Thalassemia Patients and Friends

Discussion Forums => Thalassemia Major => Topic started by: Harshul on April 15, 2014, 02:45:30 AM

Title: Gene Therepy
Post by: Harshul on April 15, 2014, 02:45:30 AM
Check this out guys. If you know any progress report, then please tell me.

http://www.bizjournals.com/boston/blog/bioflash/2014/03/bluebird-bio-begins-trial-of-second-drug-for-rare.html?page=all
Title: Re: Research
Post by: Andy Battaglia on April 15, 2014, 02:59:39 AM
This is the gene therapy trial being run by Bluebird. Their is another trial going on in the US that has been going for a longer period. We expect an update by this summer.
Title: Re: Gene Therepy
Post by: Harshul on April 15, 2014, 06:04:31 AM
Hope they succeed.  :thumbsup
Title: Re: Gene Therepy
Post by: Bostonian_04 on May 07, 2014, 12:09:37 AM
Summer is almost here. Hoping for the best
Title: Re: Gene Therepy
Post by: Sharmin on May 07, 2014, 04:47:03 PM
Hoping for good results from the various trials taking place.
Title: Re: Gene Therepy
Post by: souly on May 08, 2014, 09:03:09 AM
Wishing all the patients on trial a very safe and success. I am thrilled to wait for the outcome.
Title: Re: Gene Therepy
Post by: Canadian_Family on May 08, 2014, 02:32:51 PM
I saw the article and hope their trial is a success. The company is listed on NASDAQ, share is trading around $20, I am not sure if this company is in the business of making money or really thinking to benefit the thal patients. They expect to charge $450,000 (approx) for one treatment REALLY !!!!!!

$750MM in revenue first year, this does not sound right to me.

I question the way they project themselves.

Title: Re: Gene Therepy
Post by: Andy Battaglia on May 08, 2014, 02:46:23 PM
I too, have many questions about Bluebird. My requests for information about their process resulted in nothing more than a few emails back and forth and zero information being shared. This is the complete opposite of what we have grown accustomed to from Dr Sadelain. I don't want to make judgments based on this, but transparency would be appreciated and we are not getting that from Bluebird.
Title: Re: Gene Therepy
Post by: Pratik on May 08, 2014, 03:43:37 PM
Companies trying to make a buck is nothing new. Sadly, we all are "customers" to every product we use - be it Exjade or gene therapy - we will be charged too for it but that's okay. That's how this world works.
Title: Re: Gene Therepy
Post by: JV on May 08, 2014, 06:52:44 PM
Completely agree with Andy about Bluebird...from personal experience as well. They also gave 25,000 to Cooley's Anemia this past year but refuse to answer specific questions.
Title: Re: Gene Therepy
Post by: Andy Battaglia on May 08, 2014, 09:48:07 PM
What I find ironic about Bluebird is that they are preventing their PR department from giving them the PR that could put them in a more positive view in the public's eye.
Title: Re: Gene Therepy
Post by: Canadian_Family on May 09, 2014, 07:50:36 PM
By no means this is an investment advice, I am trying to assess the viability of BlueBird to continue in operation (which eventually helps Thalassemia patients).

bluebird bio to Present at Three Upcoming Health Care Conferences

For details : http://www.nasdaq.com/press-release/bluebird-bio-to-present-at-three-upcoming-health-care-conferences-20140501-01726#ixzz31FVUPVaL

I checked the volume of share trade, it is not high. Bluebird is featured in Investopidia as one of three companies to profit from.

The liquidity is strong and revenues have grown since 2011.

Conclusion

I believe this company is not strapped for cash. However, there is always a responsibility to generate profits for shareholders, which means expensive treatments for patients.
Title: Re: Gene Therepy
Post by: Andy Battaglia on May 10, 2014, 05:17:11 PM
They are well financed, but no one is allowed to talk about what they're doing. The lawyers are in charge. Can't say that's a good thing.
Title: Re: Gene Therepy
Post by: leonardo on May 30, 2014, 01:01:57 PM
But how we can talk about mutagens drug for beta thal when gene therapy is on second step of trial?  .....Ivano Argiolas from Italy is still testing therapy......