RARE Daily

Neurogene Raises $68.5 to Advance Gene Therapies for Rare Neurological Diseases

February 12, 2019

New York City-based biotech Neurogene said it completed a $68.5 million series A financing to advance gene therapies to treat rare neurological diseases.

Neurogene’s plans include partnering with leading academic researchers, patient advocacy organizations, and caregivers to bring therapies that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options exist today.

The company will use the funding to advance multiple gene therapy programs through preclinical studies and begin clinical trials for patients with rare neurological diseases, to develop novel technologies for indications not addressed by traditional gene therapy approaches, and to establish a viral vector manufacturing facility.

Neurogene’s preclinical programs include gene therapy treatments for aspartyglucosaminuria, an inherited enzyme deficiency that causes progressive cognitive decline, and Charcot-Marie-Tooth disease, an inherited disorder that affects the peripheral nerves of the limbs, causing muscle weakness and pain.

“Through partnerships and our own internal expertise, we are advancing our gene therapy programs for rare neurological disorders,” said Rachel McMinn, founder and CEO of Neurogene. “The vast majority of rare diseases remain unaddressed, and our goal is to enable a better future for patients with these diseases.”

Investors in the financing include Samsara BioCapital, EcorR1 Capital, Cormorant Asset Management, Redmile Group, and an undisclosed leading healthcare investment fund.

Photo: Rachel McMinn, founder and CEO of Neurogene

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.