Amicus Therapeutics

Protalix

This company develops and produces plant-based recombinant therapeutic proteins to treat patients with rare diseases, including Fabry and Gaucher disease.

Akero Therapeutics

This company develops metabolic disease treatments for patients with metabolic dysfunction-associated steatohepatitis and other liver conditions.

Comet Therapeutics

This biotechnology company develops coenzyme A metabolism therapies to treat patients with inherited metabolic diseases and orphan neurological disorders.

Allievex

This clinical-stage biotechnology company develops enzyme replacement therapies for children with rare pediatric neurodegenerative diseases, specifically Sanfilippo syndrome type B.

Carmot Therapeutics

This biotechnology company develops drug therapies to treat metabolic diseases, such as obesity and diabetes, for patients with these conditions.

Actio Biosciences

This biotechnology company develops small molecule medicines to treat rare genetic diseases and common conditions for patients with high unmet medical needs.

Aera Therapeutics

This biotechnology company develops delivery platforms for genetic medicines to treat various diseases for biopharmaceutical partners and patients.

Ultragenyx

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