AavantiBio

4D Molecular Therapeutics

This digital health company develops precision gene therapies for patients with retinal, pulmonary, and cardiac diseases using a proprietary vector evolution platform.

Affinia Therapeutics

This company develops rationally designed gene therapies and viral vectors for patients with heart and central nervous system diseases.

Stridebio

This company develops engineered viral vectors and gene therapies for patients with rare genetic diseases.

Lexeo Therapeutics

This clinical-stage company develops gene therapies for patients with genetically defined cardiovascular diseases and Alzheimer's disease.

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.

Design Therapeutics

Design Therapeutics develops small-molecule therapies for patients with serious degenerative genetic diseases, including Friedreich ataxia and myotonic dystrophy type-1.

Asklepios BioPharmaceutical

This clinical-stage company develops and manufactures adeno-associated virus gene therapies for patients with rare genetic, neuromuscular, central nervous system and cardiovascular disorders.

Sanius Health

This company provides a health data platform and wearable devices for patients with rare diseases and researchers to monitor symptoms and accelerate drug discovery.