Glycomine

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.

Genespire

This company develops off-the-shelf gene therapies for children and adults affected by inherited metabolic diseases and primary immunodeficiencies.

Uncommon Therapeutics

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

Dogma Biotech

This company develops AI-designed glycans to enhance protein therapeutic performance for patients with central nervous system and lysosomal storage diseases.

Rampart Biosciences

This company develops non-viral, DNA-based gene medicines for patients with serious genetic disorders, specifically focusing on liver-related conditions like hypophosphatasia.

GondolaBio

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

Phlox Therapeutics

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

Allievex

This clinical-stage biotechnology company develops enzyme replacement therapies for children with rare pediatric neurodegenerative diseases, specifically Sanfilippo syndrome type B.