Neurogene Announces New Development Program in Rett Syndrome Utilizing Novel EXACT Technology Platform

Promising preclinical data for NGN-401 presented at 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) demonstrate significant survival benefit and reduced disease burden in Rett syndrome mouse model

Results show Neurogene’s EXACT gene therapy regulates MECP2 gene expression to target levels, avoiding overexpression related toxicity

EXACT platform holds potential to develop genetic medicines for complex diseases not addressable by conventional gene therapy

Neurogene Inc., a company founded on the vision to push the boundaries of genetic medicine to address complex and devastating neurological diseases, announced the addition of NGN-401 for Rett syndrome to the Company’s development pipeline. NGN-401 is Neurogene’s first product candidate using the Expression Attenuation via Construct Tuning (EXACT) gene regulation platform technology, and has shown in preclinical models to deliver efficacious levels of MeCP2, while avoiding toxicity observed with conventional MECP2 gene therapy. EXACT is a self-regulating gene therapy platform technology that can be tuned to deliver a desired level of transgene expression within a narrow range, and is compatible with viral and non-viral delivery platforms.

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“I am pleased that NGN-401, our lead program utilizing EXACT technology, has demonstrated a significant survival benefit and reduced disease burden in a robust preclinical Rett model.”

Preclinical efficacy and safety data for NGN-401 were presented today at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Washington, D.C., in an oral presentation of the abstract entitled, “A Self-regulating Gene Therapy for Rett Syndrome.” NGN-401 delivers a full-length human MECP2 transgene, which is under the control of Neurogene’s EXACT self-regulating technology.

“Rett syndrome is a complex disorder not readily addressable by conventional gene therapy because the MECP2 gene is toxic if expressed at high levels. As someone who has been working in Rett syndrome research for more than 15 years, I am pleased that NGN-401, our lead program utilizing EXACT technology, has demonstrated a significant survival benefit and reduced disease burden in a robust preclinical Rett model,” said Stuart Cobb, Ph.D., Neurogene’s Chief Scientific Officer and a Simons Fellow and Reader in Neuroscience at the Patrick Wild Centre and Centre for Discovery Brain Sciences, at the University of Edinburgh. “These data, along with a substantial body of additional preclinical data are foundational to advancing NGN-401 to the clinic for Rett syndrome.” Dr. Cobb’s laboratory has been collaborating with the Rett Syndrome Research Trust (RSRT) and numerous researchers in the field to develop a gene therapy with a strong profile to advance to the clinic.

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Neurogene is grateful for RSRT’s 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).

Monica Coenraads, CEO of the Rett Syndrome Research Trust (RSRT) shared, “We are thrilled that NGN-401 has demonstrated promising preclinical results. RSRT’s long-term support of Dr. Cobb’s laboratory at the University of Edinburgh demonstrates our commitment to drive innovation in research for Rett Syndrome. 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.”

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