Discussion Forums > Working Towards a Cure

Bluebird Bio Releases Data on Current Gene Therapy Trials

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Rajiv:
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

Andy Battaglia:
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.

Sushil:
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

Andy Battaglia:
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.

Canadian_Family:
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).

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