Design Therapeutics

Dyne Therapeutics

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

ARTHEx Biotech

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

Yumanity Therapeutics

This company develops small molecule drugs to treat neurodegenerative diseases for patients with conditions such as Parkinson's disease and amyotrophic lateral sclerosis.

Ribonaut Therapeutics

Ribonaut Therapeutics develops small molecule drugs that target and degrade disease-causing RNA to treat neurological conditions and movement disorders for human patients.

Shape Therapeutics

The company develops programmable RNA medicines and delivery systems for patients with genetic disorders and other hard-to-treat diseases.

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.

Typewriter Therapeutics

The company develops RNA-based genetic medicines for patients with cancer, autoimmune conditions and rare genetic diseases.

Gain Therapeutics

This biotechnology company develops allosteric small molecule therapies for patients with neurodegenerative diseases and rare genetic disorders using an artificial intelligence drug discovery platform.