The Structural Genomics Consortium (SGC) and CHDI Foundation have entered into a unique open-access research collaboration to discover and characterize new drug targets for Huntington’s disease (HD) using structural and chemical biology In this first partnership of its kind, SGC and CHDI have explicitly agreed not to file for patents on any of the collaborative research and to make all reagents and knowledge available without restriction to the wider research community, including pharmaceutical, biotech, and academic research groups.
The SGC is a public-private partnership accelerating discovery of new drugs through an open-access research approach, with particular focus on neglected areas of the human genome, creating an open collaborative network of scientists in hundreds of universities and in nine major pharmaceutical companies. The collaboration with CHDI—a not-for-profit drug development organization with a mission to rapidly develop drugs that will slow the progression of HD—further focuses this approach within a specific disease domain. As part of the collaboration, CHDI will support scientists at the SGC to generate research tools to solve protein structures of potential drug targets for HD.
“This is a pioneering move by CHDI, and a template for how patient-orientated funders can help the research community develop new drugs,” said Aled Edwards, CEO of the SGC. “With this collaboration the SGC is now pushing our open-access research to exploit specific chemical probes and define protein structures in a particular disease, in order to further clarify the underlying disease biology and aid drug discoverers in academia, biotech and pharma with the highest quality information about new drug targets.”
The SGC will make any research tools developed freely available to the research community immediately and without restriction; working with a disease foundation will enable a targeted dissemination and rapid uptake of the developed research tools within the specific disease field.
“Ours is a knowledge-driven industry. By providing open access to their discoveries, the SGC and CHDI are doing exactly what is needed to help us discover and develop new medicines,” said Tetsuyuki Maruyama, Head of Research at Takeda Pharmaceuticals. “This targeted approach within a specific disease domain will invite pharmaceutical sector researchers to look more closely at Huntington’s disease as a tractable neurodegenerative disorder.”
Since no patent protection on the results of this collaboration will be sought, any investigator will have complete freedom to operate. As part of the collaboration CHDI will facilitate the dissemination of SGC’s existing novel inhibitors of epigenetics pathways into its network of scientists and clinicians to uncover potential new therapeutic targets for HD.
“We often refer to CHDI as a collaborative enabler with the aim of stimulating drug discovery research in Huntington’s disease,” said Robi Blumenstein, President of CHDI Management. “CHDI has long operated under the principle that the underlying disease biology should be freely accessible in a pre-competitive manner. This collaboration with the SGC will expand that pre-competitive domain to include biological structures, chemical probes and associated research data. Our aim is to ensure the HD research community has access to the best possible reagents and tools and that findings are made available rapidly and with the fewest restrictions to attract as many researchers as possible to work on HD. The ultimate goal is to get effective drugs to patients in the shortest possible time”.
About Huntington’s disease
Huntington’s disease is an inherited neurodegenerative disorder caused by a mutation in the huntingtin gene. The defect causes a DNA sequence called a CAG repeat to occur many more times than normal. Each child of a parent with a mutation in the huntingtin gene has a 50% chance of inheriting the mutation. As a result of carrying the mutation, an individual’s brain cells degenerate leading to behavioral, cognitive, and motor impairments that, over the course of the disease, significantly reduce the individual’s quality of life and ultimately cause death within 15 to 25 years of overt symptom onset. There are currently no therapeutics approved that slow the progression of Huntington’s disease.
About the Structural Genomics Consortium (SGC)
With active research facilities at the Universities of Toronto and Oxford, the not-for-profit organization supports the discovery of new medicines by carrying out open-access research in structural and chemical biology. More than 200 researchers in academia and in nine pharmaceutical companies collaborate within SGC to accomplish these goals. The SGC is also funded by the Canadian Foundation for Innovation, Genome Canada, the Ontario Ministry of Economic Development and Innovation, the Wellcome Trust and nine pharmaceutical companies. More information is available at www.thesgc.org
About CHDI Foundation, Inc.
CHDI Foundation, Inc. is a privately-funded, not-for-profit, biomedical research organization that is exclusively dedicated to rapidly discovering and developing therapies that slow the progression of Huntington’s disease (HD). As a collaborative enabler, CHDI seeks to bring the right partners together to identify and address critical scientific issues and move drug candidates to clinical evaluation as quickly as possible. Our scientists work closely with a network of more than 600 researchers in academic and industrial laboratories around the world in the pursuit of these novel therapies, providing strategic scientific direction to ensure that our common goals remain in focus. More information about CHDI can be found at http://chdifoundation.org/.