Neurogene Raises $115 Million to Advance Gene Therapies for Rare Neurological Diseases

Rare Daily Staff

Neurogene completed a $115 million series B financing to advance its pipeline of experimental gene therapies to treat rare neurological diseases, ten months after raising $68.5 million in a series A round.

EcoR1 Capital led financing round, with participation from existing investors Redmile Group, Samsara BioCapital, Cormorant Asset Management and an undisclosed leading healthcare investment fund. New investors include funds and accounts managed by BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, and Alexandria Venture Investments.

Venture investment in gene therapies and platforms is on soaring, as Neurogene’s financing brings the total invested 2020 in the modality to $2.475 billion into 24 companies, with ten of them series A rounds.

Proceeds from the financing will be used to advance Neurogene’s portfolio of multiple gene therapy programs into the clinic, accelerate investment in novel gene therapy product designs and Neurogene’s technology platform addressing key limitations in conventional gene therapy, and build out Neurogene’s state-of-the-art adeno-associated virus (AAV) vector GMP manufacturing capabilities.

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. The company anticipates filing for its first IND in 2021.

“Gene therapy has generated tremendous hope for the many families and patients with severe genetic disorders. We believe our focus on improved product design, innovative technology, cutting-edge vector manufacturing and premier analytics will help fulfill the potential of genetic treatments,” said Rachel McMinn, founder and CEO of Neurogene. “Proceeds from this round of financing will help us execute on our mission to develop safe and effective genetic therapies for patients and families living with neurological diseases.”

Photo: Rachel McMinn, founder and CEO of Neurogene