We are partnering with the world’s leading academic specialists and patient advocates to accelerate new therapies to patients in need. Our most advanced programs have a strong body of preclinical proof-of-concept data generated by our academic collaborators, a team of passionate and committed experts in adeno-associated virus (AAV) vector design and CNS delivery, neurology, neuroimmunology, and neurodevelopment. Neurogene is also investing in novel therapeutic approaches with the potential to address the underlying cause of genetic diseases that are not well-served by gene therapy. Our vision is to build a sustainable genetic medicines company that brings life-altering therapies to the many rare, unaddressed genetically-defined conditions, in order to begin tackling the vast unmet need for patients and their families.

Neurogene is on a mission to REVERSE THE STATISTICS

Rare diseases are rare in number, but not rare to families caring for a sick loved one, with responsibilities that can be exhausting and life-altering. While important progress has been made against rare diseases in recent decades, the vast majority remain unaddressed, with neurological diseases continuing to be the most underserved. Recent innovations in gene therapy, however, provide tools to help tackle even the most intractable of diseases.

Neurogene’s mission is to improve patient outcomes by advancing multiple genetic medicines for as many neurological disorders for which we may make a tangible, life-altering difference. We envision a world in which all families have access to genetic medicines, even if the disorder is exceedingly rare.

Neurogene | Statistics of known rare genetic neurological diseases.

“Through our natural history studies and interactions with patients and their families, we are learning more about diseases like CLN5, CLN7 and CMT4J. This knowledge, in turn, will help us to design future gene therapy studies.”

– Effie Albanis, M.D., Chief Medical Officer


As industry enthusiasm for gene therapy grows across the medical landscape, a key challenge of developing gene therapy medicines is the increasing manufacturing capacity constraints. These constraints can lead to high costs and long wait times for products from contract manufacturers. At Neurogene, we are building out our own capabilities in order to develop internal expertise in these highly specialized programs, and to better control costs and timelines.