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However, the partners were unwilling to disclose, to date, exactly which diseases are targeted under this alliance.

The collaborative project combines Neurogene’s manufacturing and drug development capabilities with the University of Edinburgh’s new platform and neurodevelopmental disease expertise.

Under the terms of the collaboration, the US company will provide financial support to Dr Stuart Cobb’s laboratory at the University of Edinburgh, in exchange for the right to license any applicable intellectual property on agreed economic terms. Neurogene will be responsible for the advanced stage of preclinical and clinical development of all products generated as part of the collaboration.

Dr. Cobb’s lab uses a wide range of technologies to develop new treatments for neurodevelopmental disorders based on “an in-depth understanding of molecular pathology ”.

In addition to Dr Cobb’s position at the university, where he is a Simons Fellow and Neuroscience Reader, he is also Scientific Director (CSO) of Neurogene.

Rare disease pipeline

Neurogene’s main programs use adeno-associated virus (AAV) gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Its product portfolio of gene therapy candidates addresses distinct monogenic neurological diseases.

Neurogene is trying to find treatments for, among other things, Batten disease – a group of rare inherited diseases of the nervous system also known as neuronal ceroid lipofuscinosis (NCL). The company is focused on CLN5 and CLN7, two rare, late infantile and rapidly progressive subtypes of Batten disease. Children with CLN5 or CLN7 usually develop signs and symptoms of the disease at a young age, including seizures, progressive deterioration of intellectual and motor skills, and loss of vision. CLN5 is caused by a variant of the CLN5 gene, which causes disruption of the normal function of the CLN5 protein. The CLN7 subtype of Batten disease is caused by a variant of the CLN7 gene, also known as the MFSD8 gene, which causes disruption of the normal function of the CLN7 protein.

Another disease targeted by Neurogene is Charcot-Marie-Tooth disease (CMT) – a group of inherited diseases that affect the peripheral nervous system (PNS). CMTs are the most common inherited motor and sensory disorders – neuropathies.

He is also working to determine and treat the root cause of diseases such as aspartylglucosaminuria (AGU), a rare neurodegenerative lysosomal storage disorder (LSD).


In December 2020, Neurogene announced the completion of a US $ 115 million Series B financing, led by EcoR1 Capital, with the participation of existing investors Redmile Group, Samsara BioCapital, Cormorant Asset Management and an investment fund of leading in the field of health.

New investors included funds and accounts managed by BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners and Alexandria Venture Investments.

The company said the proceeds of the funding would be used to advance Neurogene’s portfolio of multiple gene therapy programs in the clinic, as well as to accelerate investment in new gene therapy product designs and the technology platform. of Neurogene addressing key limitations of conventional gene therapy, while enhancing its AAV vector GMP manufacturing capabilities.

Oleg Nodelman, Portfolio Manager, EcoR1 Capital, then said: “Neurogene has established itself as a leader in the field of gene therapy for neurological diseases. We are impressed with the innovation and achievements of the company to date and are delighted to support Neurogene in advancing medical research in this rapidly evolving field.

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