Recursion Pharmaceuticals has received support for focused work to identify a treatment for Rett Syndrome by the Rett Syndrome Research Trust. Recursion will apply its innovative drug discovery platform immediately to investigate the gene MECP2 in an effort to identify new therapeutics for the condition.
June 9, 2014
Recursion Pharmaceuticals, a biotech start-up in Salt Lake City, announced today receipt of a research award from the Rett Syndrome Research Trust that will enable the company to put its powerful drug discovery platform to work in the hunt for a treatment for Rett Syndrome.
Rett syndrome (RTT) is a physically disabling disease, and one of the most common genetic causes of severe impairment in young females. There is no cure for RTT despite the strong efforts of researchers and patient groups. Over 95% of RTT patients are identified to have mutations within the MECP2 gene, many of which are predicted to cause a loss of function of the gene.
Dr. Chris Gibson, CEO of Recursion Pharmaceuticals adds: “We are thrilled to be working to find a treatment for Rett Syndrome. The Rett Syndrome Research Trust has generously provided us with support and trust to do just that, and we will work diligently to apply our technology in the search for unexpected treatments for the condition.”
Rett Syndrome is an ideal candidate for Recursion’s novel research approach due to: (1) its strong genetic basis, (2) its postnatal onset, and (3) convincing evidence for successful rescue of the phenotype in an adult animal model. Recursion Pharmaceutical’s efficient drug discovery technology has the potential to rapidly bring FDA approved drugs to Rett Syndrome patients.
Recursion combines experimental biology and bioinformatics in a unique drug screening platform. Recursion creates human cellular models of disease and establishes a disease profile based on identifying changes in thousands of structural (morphological) and functional (activity) parameters. These structural and functional changes are then used as the basis of a chemical suppressor screen to identify compounds with strong potential for efficacy in the disease model. This unbiased approach to rescue of the disease profile has proven successful where other target-centric hypotheses have failed.
About the Rett Syndrome Research Trust – The Rett Syndrome Research Trust is a non-profit exclusively devoted to global research on Rett Syndrome and related MECP2 disorders. Their goal is to heal children and adults who will otherwise suffer the effects of these disorders for the rest of their lives. Their partners in supporting this work are parent’s organizations worldwide including Reverse Rett (UK), Rett Syndrome Research & Treatment Foundation (Israel), Rett Syndrome and CDKL5 Ireland, Skye Wellesley Foundation (UK), Stichting Rett Syndrome (Holland), Rett Syndrom Deutschland e.V., and American organizations, Girl Power 2 Cure , the Kate Foundation for Rett Syndrome Research, Eva Fini Fund at RSRT, Rocky Mountain Rett Association, Anastasi Fund, Claire’s Crusade and the New Jersey Rett Syndrome Association. See www.RSRT.org for more information.
About Recursion Pharmaceuticals – Recursion Pharmaceuticals, LLC, is a drug discovery company founded in 2013 in Salt Lake City, Utah. Recursion uses a novel drug screening platform to efficiently reposition drugs to treat rare genetic diseases. The company’s focus on rare genetic diseases is much-needed, as there are more than 5,000 such conditions that together affect millions of Americans, and more than 95% of these diseases have no approved therapy. Recursion’s novel drug screening platform combines experimental biology and bioinformatics in a massively parallel system to quickly and efficiently identify treatments for multiple rare genetic diseases. The core of the approach revolves around high-throughput automated screening using high-content assays in human cells, which allows the near simultaneous modeling of hundreds of genetic diseases. Rich data from these assays is probed using advanced statistical and machine learning approaches, and the effects of thousands of known drugs and shelved drug candidates can be investigated efficiently to identify those holding the most promise for the treatment of any one rare genetic disease.