Eton Pharmaceuticals

Ultragenyx

This biopharmaceutical company develops and commercialises therapies for patients with serious rare and ultra-rare genetic diseases.

Krystal Biotech

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

Harmony Biosciences

This pharmaceutical company develops and commercialises therapies for patients living with rare neurological diseases, such as narcolepsy and other sleep-wake disorders.

Rocket Pharma

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

VectivBio

This company develops biopharmaceutical treatments for severe rare gastrointestinal conditions, including short bowel syndrome, for patients with orphan diseases.

Abliva

This biopharmaceutical company develops novel medicines for patients suffering from rare and severe primary mitochondrial diseases.

Pharos IBT

This company develops artificial intelligence platforms for discovering and developing treatments for rare and refractory diseases for the pharmaceutical and biotechnology industries.

HRMY

This pharmaceutical company develops and commercialises therapies for patients with rare neurological disorders and central nervous system diseases.