Neurogene Announces New Development Program in Rett Syndrome Utilizing Novel EXACT Technology Platform
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Wednesday, May 18, 2022
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Rett syndrome is a complex disorder not readily addressable by conventional gene therapy because the MECP2 gene is toxic if expressed at high levels.
Key Points:
- Rett syndrome is a complex disorder not readily addressable by conventional gene therapy because the MECP2 gene is toxic if expressed at high levels.
- Neurogene is grateful for RSRTs support in advancing the scientific knowledge for the treatment of Rett syndrome, which ultimately enabled the early development of the EXACT technology.
- Neurogene is also appreciative of the broader Rett syndrome patient community, including the International Rett Syndrome Foundation and Reverse Rett (UK).
- We hope this novel approach will continue to advance in its development and ultimately provide a much-needed treatment option for families impacted by Rett syndrome.