Dyne Therapeutics

Dyne

This company develops therapeutics for patients with genetically driven neuromuscular diseases.

ARTHEx Biotech

This clinical-stage biotechnology company develops RNA-based treatments for patients with rare neuromuscular genetic diseases, including myotonic dystrophy type 1.

Design Therapeutics

Design Therapeutics develops small-molecule therapies for patients with serious degenerative genetic diseases, including Friedreich ataxia and myotonic dystrophy type-1.

Creyon Bio

This company develops AI-designed oligonucleotide-based medicines and RNA-targeted therapies for pharmaceutical partners and patients with neuromuscular and immunological diseases.

Edgewise Therapeutics

This biopharmaceutical company develops small molecule therapies for patients with serious cardiovascular diseases and musculoskeletal conditions like Duchenne and Becker muscular dystrophy.

Stoke Therapeutics

This biotechnology company develops antisense oligonucleotide medicines to treat severe genetic diseases by restoring protein expression for patients with conditions such as Dravet syndrome.

Soufflé Therapeutics

This biotechnology company develops cell-specific oligonucleotide therapies for patients with muscular dystrophy, heart failure, and metabolic disorders.

Vico Therapeutics

This company develops antisense oligonucleotide therapies to treat patients with rare genetic neurological disorders, including Huntington's disease and spinocerebellar ataxias.