Ultragenyx

Krystal Biotech

This biotechnology company develops and commercialises redosable gene therapies for patients with rare genetic diseases, including dystrophic epidermolysis bullosa.

Rocket Pharma

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

Eton Pharmaceuticals

This company develops and commercialises prescription medicines to treat patients living with rare diseases.

LogicBio

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

Uncommon Therapeutics

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

Calliditas therapeutics

This biopharmaceutical company develops and commercialises medicines for patients with rare renal and hepatic diseases.

SpringWorks Therapeutics

This biopharmaceutical company develops and commercialises targeted oncology medicines and therapies for patients with rare diseases and cancer.

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.