RNAi Gene Therapy to Inhibit BCL11A for Sickle Cell Disease and Beta Thalassemia is under clinical development by bluebird bio and currently in Phase II for Sickle Cell Disease. According to GlobalData, Phase II drugs for Sickle Cell Disease have a 59% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how RNAi Gene Therapy to Inhibit BCL11A for Sickle Cell Disease and Beta Thalassemia’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
RNAi Gene Therapy to Inhibit BCL11A for Sickle Cell Disease and Beta Thalassemia overview
RNAi gene therapy is under development for the treatment of sickle cell disease. It is administered by intravenous route. The drug candidate comprises autologous bone marrow-derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA and acts by targeting BCL11A. The drug candidate was under development for the treatment of beta thalassemia beta thalassemia.
Bluebird bio overview
bluebird bio is a biotechnology company that focuses on the development and commercialization of gene therapies for the treatment of severe genetic diseases and cancer. The company develops its products based on integrated product platforms such as lentiviral gene addition platform, cancer immunotherapy and gene editing. Its product pipeline includes elivaldogene autotemcel for the treatment of cerebral adrenoleukodystrophy (CALD); betibeglogene autotemcel for transfusion-dependent beta thalassemia; Lovo-cel for the treatment of sickle cell disease, besides compounds under development for the treatment of various cancers. The company has collaboration with pharmaceutical companies to discover, develop and commercialize gene therapy products. bluebird bio is headquartered in Cambridge, Massachusetts, the US.
For a complete picture of RNAi Gene Therapy to Inhibit BCL11A for Sickle Cell Disease and Beta Thalassemia’s drug-specific PTSR and LoA scores, buy the report here.
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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.
GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.
