In 2017, RSRT launched the first ever three-year, $33 million research plan, Roadmap to a Cure, founded on scientific knowledge generated over many years. The goal was to identify and advance all possible genetic medicine avenues that address the root cause of Rett.
Families around the world rallied to raise the necessary funds. Remarkably, at the start of Roadmap there were ZERO biopharmaceutical companies with genetic medicines targeting MECP2, by the end there were SIX!
Roadmap to a Cure not only laid the foundation for the current gene therapy clinical trials but it set the stage for the exciting genetic medicines being developed today.
Roadmap Pillars
Cure
The heart and soul of Roadmap included all of the genetic medicine programs.
Treat
Non-genetic approaches that could improve a symptom(s) but not meant to be curative.
Enable
Efforts to leverage patient data, develop objective symptom assessment tools and discover biomarkers.
Learn
Basic science that enabled the pursuit of genetic medicines. A major focus being the MECP2 Consortium.
