Collaboration Aims to Overcome Delivery Challenges for Neurological Disorders
Why has gene editing for Rett syndrome remained elusive despite its potential to reverse symptoms? Evox Therapeutics Ltd (Evox), a biotechnology company focused on developing innovative therapies for genetically driven neurodegenerative diseases, has announced a strategic collaboration with the Rett Syndrome Research Trust (RSRT). This partnership aims to assess the feasibility of using Evox’s ExoEdit® exosome-based delivery platform to develop a gene editing treatment for Rett syndrome, a severe neurodevelopmental disorder that profoundly impacts motor function, speech, and breathing.
Evox Therapeutics is initiating a collaboration with the Rett Syndrome Research Trust (RSRT) to evaluate the potential of its ExoEdit® technology in delivering gene editing treatments for Rett syndrome. “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. This collaboration could establish the foundation for an expanded preclinical program, ultimately aiming to develop an exosome-enabled gene editing therapy for the majority of Rett syndrome patients whose disease is caused by mutations affecting the MECP2 gene.
Key Insights at a Glance
- Gene Editing Potential: Evox’s ExoEdit® technology has the potential to enable gene editing medicines to safely and effectively reach the brain.
- Rett Syndrome Impact: Rett syndrome profoundly impairs motor function, speech, and breathing, often requiring lifelong, around-the-clock care.
- Collaborative Effort: The partnership with RSRT aims to investigate how exosome-based therapies could provide an urgently needed treatment for Rett syndrome patients.
- Preclinical Goals: The collaboration seeks to establish the foundation for an expanded preclinical program in Rett syndrome.
The Challenge of Safe Gene Delivery
Rett syndrome is a severe genetic neurodevelopmental disorder that predominantly affects girls and is typically diagnosed between the ages of 12 to 18 months. The condition presents with a wide spectrum of clinical manifestations, including loss of speech, purposeful hand use, and difficulty breathing. Over 90% of Rett syndrome cases are caused by mutations in the MECP2 gene, which is essential for neuronal function. The challenge lies in safely and efficiently delivering gene editing therapies to the central nervous system, a critical barrier that has long hindered progress in treating this debilitating condition.
A Fresh Approach to a Persistent Problem
Just as a key can unlock a door, Evox’s ExoEdit® technology aims to unlock the brain’s defenses to deliver gene editing therapies. This innovative approach uses exosomes, which are naturally occurring vesicles that can cross the blood-brain barrier, to transport gene editing cargo directly to the brain. “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. This collaboration represents a significant step forward in addressing the urgent need for effective treatments for Rett syndrome patients.
Evox’s ExoEdit® Technology in Action
Evox Therapeutics is leveraging its ExoEdit® exosome-based delivery platform to overcome the critical challenge of safe and efficient gene delivery to the brain. Evox’s technology has the potential to enable gene editing medicines to reach the brain, making it possible to correct the root causes of devastating neurological diseases such as Rett syndrome.
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,” said Per Lundin, PhD, Co-founder and Chief Executive Officer of Evox. The company’s mission is to revolutionize the treatment of genetically driven neurodegenerative diseases, and this collaboration with RSRT is a crucial step toward achieving that goal.
Future Outlook
The collaboration between Evox and RSRT marks a significant milestone in the development of gene editing treatments for Rett syndrome. If successful, the findings could establish the foundation for an expanded preclinical program, with the ultimate goal of bringing an exosome-enabled gene editing therapy to clinical trials. The potential impact on the lives of Rett syndrome patients and their families is profound, and the scientific community is closely watching this collaboration as a potential breakthrough in the field of neurodegenerative disease treatment.
Conclusion
This partnership between Evox Therapeutics and the Rett Syndrome Research Trust represents a critical step forward in addressing the urgent need for effective treatments for Rett syndrome. For the biotechnology industry, this collaboration highlights the importance of innovative delivery mechanisms in overcoming the challenges of gene editing. How is your organization preparing to leverage such advancements in gene therapy? Join the conversation in the comments below.
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.
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.
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