Who we are
Created in partnership with CHDI Foundation, a privately-funded, not-for-profit biomedical research organization devoted solely to Huntington’s disease, and the Critical Path Institute, the Huntington’s Disease Regulatory Science Consortium (HD-RSC) was formed with the aim of improving the efficiency of emerging therapies for Huntington’s disease. This global initiative aims to facilitate collaboration among the bio-pharmaceutical industry partners, technology industry partners, academic institutions, government agencies, and patient-advocacy associations. HD-RSC fosters consensus and data-driven research to increase efficiency, safety, and speed in developing new therapies.
Huntington’s disease (HD) is a rare, inherited genetic disorder for which no cure currently exists. The degeneration of nerve cells of the brain in HD results in a loss of motor function and muscle control, a loss of cognitive ability (thinking), and mood changes including depression and irritability. Research indicates that changes in the brain occur up to 15 years prior to the onset of motor symptoms, highlighting the need for better disease progression understanding and early intervention.
The overall goal of the Huntington’s Disease Regulatory Science Consortium (HD-RSC) is to create a regulatory science strategy for HD, offering additional incentive and de-risking for HD therapeutic development by all stakeholders. HD-RSC will provide a forum and structure to bring together the necessary participants from the HD community for data contribution and tool development, leading to efficiencies in development of new therapies.
How we do it
Critical Path Institute serves as the neutral 3rd party to bring all stakeholders together, share data, and develop novel methodologies under the advisement of global regulatory agencies. CHDI Foundation has the broad network, scientific experts, and resources to enable the success of the HD consortium.
Thus, HD-RSC represents a collaborative initiative that builds upon CHDI’s established leadership in HD and C-Path’s expertise and previous successes in regulatory and data science.
To achieve success, HD-RSC will leverage a deep knowledge of HD gained from working with patients, researchers, neurologists, and clinical scientists as well as learnings from other C-Path consortia (Critical Path for Parkinson’s consortium, Duchenne Regulatory Science Consortium, Critical Path for Alzheimer’s disease, Multiple Sclerosis Outcomes Assessments Consortium). 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.
HD-RSC will collect and standardize natural history and clinical trial data from HD patients around the world to develop an integrated disease database of patient level data. Using this database, we will 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.