Mozafancogene autotemcel is under clinical development by Rocket Pharmaceuticals and currently in Phase II for Fanconi Anemia. According to GlobalData, Phase II drugs for Fanconi Anemia 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 Mozafancogene autotemcel LoA Report. 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.

Mozafancogene autotemcel overview

RPL-102 is under development for the treatment of Fanconi anemia type A. The therapeutic candidate constitutes lentiviral vector carrying FANC-A gene as part of the PGK-FANCA-WPRE expression cassette which includes a phosphoglycerate kinase (PKG) promoter and an optimized woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). It is administered through intravenous route. The therapeutic candidate contains autologous CD34+ enriched cells, these CD34+ cells are mobilized by Granulocyte colony-stimulating factor (G-CSF) and plerixafor.

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 Mozafancogene autotemcel’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 10 June 2024

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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.