GALGT2 (Nationwide Children’s) is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. According to GlobalData, Phase II drugs for Duchenne Muscular Dystrophy have a 65% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how GALGT2 (Nationwide Children’s)’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.
GALGT2 (Nationwide Children’s) overview
Gene therapy is under development for the treatment of Duchenne muscular dystrophy. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). It is administered by intravascular and intramuscular routes. The therapeutic candidate acts by targeting Beta-1, 4 N-acetylgalactosaminyltransferase 2 (GALGT2). It is developed based on exon skipping technology.
Sarepta Therapeutics overview
Sarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. The company’s platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Its commercial products include Exondys 51, Vyondys 53 and Amondys 45 indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene. Its pipeline product includes SRP-5051, SRP-9001, SRP-9003 and SRP-5045 indicated for the treatment of DMD, limb-girdle muscular dystrophies (LGDMs) and other neuromuscular and central nervous system disorders. Sarepta is headquartered in Cambridge, Massachusetts, the US.
For a complete picture of GALGT2 (Nationwide Children’s)’s drug-specific PTSR and LoA scores, buy the report here.