PGN-EDODM1
Myotonic Dystrophy Type 1 (DM1)
About PepGen
PepGen is advancing a pipeline of disease-modifying oligonucleotide therapeutics targeting the root cause of severe neuromuscular and neurological diseases. Leveraging its proprietary Enhanced Delivery Oligonucleotide (EDO) platform, the company aims to solve the critical delivery and uptake limitations of previous therapies, particularly in hard-to-reach tissues like heart and skeletal muscle. With a lead program in Phase 2 for DM1 and a robust discovery pipeline, PepGen is positioned to potentially transform treatment outcomes for patients with devastating genetic disorders. The company is publicly traded and led by an experienced team with deep expertise in rare disease drug development.
View full company profileAbout PepGen
PepGen is advancing a pipeline of disease-modifying oligonucleotide therapeutics targeting the root cause of severe neuromuscular and neurological diseases. Leveraging its proprietary Enhanced Delivery Oligonucleotide (EDO) platform, the company aims to solve the critical delivery and uptake limitations of previous therapies, particularly in hard-to-reach tissues like heart and skeletal muscle. With a lead program in Phase 2 for DM1 and a robust discovery pipeline, PepGen is positioned to potentially transform treatment outcomes for patients with devastating genetic disorders. The company is publicly traded and led by an experienced team with deep expertise in rare disease drug development.
View full company profileTherapeutic Areas
Other Myotonic Dystrophy Type 1 (DM1) Drugs
| Drug | Company | Phase |
|---|---|---|
| AOC Program | Avidity Biosciences | Clinical |
| DYNE-101 | Dyne Therapeutics | Phase 1/2 |
| GeneTAC Platform for DM1 | Design Therapeutics | Preclinical |
| ENTR-701 | Entrada Therapeutics | Preclinical |