RPA-501 is under clinical development by Rocket Pharmaceuticals and currently in Phase II for Glycogen Storage Disorders (GSD). According to GlobalData, Phase II drugs for Glycogen Storage Disorders (GSD) does not have sufficient historical data to build an indication benchmark PTSR for Phase II. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the RPA-501 LoA Report. Buy the report here.
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RPA-501 overview
RPA-501 is under development for the treatment of Danon disease (a glycogen storage disease Type IIb). It is developed based on AAV technology. It comprises of recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene. The drug candidate 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 RPA-501’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.
