Neurogene in tie up with university to advance gene therapy technologies

By Jane Byrne

- Last updated on GMT

© GettyImages/Rost-9D
© GettyImages/Rost-9D

Related tags Gene therapy AAV platform Rare disease

New York based, Neurogene, has announced a research collaboration with the University of Edinburgh for the development of a multiple-platform approach to diseases not addressable by conventional gene therapy.

However, the partners were not willing to disclose, as of today, which diseases exactly are being targeted under this alliance.

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

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

Dr Cobb’s lab uses a broad range of technologies to develop novel treatments for neurodevelopmental disorders based on “a deep understanding of the molecular pathology”. 

In addition to Dr Cobb’s position at the university, where he is a Simons fellow and reader in neuroscience, he is also Neurogene’s chief scientific officer (CSO). 

Rare disease pipeline 

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

Neurogene is trying to find treatments for, among others, Batten disease - a group of rare, inherited diseases of the nervous system also called neuronal ceroid lipofuscinoses (NCLs). The company is focusing on CLN5 and CLN7, two rare, late infantile and rapidly progressive subtypes of Batten disease. Children with CLN5 or CLN7 typically develop signs and symptoms of the diseases at a young age, including seizures, progressive deterioration in intellectual and motor capabilities, and loss of vision. CLN5 is caused by a variant in the CLN5 gene, which leads to disruption of normal CLN5 protein function. The CLN7 subtype of Batten disease is caused by a variant in the CLN7 gene, also called the MFSD8 gene, which leads to disruption of normal CLN7 protein function.

Another disorder Neurogene is targeting 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 nerve disorders - neuropathies. 

It is also working to determine and address 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$115m Series B financing, which was led by EcoR1 Capital, with participation from existing investors Redmile Group, Samsara BioCapital, Cormorant Asset Management and an undisclosed leading healthcare investment fund.

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 proceeds from the financing would be used to advance Neurogene’s portfolio of multiple gene therapy programs into the clinic, as well as accelerate investment in novel gene therapy product designs and Neurogene’s technology platform addressing key limitations in conventional gene therapy, while building out its AAV vector GMP manufacturing capabilities.

Oleg Nodelman, portfolio manager, EcoR1 Capital, said then: “Neurogene is establishing itself as a leader in the gene therapy arena for neurological diseases. We are impressed by the company’s innovation and accomplishments to date and are pleased to provide our support to Neurogene to advance medical research in this rapidly evolving area.”

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