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Commercial collaboration offers hope of new treatments for Rett syndrome

At the forefront of research to develop gene therapy treatment for Rett syndrome is the University of Edinburgh’s Dr Stuart Cobb, his team and their partnership with Neurogene, a US-based developer of genetic medicines for neurological disorders.

Neurogene began working with Dr Stuart Cobb shortly after the company was incorporated in early 2018. Interested in collaborating with the University of Edinburgh to incorporate innovation into the organisation, Neurogene CEO and Founder Rachel McMinn reached out to Dr Cobb, Reader in Neuroscience at the University’s Centre for Discovery Brain Sciences.

Following a visit to Edinburgh, their partnership to find a gene therapy for Rett syndrome – a genetic neurological disorder – began.

Creating new gene therapy technologies

Knowing that more conventional therapies were unlikely to meaningfully improve the lives of people with Rett syndrome, Dr Cobb, his research team and Neurogene focused on finding innovative gene therapies for the disorder. The team is working to do this by creating new technologies that will provide a more precise, safe and effective gene therapy.

A shared purpose and mission

There is strong alignment between Neurogene and the University. They both recognise the importance of collaboration, innovation and the impact of their work. They both share a sense of urgency when it comes to finding solutions for treating a complex disorder like Rett syndrome. And they both appreciate the impact an effective treatment could have on the lives of patients and their families – it is this that motivates the team to constantly innovate and test ideas.

Edinburgh Innovations’ ongoing support

Neurogene is collaborating with the University and Edinburgh Innovations (EI) on more than one research project. Despite disruption at the beginning of the Covid-19 pandemic, the collaboration has successfully continued, and Neurogene and EI have worked effectively together to uphold a robust and productive scientific exchange.

Dr Cobb now serves as Chief Scientific Officer for Neurogene, and in early 2021 he signed a multi-year agreement with Neurogene to advance development of multiple platform approaches to improve gene therapy technologies.

From a business standpoint, EI has been instrumental in the progress and success so far. EI’s legal and business support allows the researchers and Neurogene to focus on what they do best – developing life-changing treatments for people living with Rett syndrome.

Success founded in innovation

So far, Dr Cobb’s team have successfully developed a technology which allows the disruptive gene that causes Rett syndrome to be replaced with a healthy copy. This technology delivers gene therapy to the cells that need replacing with more precision than any standard gene therapy. Such early progress means the team will probably be able to develop treatments with the potential to transform patients’ lives, and they hope to be able to take these treatments to clinical trials.

Neurogene’s investment in gene therapy innovation and technology has opened up an incredible world of possibility for Dr Cobb and his team, and has the capacity to go on to impact the field as a whole.

We really value our collaboration with Edinburgh Innovations and the University, and believe that we are all working with a fundamental belief that investing in innovation is critical to successfully developing meaningful treatments for complex diseases like Rett syndrome.

Dr Rachel McMinn, CEO and Founder, Neurogene

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