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[Sharmin]

Andy,

Thank you for your constant service to thalassemia patients. 

[Canadian_Family]

Hi Andy,

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

[Rajiv]

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

[EugenioLaMesa]

Dear Andy,

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

Best
Eugenio

[Sara76]

I echo the above sentiments! 
The whole world would benefit from more people like Andy!

 
Suelou

[Bostonian_04]

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.

[catchR]

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

[Andy Battaglia]

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.

[Sharmin]

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!

[Parin]

All the very best....hope for the best only..

Regards,
Ashutosh

[bomboi]

When is the next update on the study , i read somewhere that it should be coming soon

[Andy Battaglia]

I believe it is going to be updated at the ASH conference this month.

[Canadian_Family]

Lot of insiders selling the stock recently. Does it mean anything, probably not or perhaps yes.....

[Sharmin]

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...

[Andy Battaglia]

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.