Marnetegragene autotemcel is under clinical development by Rocket Pharmaceuticals and currently in Pre-Registration for Leukocyte Disorders (White Blood Cell Disorders). According to GlobalData, Pre-Registration drugs for Leukocyte Disorders (White Blood Cell Disorders) have an 82% phase transition success rate (PTSR) indication benchmark for progressing into Marketed. GlobalData’s report assesses how Marnetegragene autotemcel’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.

Marnetegragene autotemcel overview

RPL-201 is under development for the treatment of leukocyte adhesion deficiency (LAD) type 1. The therapeutic candidate constitutes patient-derived hematopoietic stem cells modified with a lentiviral vector containing the CD18 gene. It is administered through intravenous route. It acts by targeting integrin beta 2.

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

This content was updated on 20 May 2024

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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.