Advancing Revolutionary Therapies to Treat Degenerative Neuromuscular Diseases

Using our novel Enhanced Delivery Oligonucleotide (EDO) platform, we are developing a broad pipeline of disease-modifying peptide-conjugated oligonucleotide candidates to treat a variety of degenerative neuromuscular diseases.

People with serious neuromuscular diseases including Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) have limited therapeutic options. In spite of approved therapies for DMD, the promises of previous therapeutic breakthroughs have not materialized or have resulted in treatment options that force an unacceptable tradeoff between toxicity and efficacy.

PROGRAM TARGET DISCOVERY PRECLINICAL FIH/FIP ESTIMATED PATIENTS
DMD
Exon 51
Q1 2022 US: >10,000 width DMD,
PepGen will address 35%+
Other
Exons
DM1
DMPK
Q4 2022 US: ~40,000
NMD INDICATION
CNS INDICATION