Who we are
The Huntington’s Disease Regulatory Science Consortium (HD-RSC) was formally launched in 2018 with the goal of improving the regulatory path for emerging therapies for Huntington’s disease. This global initiative will facilitate collaboration among biopharmaceutical industry partners, technology industry partners, academic institutions, government agencies, and patient-advocacy organizations. The HD-RSC fosters consensus and data-driven research to increase efficiency, safety, and speed in developing new therapies.
Huntington’s disease is an autosomal-dominant neurodegenerative disorder caused by expansion of a CAG repeat in the huntingtin gene that encodes a mutant huntingtin protein, and is characterized by devastating motor impairment, cognitive decline, and neuropsychiatric disturbances. Research suggests that changes in the brain occur up to 15 years prior to the onset of motor symptoms, highlighting the need for a better understanding of disease progression and early intervention.
The overall goal of the HD-RSC is to create a regulatory science strategy for HD therapeutics that will offer additional incentives to drug developers and help further de-risk HD therapeutic development. The HD-RSC will provide a forum and framework to bring together the necessary participants from the HD community to contribute data and develop tools, leading to efficiencies in the development of novel therapies.
How we do it
C-Path serves as a neutral third party to bring all stakeholders together, share data, and develop novel methodologies under the advisement of global regulatory agencies.
To achieve success, the HD-RSC will leverage a deep knowledge of HD gained from working with patients, families, researchers, neurologists, and clinical scientists, as well as previous experience and learnings from other C-Path neuroscience consortia. The HD-RSC will facilitate interaction between biotech and pharmaceutical industry partners with regulatory agencies to work towards the qualification or regulatory acceptance of drug development tools, biomarkers, and better clinical assessments.
The HD-RSC will collect and standardize natural history and clinical trial data from HD patients around the world to develop an aggregated disease database of patient-level data. We will use this to gain a better understanding of disease progression and work towards model-informed drug development to de-risk preclinical HD programs and accelerate the global regulatory approval of urgently needed HD therapies.