PGN-EDODM1
Myotonic Dystrophy Type 1 (DM1)
About PepGen
PepGen is a clinical-stage biotech focused on developing transformative oligonucleotide therapies for severe genetic neuromuscular and neurological diseases. Its core strategy leverages a proprietary Enhanced Delivery Oligonucleotide (EDO) platform designed to solve the critical challenge of delivering therapeutic cargo to deep tissues like muscle and nerve cells. The company's lead candidate, PGN-EDODM1 for myotonic dystrophy type 1 (DM1), has generated compelling early clinical data, including unprecedented splicing correction, and is advancing in Phase 2 studies. With a modular platform and a pipeline extending into Duchenne muscular dystrophy (DMD) and Charcot-Marie-Tooth disease (CMT1A), PepGen aims to establish a new class of disease-modifying genetic medicines.
View full company profileAbout PepGen
PepGen is a clinical-stage biotech focused on developing transformative oligonucleotide therapies for severe genetic neuromuscular and neurological diseases. Its core strategy leverages a proprietary Enhanced Delivery Oligonucleotide (EDO) platform designed to solve the critical challenge of delivering therapeutic cargo to deep tissues like muscle and nerve cells. The company's lead candidate, PGN-EDODM1 for myotonic dystrophy type 1 (DM1), has generated compelling early clinical data, including unprecedented splicing correction, and is advancing in Phase 2 studies. With a modular platform and a pipeline extending into Duchenne muscular dystrophy (DMD) and Charcot-Marie-Tooth disease (CMT1A), PepGen aims to establish a new class of disease-modifying genetic medicines.
View full company profileTherapeutic Areas
Other Myotonic Dystrophy Type 1 (DM1) Drugs
| Drug | Company | Phase |
|---|---|---|
| ATX-01 (ArthemiR trial) | ARTHEx Biotech | Phase 1/2 |
| AOC 1001 | Avidity Biosciences | Phase 1/2 |
| DYNE-101 | Dyne Therapeutics | Phase 1/2 |
| SRP-6004 | Sarepta Therapeutics | Phase 1/2 |
| Undisclosed | Design Therapeutics | Preclinical |