Actio Biosciences

Atavistik Bio

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

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.

Axovia Therapeutics

The company develops gene therapies for patients with ciliopathies, specifically targeting rare genetic conditions like Bardet-Biedl Syndrome to treat blindness and obesity.

ArQule

This biopharmaceutical company develops targeted small molecule drug candidates to treat cancers and rare diseases for patients with unmet medical needs.

GondolaBio

This biopharmaceutical company develops clinical-stage and preclinical medicinal therapeutics for patients living with rare genetic diseases.

Mighty Therapeutics

This company develops mitochondrial-targeted therapies for patients with rare genetic conditions like Barth syndrome and common age-related diseases.

Pano Therapeautics

This biotechnology company develops small molecule drugs targeting mitochondrial ion channels to treat neurodevelopmental disorders and complex diseases of aging.

Uncommon Therapeutics

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