Arthex

ARTHEx Biotech

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

DTX Pharma

This biotechnology firm develops RNA-based therapeutics for patients with rare neuromuscular and central nervous system disorders using a proprietary fatty acid delivery platform.

Exonics

This company develops gene editing therapies for patients with Duchenne muscular dystrophy and other severe genetic neuromuscular diseases.

Dyne Therapeutics

The company develops oligonucleotide therapeutics to treat patients with genetically driven neuromuscular diseases, including Duchenne muscular dystrophy and myotonic dystrophy type 1.

Armatus

The company develops vectorized RNAi therapeutics to treat genetic neurological and neuromuscular diseases for patients with rare conditions.

Vico Therapeutics

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

Exonics Therapeutics

The company develops gene editing therapies for patients with Duchenne muscular dystrophy and other genetic neuromuscular diseases.

Skyhawk Therapeutics

This company develops small molecules that modulate RNA for patients with rare neurological, oncological, and autoimmune diseases.