GeneTAC Platform for DM1
Myotonic Dystrophy Type 1 (DM1)
PreclinicalActive
Key Facts
About Design Therapeutics
Design Therapeutics is developing a new class of disease-modifying therapies, GeneTAC™ (Gene Targeted Chimeras), designed to precisely target the underlying genetic cause of degenerative diseases. The company's initial focus is on serious conditions like Friedreich ataxia and myotonic dystrophy type 1, where its lead candidate, DT-216, has entered clinical development. With a strong intellectual property position and a platform with potential application across multiple nucleotide repeat expansion disorders, Design aims to create transformative treatments for patients with limited or no therapeutic options.
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 |
| ENTR-701 | Entrada Therapeutics | Preclinical |
| PGN-EDODM1 | PepGen | Phase 2 |