RPL-301 is under clinical development by Rocket Pharmaceuticals and currently in Phase I for Anemia. According to GlobalData, Phase I drugs for Anemia have an 81% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how RPL-301’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.
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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.
RPL-301 overview
RPL-301 is under development for the treatment of pyruvate kinase deficiency. The therapeutic candidate constitutes patient-derived immature bone marrow cells (called CD34+) genetically modified with a lentivirus to express pyruvate kinase (PKLR) enzyme. It is administered through intravenous route.
Rocket Pharmaceuticals overview
Rocket Pharmaceuticals (Rocket Pharma), formerly Inotek Pharmaceuticals Corp is a clinical-stage biopharmaceutical company. It focuses on gene therapies for rare and devastating diseases. The company has three clinical-stage ex vivo lentiviral vector (“LVV”) programs which include programs for Fanconi Anemia (“FA”), a genetic defect in the bone marrow that reduces the production of blood cells or promotes the production of faulty blood cells, Leukocyte Adhesion Deficiency-I (“LAD-I”), a genetic disorder that causes the immune system to malfunction, and Pyruvate Kinase Deficiency (“PKD”), a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. It also has a clinical-stage in vivo adeno-associated virus (“AAV”) program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. Rocket Pharma is headquartered in Cranbury, New Jersy, the US.
For a complete picture of RPL-301’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.
