Neurogene Launches With $68.5 Million to Advance Gene Therapies for Rare Neurological Diseases

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New York-based Neurogene raised $68.5 million in a Series A funding round to support its pipeline of therapeutics aimed at rare neurological diseases.

Neurogene said the funding will be used to advance multiple gene therapy programs into the clinic aimed at these diseases. Among those assets the company will focus its resources on for development is likely to include an adeno-associated virus (AAV) gene therapy for aspartylglucosaminuria (AGU) and CMT4J, two rare neurological diseases. Late last week, company researchers presented preliminary, unpublished “proof of concept” data in mice demonstrating the safety and efficacy of AAV treatments at the 15th Annual WORLDSymposium conference in Orlando. The therapies are being investigated under a collaboration agreement between Neurogene and U.T. Southwestern Medical Center.

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The researchers noted that the unpublished preclinical data has not been peer reviewed. The company said that the AAV treatment for AGA, a disease caused by dysfunction of the AGA gene, resulted in “dose-dependent, complete or near-complete elimination of toxic substrate in central and peripheral tissues and body fluids.” The treatment was well-tolerated by the mice, the company said.

Likewise, the non-peer-reviewed data also showed promise in the treatment of Charcot-Marie-Tooth disease, type 4J (CMT4J) syndrome, a debilitating peripheral neuropathy caused by dysfunction of the FIG4 gene. The company’s AAV gene therapy “resulted in a dose-dependent, significant improvement in survival, gross motor function, nerve conduction velocity, and histopathology,” Neurogene said.

Neurogene's business model is to partner with academic researchers, patient advocacy organizations and caregivers to bring therapies that address the underlying genetic cause of a broad spectrum of neurological diseases where few or no effective treatment options currently exist. Rachel McMinn, founder and chief executive officer of Neurogene, said the company is “reimagining the future” when it comes to rare neurological diseases.

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

Regarding the preclinical data the company presented at the symposium, McMinn said results from the AGU and CMT4J gene therapy programs have provided “encouraging proof of principle data to support future first-in-human clinical trials.”

“We are working closely with our collaborators to advance these programs as expeditiously as feasible to help families suffering from these diseases and further our commitment to advance a pipeline of genetic medicines to treat the underlying cause of serious neurological disorders,” McMinn said.

The $65.8 million in funding was supported by Samsara BioCapital, EcoR1 Capital, Cormorant Asset Management, Redmile Group and an undisclosed leading healthcare investment fund. The company intends to advance its programs into the clinic, as well as invest in novel technologies for indications not addressed by traditional gene therapy approaches. Neurogene also said it intends to use a portion of the funding from the Series A to establish a viral vector manufacturing facility. It did not provide details on that particular plan.

In January, the company tapped neuroscience veteran Stuart Cobb as its chief scientific officer. Cobb’s duties will include leading Neurogene’s scientific research, as well as identifying novel technologies that complement the company’s pipeline. The company noted that Cobb will divide his time equally between Neurogene and the University of Edinburgh, where he is a Simons Fellow and Reader in Neuroscience.

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