Bluebird Bio Releases Data on Current Gene Therapy Trials

Good day my daughter Aurora was recently diagnosed as being beta thalesssemia
 intermediate we live in the Caribbean as a parent I would like to know how I can get my daughter cured

Right now, the only approved cure is a bone marrow transplant. This is usually only done if the patient is transfusion dependent. It should only be a few years before gene therapy will be approved and that will also definitely cure intermedia patients. Does the child take blood transfusions?

bluebird bio Q3 Update

http://investor.bluebirdbio.com/static-files/37fe317c-5c01-43af-9c1d-2decb9d3800d

We should see a new update after next week's presentation at ASH.

First presentation of data from patients with a β0/β0 genotype and a pediatric patient treated with LentiGlobin in Phase 3 Northstar-3 study both have stopped chronic blood transfusions

10 of 11 patients with non-β0/β0 genotypes and more than three months follow-up have stopped chronic transfusions in Phase 3 Northstar-2 study

Indicators of poor red blood cell production appear corrected in exploratory analysis of bone marrow following treatment with LentiGlobin


http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-presents-new-data-lentiglobin-gene-therapy

The results are spectacular. After much negativity from some quarters about the earlier results from the non beta zero trials, we have fantastic news. Yes, only 3 patients have been started so far, but all 3 beta zero patients are transfusion free. The new protocol is working.

Great news .
Dear Andy when can be this treatment available in market ?

bluebird bio has begun the approval process in Europe. It's only a guess, but I would hope that within two years, we will see the first approval for gene therapy for thalassemia.

Thanks Andy

bluebird bio Receives Positive Opinion from CHMP for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia (TDT) Who Do Not Have β0/β0 Genotype

http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-receives-positive-opinion-chmp-zynteglotm

Finally....some exciting news!!


is gene therapy from bluebird going to cost 2.12 mUSD??

2.12 m USD?   

any new updates ? Please share the information about gene therapy in India also.