bluebird bio Inc (NASDAQ:BLUE) a clinical-stage medical company and biotech stocks has published a case study on the first severe sickle cell disease patient treated with gene therapy. The case study was published in the New England Journal of Medicine. bluebird bio Inc is one of the top biotech stocks company specializing in the development of transformative gene therapies for advanced stage genetic diseases and T cell-based immunotherapies patients with cancer.
The patient was aged 13 at the time of treatment and was treated using LentiGlobin drug product. The clinical study was done in Necker Hospital based in Paris France. The information published shows a follow-up period of 15 months. It also has a summary of a 21-months follow-up on the results. The results were presented at the 58th American Society of Hematology Annual Meeting that was held in 2016.
The study’s principal investigator Professor Marina Cavazzana, said they have managed the patient for over 10 years. He says during the period, standard treatments failed to control his SCD symptoms. The patient depended on monthly blood transfusions to prevent severe pain crises. He says since being administered with the LentiGlobin, the patient has not experienced severe symptoms and has resumed normal life.
Dr. Philippe Leboulch from University Paris-Sud said they first published the therapeutic approach in the mouse model in 2011 and the current publication is a proof of the therapy’s efficiency in patients. Dr. Leboulch is the founder of bluebird bio and serves as the co-chairman of the company’s Scientific Advisory Board.
bluebird bio M.D., chief medical officer David Davidson said it is encouraging to have the case study published. He adds the positive result is a clear demonstration of the promise of a LentiGlobin gene therapy tratement for patients with advanced stage SCD. He adds that the study will serve as the company’s guide in efforts to optimize the results for future use. Davidson says the study has provided key highlights that the company intends to optimize in the ongoing HGB-206 study of LentiGlobin gene therapy in advanced stage SCD.