EveryONE Medicines

Mahzi Therapeutics

This biotechnology company develops gene and antisense oligonucleotide therapies for patients with rare genetic neurodevelopmental disorders.

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.

Allievex

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

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.

Stoke Therapeutics

This biotechnology company develops antisense oligonucleotide medicines to treat severe genetic diseases by restoring protein expression for patients with conditions such as Dravet syndrome.

Vico Therapeutics

This company develops antisense oligonucleotide therapies to treat patients with rare genetic neurological disorders, including Huntington's disease and spinocerebellar ataxias.

Dyne Therapeutics

The company develops oligonucleotide therapeutics to treat patients with genetically driven neuromuscular diseases, including Duchenne muscular dystrophy and myotonic dystrophy type 1.