Who we are
Created through a partnership between CHDI Foundation, a privately-funded nonprofit biomedical research organization devoted solely to developing therapeutics for Huntington’s disease (HD), and the Critical Path Institute (C-Path), the Huntington’s Disease Regulatory Science Consortium (HD-RSC) was formed 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 (HD) is a rare inherited neurodegenerative disorder for which there are currently no effective therapeutics. The degeneration of neurons in the brains of individuals affected by HD results in behavioral changes, including depression and irritability, disruption of daily activities, as well as a loss of motor function and cognitive ability. Research indicates that changes in the brain occur up to 15 years prior to the onset of motor symptoms, highlighting the need for 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 incentive 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 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. CHDI has the broad network, scientific expertise, and resources to enable the success of the HD consortium. The HD-RSC thereby represents a collaborative initiative that builds upon CHDI’s established leadership in the HD community and C-Path’s expertise and prior successes in regulatory and data science.
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 consortia (Critical Path for Parkinson’s consortium, Duchenne Regulatory Science Consortium, Critical Path for Alzheimer’s Disease, Multiple Sclerosis Outcomes Assessments Consortium). 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 integrated 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.