PTC Therapeutics

Censa Pharmaceuticals primary focus is the development and commercialization of therapeutics for (ultra) orphan CNS disorders.

ClearPoint Neuro formerly known as MRI Interventions, is a platform neuro company. Its mission is to improve and restore the quality of life to patients and their families by enabling therapies for the most complex neurological disorders with pinpoint accuracy. Applications of ClearPoint Neuro's current product portfolio include deep-brain stimulation, and biopsy, with future applications of laser ablation, neuro-aspiration, and delivery of drugs, biologics and gene therapy to the brain.

BioElectron Technology is a clinical-stage biotechnology company.

Agilis Biotherapeutics develops DNA therapeutics for patients with rare diseases of the central nervous system (CNS) that single-gene defects cause. Its technology enables precise targeting and restoration of lost gene function while avoiding unintended off-target effects. Agilis Biotherapeutics also provides DNA therapeutics for patients with AADC Deficiency, a rare CNS disorder arising from reductions in the enzyme aromatic L-amino acid decarboxylase (AADC) that causes the inability to develop any motor strength and control resulting in breathing, feeding, and swallowing problems. Moreover, it offers DNA therapeutics for Friedreich’s Ataxia, a rare and life-shortening neurodegenerative disease caused by a single defect in the FXN gene, which causes reduced production of the frataxin protein, and DNA therapeutics for Angelman Syndrome (AS), a rare genetic, neurological disorder characterized by severe developmental delays. Agilis Biotherapeutics has a strategic partnership with Intrexon Corporation. It began its operation in 2013, with its headquarters in Lynnfield, Massachusetts.