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February 14, 2019

Neurogene raises funds to support gene therapy development

Neurogene has secured funding during a Series A financing round to advance the development of its gene therapy candidates intended for the treatment of various neurological diseases.

Neurogene has secured funding during a Series A financing round to advance the development of its gene therapy candidates intended for the treatment of various neurological diseases.

The financing round was joined by Samsara BioCapital, EcoR1 Capital, Cormorant Asset Management, and Redmile Group, along with an undisclosed healthcare investment fund.

Neurogene focuses on providing therapies for rare neurological conditions. Its lead programmes leverage AAV-based gene therapy technology to deliver a normal gene to peoplehaving a dysfunctional gene.

It collaborates with academic researchers, patient advocacy organisations and caregivers to deliver therapies addressing the underlying genetic cause of neurological diseases that currently lack effective treatments.

The company is also working on new technologies to develop treatments for diseases that could not be adequately treated with gene therapy.

Neurogene intends to use the obtained funds to advance its gene therapy programmes into investigational new drug (IND)-enabling studies and clinical trials for rare neurological disorders.

In addition, the company will invest in new technologies and build a viral vector manufacturing facility.

Neurogene founder and CEO Rachel McMinn said: “Neurogene is reimagining the future for patients with rare neurological diseases and their families.

“Neurogene is reimagining the future for patients with rare neurological diseases and their families.”

“Through partnerships and our own internal expertise, we are advancing our gene therapy programmes 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.”

The company’s pipeline includes programmes for aspartylglucosaminuria, Charcot-Marie tooth disease, and two undisclosed lysosomal storage diseases (LSD).

Earlier this month, Neurogene reported positive pre-clinical data for its gene therapy programmes.

The aspartylglucosaminuria programme is said to have demonstrated dose-dependent, complete or near-complete removal of toxic substrate in central and peripheral tissues and body fluids of mice. It was also found to be well-tolerated at supraphysiological levels.

In case of Charcot-Marie tooth disease, the relevant therapy led to dose-dependent, significant improvement in survival, gross motor function, nerve conduction velocity and histopathology in mice dosed at younger ages prior to any significant cellular damage.

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