Sarepta Therapeutics, Inc.

Quick facts

Phase 3 pipeline

• SRP-4045 · Muscular dystrophy
  SRP-4045 is a micro-dystrophin gene therapy that aims to treat Duchenne muscular dystrophy by providing a functional copy of the dystrophin gene.
• SRP-4053 · Neuromuscular/Genetic
  SRP-4053 is an antisense oligonucleotide that modulates dystrophin production by promoting exon 53 skipping in Duchenne muscular dystrophy patients with mutations amenable to this approach.
• SRP-9003 · Neuromuscular/Genetic Disorders
  SRP-9003 is an antisense oligonucleotide that reduces production of DMPK protein by targeting mutant DMPK mRNA in myotonic dystrophy type 1.

Phase 1 pipeline

• AVI-6002
• AVI-6003
• rAAVrh74.MHCK7.DYSF.DV

Competitive intelligence

For the full portfolio CI brief — phase mix, recent regulatory actions, upcoming PDUFA dates, patent timeline, class-overlap with competitors:

• Sarepta Therapeutics, Inc. portfolio CI brief
• Sarepta Therapeutics, Inc. pipeline updates RSS