Stoke Therapeutics

Encoded Therapeutics

This biotechnology company develops precision genetic medicines for patients with severe neurological disorders, including Dravet syndrome.

Vico Therapeutics

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

Mahzi Therapeutics

This biotechnology company develops gene and antisense oligonucleotide therapies for patients with rare genetic neurodevelopmental disorders.

Dyne Therapeutics

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

Neurogene Inc

This company develops genetic medicines and gene therapies to treat patients and families affected by rare neurological diseases including Rett syndrome and Batten disease.

Uncommon Therapeutics

This company develops gene-based medicines for patients with rare neurodevelopmental disorders, with an initial focus on treating Rett Syndrome.

ARTHEx Biotech

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

Soufflé Therapeutics

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