Lexeo Therapeutics

Phlox Therapeutics

This company develops RNA-based gene therapies to treat patients with rare genetic heart diseases, including cardiomyopathies and laminopathies.

Rocket Pharma

This company develops gene therapies for patients with rare and devastating genetic cardiovascular and haematological diseases.

Affinia Therapeutics

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

Neurogene Inc

This company develops genetic medicines and gene therapies to treat patients and families affected by rare neurological diseases including Rett syndrome and Batten disease.

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.

NeuroTherapia

This clinical-stage company develops oral small-molecule therapies targeting neuroinflammation for patients with Alzheimer's disease and other neurodegenerative disorders.

Atsena Therapeutics

This clinical-stage company develops gene therapies to treat and prevent inherited retinal diseases for patients with conditions such as X-linked retinoschisis and Stargardt disease.

ARTHEx Biotech

This clinical-stage biotechnology company develops RNA-based treatments for patients with rare neuromuscular genetic diseases, including myotonic dystrophy type 1.