Evox Therapeutics Ltd (“Evox” or the “Company”), a biotechnology company developing innovative therapies for genetically driven neurodegenerative diseases using a next-generation gene editing modality, today 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.

“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,” said Per Lundin, PhD, Co-founder and Chief Executive Officer of Evox. “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.

“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,” said Monica Coenraads, Founder and Chief Executive Officer of RSRT and parent of an adult daughter with Rett syndrome. “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.”

About Rett Syndrome

Rett syndrome (RTT) is a severe genetic neurodevelopmental disorder that predominantly affects girls and is typically diagnosed between the ages of 12 to 18 months. RTT presents with a wide spectrum of clinical manifestations with the most common including loss of speech and purposeful hand use. Patients may also lose the ability to walk, have difficulty breathing and experience gastrointestinal issues. Presently, there is no cure, and treatment utilizes a multidisciplinary approach that may include physical therapy, speech therapy, and occupational therapy in addition to medications for certain symptoms. Over 90% of RTT cases are caused by mutations linked to the methyl-CpG-binding protein 2 (MeCP2) gene that is essential for neuronal function and a target for potential treatment through gene editing.

About Rett Syndrome Research Trust

The Rett Syndrome Research Trust is the only non-profit organization exclusively focused on advancing genetic medicines for Rett syndrome. It is the largest funder of Rett research worldwide. Multiple clinical trials in gene therapy for Rett syndrome are now underway, all possible because of RSRT-funded research. In 2024, RSRT launched a $40 million initiative, Roadmap to Cures, with a goal to bring three genetic medicines, beyond the current gene therapies, to clinical trials by 2028. RSRT’s mission is clear, urgent, and personal: to cure Rett syndrome and transform the lives of the people we love. To learn more, please visit ReverseRett.org.

About Evox Therapeutics

By advancing a pipeline of next-generation gene editing medicines, Evox’s mission is to revolutionise the treatment of genetically driven neurodegenerative diseases. Backed by leading investors and with an unrivalled intellectual property estate, the Company’s pipeline programs target the MSH3 gene for the treatment of Huntington’s disease and other trinucleotide repeat disorders and the ATXN2 gene for the treatment of amyotrophic lateral sclerosis and other TDP43 pathologies. To learn more, please visit www.evoxtherapeutics.com.

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