Pluvia

Anvesana

Anvesana develops therapeutics for patients with rare genetic disorders by leveraging an RNA-sequence analysis platform to identify targets for up-regulating gene expression.

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.

River Renal

This clinical-stage biotechnology company develops novel pharmaceutical therapies targeting orphan renal diseases for patients with unmet medical needs.

Protego Biopharma

This biotechnology company develops small molecule therapies for patients with systemic amyloid diseases and other protein misfolding disorders.

Aeovian Pharmaceuticals

This biopharmaceutical company develops selective small-molecule inhibitors for patients with rare genetic and age-related diseases, such as tuberous sclerosis complex refractory epilepsy.

GondolaBio

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

Gain Therapeutics

This biotechnology company develops allosteric small molecule therapies for patients with neurodegenerative diseases and rare genetic disorders using an artificial intelligence drug discovery platform.

PHVS

This company develops oral therapies to treat and prevent hereditary angioedema attacks for patients with rare diseases.