sharing Parin!


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on January 13, 2016, 07:48:28 PM
My broker called me today that share price declining as investors and funds are getting impatient about the trial results. Bluebird is planning to announce results in second half of 2016.

Note: I do not have a share position in Bluebird, but keep an eye on corporate development.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on January 13, 2016, 08:18:12 PM
That is very interesting Canadian Family.  Hoping for the best!


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on January 14, 2016, 08:56:03 AM
Excerpt from Motley Fool Article...

bluebird bio's shares plunged on Monday after investors got their first look at the company's presentation slides and didn't like what they saw. Traders sold off shares on the news that it would not be releasing updated clinical data on LentiGlobin BB305 until the next American Society of Hematology meeting in November. Investors had grown accustomed to seeing fresh data in June so the extended waiting period caused many to flee.

During its question-and-answer session with analysts, CEO Nick Leschly was asked about the rationale behind pulling back on its clinical-data reporting schedule. Leschly explained that its LentiGlobin trials have moved beyond the "proof of concept" phase and now they key clinical questions revolve around the therapy's durability. Judging the durability of LentiGlobin requires much more time to gather the clinical data, so the company felt that it didn't make sense to release partial data in June.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on January 14, 2016, 11:24:20 AM
We live in an impatient world where people expect instant gratification. I feel that one unfortunate aspect of Bluebird's openness has been that people expect updates every other week. I don't know of any drug trial where updates are made until a phase of the trial is completed, yet it's expected that we will get weekly updates on gene therapy, which will take longer to perfect than most drugs. I think the investment community has become just as affected by the need for instant gratification as anyone else. It will be really sad if this new normal results in new drugs and therapies being given up on and not funded because it's not happening fast enough to appease the immature investors. And the more I read the pharma biz press, the more I compare them to sportswriters, whose main goal is to tear down athletes. I see the same phenomenon taking place with these biz writers, who act like vultures waiting to swoop in and pronounce failure on therapies and drugs that are still in trials.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on January 14, 2016, 12:34:27 PM
Ah Andy,

U are hitting the right cord. This market is brutal, I have seen impatient people loosing their shirts as they are looking for 'Fast Money'. This market does not work on performance rather than on news (people are short). Its unfortunate but true. Big whales at TSX and Wall Street manipulate small investors like vultures......

I don't even want to say anymore as I have seen enough over the years.

What's important for Blue is to ignore the shorts and keep on doing what they do. However, low share price does impact Blue's capacity to run smoothly. Financials do matter for R&D companies like Blue.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on March 21, 2016, 03:04:05 AM
Hi Andy,
I am not getting impatient but wanted to know when we can expect an update from Bluebird on the thal trails?

warm regards,
R


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on April 25, 2016, 12:30:29 AM
Hello Andy,
is there any info. on when we can expect more data on bluebird trails??

best regards,
R


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on April 25, 2016, 10:36:56 AM
Hello catchR, I don't think bluebird has released any further information at this time. 


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on April 25, 2016, 10:50:04 AM
Don't expect any updates until the ASH meeting in December.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on April 26, 2016, 03:02:17 PM
Thanks Andy.

Any update from SMKCC trials....


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on April 26, 2016, 04:31:24 PM
Hello Canadian_Family,

I hope that you are feeling better. 

As for the SMKCC trials, I feel like SK has swallowed the vector and all of Errant Gene's rights to it. I don't know if they will ever follow through with the trials because their results were not as remarkable (marketable) as bluebird bio's initial results.  Likely because of the low intensity conditioning.

I am still a fan of Sadelain's vector and hope that trials will continue. 


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on April 26, 2016, 04:47:00 PM
FYI, I should have a call with bluebird late in May. I don't know if they'll give me any new info or if it's just so the new rep can introduce herself.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on April 27, 2016, 12:18:14 PM
That's great Andy, I look forward to hearing what they have to say.  Hopefully the e-beta thals continue to do well - I'm sure they are ambitiously pursuing a superior vector that can treat beta zero. 



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on April 28, 2016, 09:42:50 AM
Thanks Sharmin. I recovered and doing fine.

SMKCC methodology and idea was very appealing to beta thal 0 (low intensity regimen). It would be a shame if SMKCC will not follow through with the trials. I know that estimated study completion date is July 2016. I will keep my fingers crossed till that date.

Andy,

Thanks for maintaining the relationship with Bluebird. Hope you continue to get information from them.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on May 06, 2016, 12:19:09 AM
Latest article on gene therapy: Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects

Authors: Jorge Mansilla-Soto,1 Isabelle Riviere,1 Farid Boulad,1,2 and Michel Sadelain1,*

Also attached is the full text PDF of the article.

warm regards,
R


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on May 06, 2016, 10:24:25 AM
Thank you for sharing catchR.  This is all good news.  Progress is steady - sometimes feeling slow but it is progress and promise of a healtyh future for our families.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on May 10, 2016, 06:39:09 PM
Thanks for that review. I learned more about some aspects of bluebird's process from this article than I have from talking to doctors at bluebird.

Again, it seems that these groups revolving around Dr Sadelain's work have made a new major advance in terms of effect and safety with their modified vector.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on May 12, 2016, 12:27:43 PM
I agree Andy.  My hope is revived in this group.  I hope the meetings in June go well.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: djema on May 18, 2016, 07:51:05 PM
Hello friends !
do not lose hope when I read Your posts .
only to Russia not soon come to gene therapy for such patients like my daughter .
here are a few of the disease thalassemia ! we in our city alone . the city of Krasnoyarsk.
and Your only message I learn anything new about thalassemia .
health to You friends .
djamala. :bighug


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on June 07, 2016, 04:42:18 AM
Dear Andy,

did you have your planned call with bluebird bio? pls let us know if there are any updates.

warm regards,
R


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on June 07, 2016, 01:26:59 PM
CatchR,

There is a  Goldman Sachs 37th Annual Healthcare Conference (Live)
06/08/16 at 8:00 a.m. PT

http://investor.bluebirdbio.com/phoenix.zhtml?p=irol-eventDetails&c=251820&eventID=5228749


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 07, 2016, 07:03:13 PM
The call from Bluebird was more introductory. They are continuing to expand trials and will be doing separate trials for beta zero patients than for beta+. Expect some changes in dosing, etc. I expect that they will continue to work on the vector, as well, just as SK has.

The link to the long term clinical trials is at https://clinicaltrials.gov/ct2/show/NCT02633943?term=bluebird+bio&rank=1

Here is the summary of SCD data from ASH 2015:
HGB-206 is an ongoing, open-label Phase 1 study designed to evaluate the safety and efficacy of LentiGlobin BB305 product candidate in the treatment of subjects with severe sickle cell disease (SCD). We recently announced the expansion of this study from eight subjects to 20 subjects, in order to collect more data and have flexibility on regulatory options and timelines. The study is evaluating safety and efficacy as measured by changes in red cell function tests and hemolysis markers, as well as clinical events secondary to SCD, including vaso-occlusive crises or acute chest syndrome events. As of November 17, we have manufactured drug product for four patients with severe SCD and three have been infused.

Drug product vector copy number (VCN) was 0.5/0.6 in Subject 1301, 1.3 in Subject 1303 and 0.6 in Subject 1306.
VCN in peripheral blood leukocytes at three months follow up was 0.04 in Subject 1301 and 0.11 in Subject 1303.
Early data on Subjects 1301 and 1303 with greater than three months of follow up, show a gradual increase in HbAT87Q levels:
At the six-month post-infusion follow up for Subject 1303, the proportion of anti-sickling hemoglobin accounted for 16 percent of all hemoglobin production (12 percent HbAT87Q + 4 percent HbF).
At the three-month post-infusion follow up for Subject 1301, the proportion of anti-sickling hemoglobin accounted for 17 percent of all hemoglobin production (4 percent HbAT87Q + 13 percent HbF).
Longer follow up data and additional subjects are required to determine the extent of HbAT87Q production and clinical impact of LentiGlobin BB305 in severe SCD.
The safety profile in the infused patients is consistent with autologous transplantation and no drug product-related grade >3 adverse events have been reported.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on June 09, 2016, 11:07:27 AM
Thank you..Andy for sharing the updates.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Nomad on June 09, 2016, 01:29:11 PM
Andy, what about  (B° B+) ?
Does that change the odds?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 11, 2016, 11:57:06 AM
It depends on the specific beta+ mutation and how it reacts with beta zero. Many patients with that genotype have full blown thal major and would not be helped enough by the current level of gene expression seen in trials, to avoid transfusions. I think the process will improve. It is already far beyond where it was in 2010.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: BabyRiya on July 31, 2016, 08:41:52 PM
Thank you


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on August 04, 2016, 08:07:27 AM
BlueBird announced its financial results yesterday. Important announcement for us was below:

“In the second half of 2016, we look forward to continued progress on our clinical programs, including initiation of the LentiGlobin HGB-207 Phase 3 study in non-β0/β0 transfusion-dependent thalassemia (TDT), the integration of manufacturing process improvements into our LentiGlobin clinical trials, and presenting updated LentiGlobin clinical data at ASH.”

I only question if they have any success with B0/B0 transfusion dependent thalassemia. I did not see any reference to that in the conference call.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on August 04, 2016, 05:56:29 PM
The beta 0 trials have been split off into their own trials now, with their unique protocol. I don't know if we'll see any updates soon on it. Maybe at ASH in December. My understanding is that the doctors running trials are confident in the eventual success of gene therapy in all forms of thalassemia. My own observation on this process since it began years ago is that there is constant refining and improvement of the vector and the understanding of both dosing and preparation regimen. SK has a new vector that is unique and will give them control over the new genes so they can't get out of control, removing the main concern about gene therapy having any potential dangers. In the long term, it looks quite good.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on August 05, 2016, 08:37:33 AM
Thanks Andy.

Appreciate the clarification.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on August 05, 2016, 01:41:03 PM
Thanks Andy and Canadian Family for the updates...



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on September 08, 2016, 09:49:13 AM
bluebird bio Inc Opens Phase 3 Study of LentiGlobin™ Drug Product in Patients with Transfusion-Dependent Beta-Thalassemia

http://www.smarteranalyst.com/2016/09/08/company-update-nasdaqblue-bluebird-bio-inc-opens-phase-3-study-lentiglobin-drug-product-patients-transfusion-dependent-beta-thalassemia/



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on September 08, 2016, 10:28:41 AM
Thank you Canadian_Family for sharing...Its good news for all patients and parents.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on September 08, 2016, 01:42:27 PM
Quote
In this study, the process by which the patient’s cells are transduced with LentiGlobin will be modified by the addition of two additives during the transduction step of the manufacturing process, intended to increase vector copy number and the percentage of cells successfully transduced.

I know some get discouraged quickly over early results in some patients, but this process continues to be refined and improved. My understanding is that the doctors running the trials are very optimistic about the eventual outcome, including that for beta zero patients. Patience is needed.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on September 08, 2016, 02:34:42 PM
Andy,

Can you please explain this more..i didnt get that. "addition of two additives during the transduction"


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on September 10, 2016, 03:24:38 PM
The hoped for result of these new additives to the process is "intended to increase vector copy number and the percentage of cells successfully transduced" What this means is that more beta globin should be produced with these changes, which would lead to a greater increase in Hb. Even though this particular study is for non beta zero patients, these changes should mean that the process will also mean a higher increase in Hb in beta zero patients.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: BabyRiya on September 10, 2016, 08:33:52 PM
Andy
If this medicine comes in 4 or 5 years and takes care of transfusion, will there be need for chelation for Thal intermedia
Can we do anything to help expedite?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on September 10, 2016, 11:06:07 PM
Thanks Andy, i hope  beta zero patients could use this therapy soon.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on September 17, 2016, 12:13:36 PM
BabyRiya, if a patient undergoes gene therapy, they will need chelation if they were already carrying an iron load. They will not be building a new iron load, though.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: BabyRiya on September 18, 2016, 10:46:40 PM
Thx Andy


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on October 12, 2016, 02:22:20 PM
Hi Andy,

I just browsed through ASH conference preliminary program in 2016. I did not see any participation from Bluebird. Do you know if they dropped out from ASH.

http://www.hematology.org/Annual-Meeting/Program/

I see Bluebird is presenting in Gene Therapy Day seminar on October 13, 2016.

http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-calendar

Keeping fingers crossed.....??



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on October 13, 2016, 09:55:06 AM
October 13, 2016 highlights. Read for full details....

http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2211455

Important update:

LentiGlobin Manufacturing Data: A Head-to-Head In Vitro Comparison of Process 1 and Process 2
bluebird bio has recently modified the process by which the patient’s cells are transduced in LentiGlobin clinical studies with the addition of enhancers during the manufacturing process. The goal of manufacturing Process 2 is to increase the percentage of cells successfully transduced, thereby increasing vector copy number (VCN) in the drug product that is given to the patient.

Using retained samples of CD34+ stem cells collected from patients in the HGB-204 (Northstar) and HGB-206 studies, the company was able to demonstrate in a head-to-head in vitro comparison that manufacturing Process 2 substantially increased the percentage of cells transduced and VCN, as compared to manufacturing Process 1.

This in vitro data from Process 2 showed an average increase of approximately three-fold in vector-positive cells and VCN across all patient samples tested. Process 2 has been successfully scaled up for clinical manufacturing, and all LentiGlobin clinical trials moving forward will use manufacturing Process 2, including the Phase 3 HGB-207 (Northstar-2) trial and the Phase 1 HGB-206 clinical trial.

bluebird bio also highlighted progress it has made in moving from adherent manufacturing of lentiviral vectors to potentially more efficient suspension manufacturing, consistently achieving the targeted potency, purity and VCNs at increased scale.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on October 14, 2016, 08:50:46 AM
That's some good news, hopefully bluebird gets there quickly.

regards,
R


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on October 14, 2016, 07:22:10 PM
Wow, things are moving with some pace


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on October 15, 2016, 12:24:16 PM
I emailed the new rep. Hopefully, I will get a reply.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sharmin on October 18, 2016, 11:37:34 AM
Canadian_Family,

Thank you kindly for sharing this is great news for all thal patients - especially beta zeros!



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on October 19, 2016, 11:10:14 AM
My personal take on the news is that there is not much to report in December during ASH conference. The enhancers will take time and new data will not be available until 2017 and beyond. The catalyst that Blue will show positive data from process 1 is wasted. Looking forward to 2017 and beyond.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on October 19, 2016, 05:07:25 PM
Bluebird will be presenting on 204, 205 and 206 at ASH


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on October 20, 2016, 10:48:47 AM
Thanks Andy.

Is it 204, 205 and 206 are beta Zero?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on October 20, 2016, 12:35:33 PM
Thanks for the update Andy. Helpful.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on November 03, 2016, 09:29:32 AM
Update Nov 3, 2016

In Northstar, the largest study of gene therapy in patients with TDT to date, all patients have demonstrated robust expression of therapeutic hemoglobin, or HbAT87Q, with a safety profile that is consistent with autologous transplantation. We are particularly pleased to see that, across the Northstar and HGB-205 studies, all patients with non-β0/β0 genotypes and at least 12 months of follow-up remained free of transfusions for up to 31 months, adding to the growing body of evidence supporting the potential for LentiGlobin to become a transformative therapy for these patients. Additionally, we have seen clinically meaningful reductions in transfusions for patients with β0/β0 genotypes. We believe that the implementation of manufacturing process 2 going forward has the potential to further reduce transfusion requirements for patients with β0/β0 genotypes, and we will evaluate that hypothesis in our planned Phase 3 HGB-212 trial.

Details http://www.streetinsider.com/

Abstract Results, as of June 27, 2016 Data Cut-off:

Eighteen patients have received LentiGlobin drug product: eight with β0/β0 genotypes and 10 with non-β0/β0 genotypes
The median vector copy number (VCN) in drug product (DP) was 0.7 (range: 0.3 – 1.5 copies/diploid genome) and the median cell dose was 8.1 x 106 CD34+ cells/kg (range: 5.2 – 18.1 x 106 cells/kg)
The median in vivo VCN in patients with at least six months of follow-up was 0.4 copies/diploid genome (range: 0.2-1.0; n=13)
Patients of all genotypes with at least six months of follow-up (n=14) achieved a median HbAT87Q level of 4.7 g/dl and robust HbAT87Q production was sustained in the 10 patients with ≥12 months of follow-up
All patients with non-β0/β0 genotypes and at least 12 months follow-up (n=5) have discontinued transfusions and remain free of transfusions (median 19.4 months without transfusion; range: 15.3 to 24.0 months)
All patients with β0/β0 genotypes have considerably reduced transfusion requirements (median 60% reduction; from median 171.9 ml/kg/year at baseline [range: 168.1- 223.2 ml/kg/year] to 67.8 ml/kg/year post-treatment [range: 14.8 to 123.7 ml/kg/year])
The safety profile remains consistent with autologous stem cell transplantation with no ≥ Grade 3 drug product-related adverse events (AEs) reported.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on November 03, 2016, 02:41:26 PM
Bluebird's link on this announcement. http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2219192



Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on December 14, 2016, 11:09:55 AM
bluebird bio Announces First Patient Treated with LentiGlobinTM Drug Product in Northstar-2 (HGB-207) Phase 3 Trial of Patients with Transfusion-Dependent β-Thalassemia

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec. 14, 2016-- bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced treatment of the first patient in Northstar-2, the Phase 3 study of its LentiGlobin drug product in patients with transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes. This study will use LentiGlobin drug product manufactured with the addition of transduction enhancers intended to increase the drug product vector copy number and percent of cells transduced. The study’s primary endpoint is the proportion of treated subjects who meet the definition of "transfusion independence," defined as total hemoglobin levels of at least 9g/dL without any RBC transfusions for a continuous period of at least 12 months at any time during the study.

“The opening of our first Phase 3 trial in TDT is an exciting milestone for bluebird,” said David Davidson, chief medical officer. “At ASH we presented interim LentiGlobin data from the Northstar study showing substantial and durable treatment effects, with all patients with non-β0/β0 genotypes and at least twelve months of follow-up achieving freedom from transfusions. We are hopeful that our new manufacturing process incorporating transduction enhancers will build upon these results and may provide even better outcomes for patients. It is an encouraging start for Northstar-2 to have achieved a robust drug product VCN of 2.9 c/dg and 77% LVV+ for our first treated patient. We are tremendously grateful to the patients, families, study investigators, and site staff who have participated in our LentiGlobin program and enabled progress toward our goal of improving the lives of patients with TDT.”

“The current results of the HGB-204 and HGB-205 LentiGlobin studies show a reduction, and in some cases, elimination of RBC transfusions in patients with TDT,” said Mark C. Walters, M.D., UCSF Benioff Children’s Hospital Oakland, a principal investigator on the study. “The current study, Northstar-2, will test a new manufacturing method to increase the VCN in the drug product. If it is successful, this new enhanced transduction method could expand the number of patients who no longer need transfusions after the gene therapy treatment.”

bluebird bio is also moving forward with plans to initiate Northstar-3 (HGB-212), a Phase 3 trial of LentiGlobin drug product in patients with transfusion-dependent β-thalassemia with the β0/β0 genotype. This study will also be conducted under the new manufacturing process, and is expected to begin enrolling patients in 2017. The primary endpoint of this planned study is transfusion reduction.


http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2229663


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on February 03, 2017, 09:28:24 AM
First SCD patient treated under the amended study protocol in HGB-206.  For details pl. follow the link.

http://finance.yahoo.com/news/bluebird-bio-announces-first-patient-130000772.html


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on February 04, 2017, 06:57:09 PM
Thanks for sharing..its good news.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Hafiz Akhtar on February 08, 2017, 11:35:11 PM
canadian Family. pl inform when will Thal cure. if not fully cured the transfusion burden may b reduced. v r tired now. believe me


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on June 23, 2017, 11:37:39 AM
http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2282785

bluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Dependent β-Thalassemia (TDT) and Severe Sickle Cell Disease (SCD) at European Hematology Association (EHA) Annual Meeting

–Ongoing transfusion independence up to 3.5 years in patients with transfusion-dependent β-thalassemia (TDT); three patients have discontinued iron chelation –


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 24, 2017, 12:47:59 PM
You beat me to it. In was about to post this the other day when I saw you had posted.

Overall, this report was good news.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Pratik on June 26, 2017, 08:48:37 AM
Would this require Chemo?

Was there any other drug that produced Hb too?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on June 26, 2017, 06:55:13 PM
Pratik, chemotherapy is required to prepare the patient for the procedure. Gene therapy is a permanent cure. It is very good news that some of the earlier patients in the trials are no longer chelating. Also, their new product appears to be improved, which is good news as they head to a beta zero only trial.

The drug we've been following that increases Hb is Luspatercept. This is not a cure, but an ongoing treatment, but it will probably be the more easily accessible, as gene therapy will come with a high price tag. Phase 3 trials for Luspatercept are now full and underway. There is a good chance it will hit the market by 2020 if the trials go well. The early trials have gone exceptionally well. Depending on the transfusion needs of the patient, this drug should eliminate the need for transfusion in intermedia patients, including many with HbE beta thal, and also greatly reduce the transfusion and chelation requirements of almost all thals. The drug reduces ineffective erythropoiesis, which lowers iron absorption, while also reducing the amount of iron being taken from transfusions. I anticipate that many patients currently using drugs like hydroxyurea and thalidomide will switch to Luspatercept once it is available. I see the other drugs as a temporary measure until better comes along. Better is really close.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Sunny2701 on April 14, 2018, 03:26:39 PM
Thats a great insight Andy. Are there any new updates on it since your last post?


Pratik, chemotherapy is required to prepare the patient for the procedure. Gene therapy is a permanent cure. It is very good news that some of the earlier patients in the trials are no longer chelating. Also, their new product appears to be improved, which is good news as they head to a beta zero only trial.

The drug we've been following that increases Hb is Luspatercept. This is not a cure, but an ongoing treatment, but it will probably be the more easily accessible, as gene therapy will come with a high price tag. Phase 3 trials for Luspatercept are now full and underway. There is a good chance it will hit the market by 2020 if the trials go well. The early trials have gone exceptionally well. Depending on the transfusion needs of the patient, this drug should eliminate the need for transfusion in intermedia patients, including many with HbE beta thal, and also greatly reduce the transfusion and chelation requirements of almost all thals. The drug reduces ineffective erythropoiesis, which lowers iron absorption, while also reducing the amount of iron being taken from transfusions. I anticipate that many patients currently using drugs like hydroxyurea and thalidomide will switch to Luspatercept once it is available. I see the other drugs as a temporary measure until better comes along. Better is really close.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on April 20, 2018, 04:37:21 PM
Yes, there is an update and you posted it. Hehe. The big news is that approval will be sought in Europe this year.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: nijaguna on April 24, 2018, 11:45:42 PM
Pratik, chemotherapy is required to prepare the patient for the procedure. Gene therapy is a permanent cure. It is very good news that some of the earlier patients in the trials are no longer chelating. Also, their new product appears to be improved, which is good news as they head to a beta zero only trial.

The drug we've been following that increases Hb is Luspatercept. This is not a cure, but an ongoing treatment, but it will probably be the more easily accessible, as gene therapy will come with a high price tag. Phase 3 trials for Luspatercept are now full and underway. There is a good chance it will hit the market by 2020 if the trials go well. The early trials have gone exceptionally well. Depending on the transfusion needs of the patient, this drug should eliminate the need for transfusion in intermedia patients, including many with HbE beta thal, and also greatly reduce the transfusion and chelation requirements of almost all thals. The drug reduces ineffective erythropoiesis, which lowers iron absorption, while also reducing the amount of iron being taken from transfusions. I anticipate that many patients currently using drugs like hydroxyurea and thalidomide will switch to Luspatercept once it is available. I see the other drugs as a temporary measure until better comes along. Better is really close.

Maybe I am wrong but will the gene therapy be tried on minors as well? Does the gene therapy, if successful, be able to eliminate the genes from the germline thereby preventing future generations from the disease?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Canadian_Family on April 25, 2018, 10:51:25 AM
Hi,

Minors do not need a gene therapy considering its risks and cost. Prevention is the only way to eliminate thalassemia. All thal minors should check in with their potential spouses if they have thal minor. Take a calculated risk....


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on April 29, 2018, 12:18:02 PM
Agreed with CF. Because it requires chemo conditioning, it would not be considered for minors.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: nitarora on April 30, 2018, 09:04:45 AM
Hi Andy,

I am a silent Visitor for this website since long.. Now feeling to get some query addressed

My son is Major one & we are regularly Visting Dr. Vijay Pune-India. Now My query to you is Besides Blue bird I heard from Dr. Vijay that Gene Therapy  had been successfully Tested in France too by Dr. Marina Cavazaana.

Do you  have any Informationfor the same?

BR//
Nitin Arora


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on May 09, 2018, 08:17:15 PM
Professor Cavazzana is the primary investigator of the HGB-205 study for bluebird.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on July 28, 2018, 01:58:47 AM
bluebird bio’s LentiGlobin™ Gene Therapy Granted Accelerated Assessment by European Medicines Agency for the Treatment of Transfusion-Dependent β-Thalassemia

http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bios-lentiglobintm-gene-therapy-granted-accelerated


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on July 28, 2018, 02:45:05 PM
It's very close to a reality.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on July 30, 2018, 12:20:21 AM
Yes, any idea about USA?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on July 31, 2018, 08:25:51 PM
No idea yet about the US, but the reason why Europe was chosen first is explained in a very in depth article at http://www.pmlive.com/pharma_intelligence/Bluebird_Bio_on_the_cusp_of_a_gene_therapy_revolution_1246188


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: princessaurora on October 27, 2018, 10:54:49 AM
Good day my daughter Aurora was recently diagnosed as being beta thalesssemia
 intermediate we live in the Caribbean as a parent I would like to know how I can get my daughter cured


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on November 03, 2018, 01:56:46 PM
Right now, the only approved cure is a bone marrow transplant. This is usually only done if the patient is transfusion dependent. It should only be a few years before gene therapy will be approved and that will also definitely cure intermedia patients. Does the child take blood transfusions?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on November 12, 2018, 10:56:18 PM
bluebird bio Q3 Update

http://investor.bluebirdbio.com/static-files/37fe317c-5c01-43af-9c1d-2decb9d3800d





Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on November 29, 2018, 03:21:38 PM
We should see a new update after next week's presentation at ASH.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on December 03, 2018, 11:42:19 PM
First presentation of data from patients with a β0/β0 genotype and a pediatric patient treated with LentiGlobin in Phase 3 Northstar-3 study both have stopped chronic blood transfusions

10 of 11 patients with non-β0/β0 genotypes and more than three months follow-up have stopped chronic transfusions in Phase 3 Northstar-2 study

Indicators of poor red blood cell production appear corrected in exploratory analysis of bone marrow following treatment with LentiGlobin


http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-presents-new-data-lentiglobin-gene-therapy


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 04, 2018, 09:29:07 AM
The results are spectacular. After much negativity from some quarters about the earlier results from the non beta zero trials, we have fantastic news. Yes, only 3 patients have been started so far, but all 3 beta zero patients are transfusion free. The new protocol is working.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: tahirkhan on December 04, 2018, 02:38:02 PM
Great news .
Dear Andy when can be this treatment available in market ?


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Andy Battaglia on December 04, 2018, 05:49:50 PM
bluebird bio has begun the approval process in Europe. It's only a guess, but I would hope that within two years, we will see the first approval for gene therapy for thalassemia.


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on December 05, 2018, 01:00:06 AM
Thanks Andy


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on March 31, 2019, 01:13:30 AM
bluebird bio Receives Positive Opinion from CHMP for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia (TDT) Who Do Not Have β0/β0 Genotype

http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-receives-positive-opinion-chmp-zynteglotm


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: catchR on April 02, 2019, 06:30:06 AM
Finally....some exciting news!!


is gene therapy from bluebird going to cost 2.12 mUSD??


Title: Re: Bluebird Bio Releases Data on Current Gene Therapy Trials
Post by: Parin on April 02, 2019, 09:08:48 PM
2.12 m USD?   :rolleyes :rolleyes