Bluebird Bio Inc., a leading gene therapy biotech company based in Cambridge, Massachusetts, announced on February 2 that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to one of its investigational drugs, LentiGlobin® BB305 for the treatment of transfusion-dependent patients with beta-thalassemia major.

Beta-thalassemia is a rare genetic disease affecting 40,000 newborn children annually worldwide.  It is caused by mutations in the beta-globin gene (HBB).  HBB encodes beta chains of hemoglobin and mutation of this gene causes different types of rare blood genetic diseases (Sickle Cell disease or beta thalassemia).  Depending on the severity of symptoms, beta-thalassemia is clinically divided into two types: thalassemia major and thalassemia intermedia with thalassemia major being more severe. Presently, the existing treatment options for these patients have significant side effects and limitations.

LentiGlobin BB305 developed by Bluebird Bio utilizes an improved lentiviral vector to insert a correct copy of human beta-globin gene into the patient’s own hematopoietic stem cells ex vivo and then transplanting those modified cells into the patient through infusion into the bloodstream.  LentiGlobin BB305 is currently undergoing three clinical trials globally aimed at treating both beta-thalassemia and sickle cell disease.

 

Related links:

http://ghr.nlm.nih.gov/condition/beta-thalassemia
http://ghr.nlm.nih.gov/condition/sickle-cell-disease
http://www.bluebirdbio.com/product-overview.php

12 COMMENTS

  1. Please tell me when we will be able to get this treatment
    How much it will cost and also the success rate.please provide me
    Latest updates

  2. Dear Dr.
    I am from Bangladesh, MY boy’s pediatric Dr. guess my boy probably has Thalassemia major.
    His HB is 7 and hb electrophoresis on process.

    Can you please let me know is the gene theraphy working? when it will be available for public ?
    How much it will cost, I am so much worried and feel helpless.

    Please reply with your comment.

    Thanks
    Shahed

    • Hi Shahed,

      This gene therapy treatment LentiGlobin BB305 is currently undergoing three clinical trials globally. I would suggest that you reach out to Bluebird Bio for more information about the clinical trial enrollment and other information. clinicaltrials@bluebirdbio.com

  3. Hi I have Thal Major, I am 34 and live in the UK. When is this study coming to England? I am happy to go on trial, please support me!

  4. Hi

    I am from India and My 4 years old daughter is thal major it was diagnosed when she was 2 years old. she is talking monthly transfusion, in sometimes her HB goes below than 10 but not less than 9.5. otherwise she maintain HB above 10 like 10.2 or 10.5. Can you suggest she is major or intermediate?

    Any new treatment is available now for her.

    Regards
    Siddharth Singh

    • Hi Siddarth,

      Sorry we would not be able to make any suggestions pertaining to your child’s condition. I suggest that you reach out to Bluebird Bio for more information about the clinical trial enrollment and other information. clinicaltrials@bluebirdbio.com

  5. Hi,
    I am from india and My daughter Arya is 10 year old. She is a patient of thallasemia major .
    Kindly suggest if she can undergo gene therapy.
    Regards

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