Allievex

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.

EveryONE Medicines

The company develops individualized antisense oligonucleotide therapies for children with rare life-threatening neurodegenerative diseases caused by unique genetic mutations.

Allena

This biopharmaceutical company develops oral enzyme therapeutics for patients with rare and severe metabolic and kidney disorders, such as hyperoxaluria.

Quince Therapeutics (formerly EryDel)

This biotechnology company develops red blood cell-based drug delivery systems for patients with rare neurodegenerative diseases such as ataxia-telangiectasia.

Genespire

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

Allyx

This clinical-stage biotechnology company develops oral small molecule therapies to protect synapses in patients with Alzheimer's disease and other neurodegenerative conditions.

Minoryx

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

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.