Evox Therapeutics enters partnership to evaluate new gene editing technology for Rett Syndrome

27 February 2026

Evox Therapeutics, a biotechnology company developing innovative therapies for genetically driven neurodegenerative diseases using a next-generation gene editing modality, has announced it is initiating a collaboration with the Rett Syndrome Research Trust (RSRT) to assess the feasibility of using the company’s ExoEdit® exosome-based delivery platform to enable the development of a gene editing treatment for Rett syndrome.

Rett syndrome is a severe neurodevelopmental disorder that profoundly impairs motor function, speech, and breathing, often requiring lifelong, around-the-clock care and specialized teams to manage complex, multi-system symptoms.

Per Lundin, Co-founder and CEO of Evox Therapeutics, said:

“Gene editing offers the possibility of correcting the mutations responsible for Rett syndrome and potentially reversing symptoms even after disease onset, though safe and efficient delivery to the central nervous system remains a key challenge.

“Our ExoEdit® technology has the potential to enable gene editing medicines to safely and effectively reach the brain, making it possible to leverage gene editing to correct the root causes of devastating neurological diseases such as Rett syndrome. We are honored to partner with RSRT to investigate how our exosome-based therapies could provide an urgently needed treatment for Rett syndrome patients and their families.”

If the collaboration is successful, the findings could establish the foundation for an expanded preclinical program in Rett syndrome with the ultimate goal of developing an exosome-enabled gene editing therapy for the large majority of Rett syndrome patients whose disease is caused by mutations affecting the MECP2 gene.

Monica Coenraads, Founder and Chief Executive Officer of RSRT , said:

“Non-viral delivery to the brain has long been a major obstacle to establishing gene editing as a therapy for Rett syndrome. We see great potential in Evox’s ExoEdit® technology to efficiently and safely deliver editing cargo throughout the brain. My colleagues and I are enthusiastic about this study and look forward to seeing the results, which may open the door to a new clinical pathway for Rett patients.”

www.evoxtherapeutics.com

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Evox Therapeutics enters partnership to evaluate new gene editing technology for Rett Syndrome