OTL-201 is under clinical development by Orchard Therapeutics and currently in Phase II for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ). According to GlobalData, Phase II drugs for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) 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 OTL-201 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.

OTL-201 overview

OTL-201 is under development for the treatment of mucopolysaccharidosis-IIIA (Sanfilippo Syndrome Type A). The therapeutic candidate is developed based on ex-vivo lentiviral gene therapy. It is an autologous MPSIIIA HSCs transduced with either LV-PGK-coSGSH or LV-CD11b-coSGSH vectors expressing codon-optimized SGSH. The therapeutic candidate acts by targeting N-sulfoglucosamine sulfohydrolase (SGSH) enzyme.

Orchard Therapeutics overview

Orchard Therapeutics (Orchard) is a biotechnology company that develops gene therapies for the treatment of serious and life-threatening orphan diseases. The company’s pipeline products include OTL-200, OTL-203, OTL-201, OTL-204, OTL-103, OTL-104 and OTL-105. Its pipeline candidates treat metachromatic leukodystrophy (MLD), mucopolysaccharidosis type I (MPS-I), mucopolysaccharidosis type IIIA and Wiskott Aldrich syndrome (WAS). It markets under the brand name of Strimvelis, Libmeldy.

For a complete picture of OTL-201’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 20 May 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.