Patidistrogene bexoparvovec is under clinical development by Sarepta Therapeutics and currently in the Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.
According to GlobalData, the latest event to affect Patidistrogene bexoparvovec’s likelihood of approval (LoA) and phase transition for Limb-Girdle Muscular Dystrophy took place on 23 Aug 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway.
GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Patidistrogene bexoparvovec Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.
Patidistrogene bexoparvovec overview
patidistrogene bexoparvovec (SRP-9004) is under development for the treatment of limb-girdle muscular dystrophy type 2D (LGMD2D). It is administered through injection. The therapeutic candidate comprises self-complementary recombinant adeno-associated virus serotype rhesus 74 (AAVrh.74) encoding alpha-sarcoglycan.
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.
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