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Author Topic: Bluebird Bio Releases Data on Current Gene Therapy Trials  (Read 94915 times)
Sharmin
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Little A


« Reply #120 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. 
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Andy Battaglia
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« Reply #121 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.
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Andy

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Sharmin
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Little A


« Reply #122 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. 

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Sharmin
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« Reply #123 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.
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Regards.
catchR
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« Reply #124 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
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Sharmin
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Little A


« Reply #125 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.
« Last Edit: May 12, 2016, 12:27:00 PM by Sharmin » Logged

Sharmin
Andy Battaglia
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« Reply #126 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.
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Andy

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Sharmin
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Little A


« Reply #127 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.
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Sharmin
djema
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« Reply #128 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.
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catchR
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« Reply #129 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
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« Reply #130 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
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Andy Battaglia
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« Reply #131 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.
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Andy

All we are saying is give thals a chance.
Parin
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« Reply #132 on: June 09, 2016, 11:07:27 AM »

Thank you..Andy for sharing the updates.
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« Reply #133 on: June 09, 2016, 01:29:11 PM »

Andy, what about  (B° B+) ?
Does that change the odds?
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Andy Battaglia
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« Reply #134 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.
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Andy

All we are saying is give thals a chance.
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