Vico Therapeutics

Mahzi Therapeutics

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

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.

Uncommon Therapeutics

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

Scenic Biotech

This company develops disease-modifying small molecule therapeutics for patients with rare genetic and neurodegenerative disorders using its proprietary genetic modifier discovery platform.

LoQus23

This biotechnology company develops small molecule oral therapies targeting DNA mismatch repair to treat patients with Huntington's disease and other triplet repeat disorders.

ARTHEx Biotech

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

Dyne Therapeutics

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

Coave Therapeutics

The company develops targeted gene therapies for patients with neurodegenerative and ocular diseases.