Mahzi Therapeutics

Uncommon Therapeutics

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

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.

Minoryx

This biotechnology company develops orphan drugs and therapies for patients suffering from rare, life-threatening genetic diseases of the central nervous system.

Vico Therapeutics

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

Noema Pharma

This company develops clinical-stage therapeutic products for patients with central nervous system disorders and orphan neurological conditions.

Pano Therapeautics

This biotechnology company develops small molecule drugs targeting mitochondrial ion channels to treat neurodevelopmental disorders and complex diseases of aging.

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.

Minoryx Therapeutic

This biotech company develops therapies for orphan central nervous system diseases, such as X-linked adrenoleukodystrophy, for patients with high unmet medical needs.