Solid Biosciences

Rocket Pharma

This company develops gene therapies for patients with rare and devastating genetic cardiovascular and haematological diseases.

LogicBio

This company develops gene editing and delivery platforms to provide one-time treatments for patients with serious, early-onset rare genetic diseases.

Audentes Therapeutics

The company develops gene therapy products to treat patients with serious and rare neuromuscular diseases.

Capricor

This biotechnology company develops cell and exosome-based therapies to treat Duchenne muscular dystrophy and other serious diseases for patients.

Stridebio

This company develops engineered viral vectors and gene therapies for patients with rare genetic diseases.

Affinia Therapeutics

This company develops rationally designed gene therapies and viral vectors for patients with heart and central nervous system diseases.

Castle Creek Biosciences

This company develops cell and gene therapies for patients suffering from rare genetic skin and connective tissue diseases.

Edgewise

This biopharmaceutical company develops oral small molecule therapies to treat patients with rare skeletal and cardiac muscle diseases.