Azafaros

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.

Minoryx Therapeutics

This company develops leriglitazone, a pharmacological treatment for patients with rare genetic central nervous system diseases such as X-linked adrenoleukodystrophy.

Amicus Therapeutics

This biotechnology company develops and delivers medicines to treat individuals living with rare metabolic conditions, specifically lysosomal storage disorders such as Fabry and Pompe disease.

Northsea Therapeutics

This company develops structurally engineered fatty acid drugs for patients with metabolic, inflammatory, and fibrotic diseases such as rare liver disorders.

Avalyn Pharma

The company develops inhaled medicines to treat patients with rare respiratory diseases, including pulmonary fibrosis and other interstitial lung diseases.

Atavistik Bio

This biotechnology company develops oral allosteric small molecule therapeutics for patients with rare hematological diseases and cancers.

Alesta Therapeutics

This biotechnology company develops oral small molecule therapies for patients with rare genetic disorders including hypophosphatasia and Charcot-Marie-Tooth disease.

Mereo BioPharma

The company develops clinical-stage therapies for patients with rare diseases, including osteogenesis imperfecta and alpha-1 antitrypsin deficiency-associated lung disease.