GT-0001X is under clinical development by Gene Therapy Research Institution and currently in Phase II for Amyotrophic Lateral Sclerosis. According to GlobalData, Phase II drugs for Amyotrophic Lateral Sclerosis have a 65% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how GT-0001X’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.
GT-0001X is under development for the treatment of sporadic amyotrophic lateral sclerosis. The drug candidate is developed using modified Adeno-associated virus (AAV) vector to deliver ADAR2. The therapeutic candidate acts by targeting adenosine deaminase acting on RNA 2 (ADAR2). It is administered through intrathecal route.
For a complete picture of GT-0001X’s drug-specific PTSR and LoA scores, buy the report here.