Clinical Research, Huntington's Disease

Critical Path Institute (C-Path) is a nonprofit, public-private partnership with the Food and Drug Administration (FDA), created under the auspices of the FDA’s Critical Path Initiative program in 2005.  

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.