A cure for Rett Syndrome. You want it. I want it. More than anything. So how is the Rett Syndrome Research Trust (RSRT) accomplishing this most ambitious goal?
Founded on scientific knowledge generated over many years, in early 2017 RSRT launched a three-year, $33 million research plan, Roadmap to a Cure. The goal was to identify, launch and expand therapeutic programs that target the root cause of Rett Syndrome: MECP2 mutations.
RSRT has delivered on our plan. With the passionate support of our families and their networks we raised the necessary $33 million and we launched, expanded or facilitated 15 therapeutic programs. These programs, mostly academic, fall into six strategic areas (gene replacement, gene editing, MECP2 reactivation, RNA editing, RNA trans-splicing, protein replacement) all of which target Rett at its core.
While the majority of scientific discoveries are made in academic labs, drugs are developed by biopharma. The gap between academia and biopharma is often referred to as the “Valley of Death” because all too often it’s where discoveries go to die. Navigating the transition from academia to biopharma is critically important and a key focus of RSRT.
Remarkably, at the start of Roadmap there were ZERO companies with therapeutic programs targeting MECP2. Today, there are SIX with more to come. While Roadmap resulted in many important accomplishments, this alone is palpable evidence of our impact over the last three years.
But we can’t stop now. We need biopharma to advance Rett programs in all of the therapeutic strategic areas. The cure will come from one or more of these strategies, of that we have no doubt. So the more shots on goal the better.
So today we launch the next phase of our attack on Rett Syndrome, CURE 360. In this next phase RSRT is equally focused on driving forward the six therapeutic strategies AND on business development. CURE 360 further evolves RSRT from a disease research organization into an MECP2 therapeutic incubator, ensuring that biopharma picks up academic programs advanced by us. In fact, we feel so strongly about the business development aspect of our work that we have modified our logo.
Our goals over the next several years are to continue to incubate projects in each of the six therapeutic strategies, to ensure that additional biopharma companies launch Rett programs in all of the therapeutic strategies, and to assist companies in their MECP2 therapeutic clinical development efforts.
RSRT now has a proven track record of identifying the key therapeutic strategic areas, recruiting top scientists to work in these areas, raising the necessary funds to support these scientists, and, importantly, executing on the challenging transition from academia to biopharma.
No words I say here could adequately express how grateful I am to my colleagues, to the Rett community, and to all our donors for your trust, your confidence and importantly, your ongoing support. We would not be in this exciting new phase without you. Your involvement, energy and generosity will be more vital than ever in this next phase of the research.
Upwards and onwards!