Therapeutic Development Initiatives
Clinical and scientific initiatives are key to the advancement of our six priority strategies.
Cures aren’t developed in isolation. They require a myriad of resources that encourage and enable progress. Spanning from clinical to basic science, collectively these resources create a tool kit that supports drug discovery.
With thousands of potential disease indications to choose from, biopharmaceutical companies gravitate to those diseases that are translationally mature, the ones that have many of these resources in place.
By spearheading and supporting these foundational initiatives, RSRT seeks to encourage and facilitate biopharma engagement.
Individuals with Rett are at the heart of everything we do at RSRT. And they are also at the heart of drug development. Our clinical resources are focused on learning as much as possible from those affected by Rett to maximize the effectiveness and efficiency of clinical trials, as well as inform the scientific process of drug development.
All of our Cure 360 strategies will require the delivery of therapeutics to the brain and ideally the ability to regulate how much of the MECP2 protein is made. Understanding how the MECP2 protein functions informs the ability for regulation. Our science resources increase our foundational knowledge base, exponentially increasing our chances of success.