Bluebird Bio Releases Data on Current Gene Therapy Trials

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.

This is great Andy, thank you for all your efforts

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?

Thanks Andy...for sharing the great news..hope soon it will available to all....

This is really amazing news, thank you Andy for sharing the updates!! Awaiting results from Dr. Sadelain on their trails...

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

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.

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

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.

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).

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.

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

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.

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.

Andy, Truly appreciate your self-less efforts for the good and betterment for all the thals across the word!!


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