IRSF Collaboration Opportunities For Industry
Our Foundation has the experience and tools you need to accelerate your Rett syndrome treatment development program.
IRSF has established resources to facilitate every phase of Rett syndrome treatment development. We offer them to industry at no cost for research that aligns with our goals. Let’s work together to address the unmet need for treatments for Rett syndrome, a genetic neurodevelopmental disorder that causes severe disabilities in children and impacts every aspect of their lives.
Learn more about how we support our industry partners. Contact us.
Mark Carlton, PhD, Cerevance CSO
Acadia discussing IRSF’s support of their Trofinetide clinic
Christopher U. Missling, PhD, President and Chief Executive
Rett syndrome is a rare disorder that affects 1 in every 10,000 children worldwide. The disorder typically appears between 6 and 18 months of age and dramatically increase in severity in early childhood. It causes impairments that impact almost all aspects of an individual and their family members’ lives. There are currently no approved treatments for Rett syndrome. However, we believe that life-altering treatments are within reach because Rett syndrome has:
- One Causal Gene
- A Predictive Mouse Model
- 15 Years of Longitudinal Natural History Data
- Established Centers of Excellence
- Clinical Endpoints in Development
THERAPEUTIC TARGETS & APPROACHES
Research has identified several targets and approaches that show promise for treating Rett syndrome. These include:
- Sigma-1 Receptor
As the leading Rett syndrome research and advocacy organization, the International Rett Syndrome Foundation (IRSF) builds upon our nearly 40-year commitment to breakthrough discoveries and life-changing advancements in research toward a cure while supporting families affected by Rett syndrome.
Through our legacy foundation pioneers, we have invested over $54M in research leading to identifying Rett syndrome’s cause, proving Rett syndrome is reversible in mice and developing multiple clinical trials for Rett syndrome. We fight for families living with Rett syndrome and a world without it.
- Funded the discovery of the gene that causes Rett syndrome (MECP2)
- Funded the discovery of reversibility of Rett syndrome in mice
- Funded early gene therapy research in Rett syndrome
- Funded the first Rett syndrome clinical trial
- Funded the development of the first Phase 3 clinical trial
- Convened parents and experts to publish the Rett Syndrome Handbook
- Designated 15 Rett Syndrome Centers of Excellence
Companies We’ve Worked With
We have our finger on the pulse of all relevant emerging science and technologies, and we won’t stop until we have treatment options for all those living with Rett syndrome. We’re ready to share our knowledge and experience with those who share our goals. Contact us to get started.