CHDI Foundation is a privately-funded, nonprofit biomedical research organization devoted to a single disease – Huntington’s disease (HD). Our mission is to develop therapeutics that will provide meaningful clinical benefit to patients as quickly as possible. To achieve this CHDI manages a diverse portfolio of research projects through a novel virtual model that encourages scientific collaboration to more directly connect innovative research, drug discovery, and clinical development. This helps bridge the translational gap that often exists between academic and industrial research pursuits, and which adds costly delays to therapeutic development. Our activities extend from exploratory biology to the identification and validation of therapeutic targets, and from drug discovery and development to clinical studies and trials.

We work in a number of different ways:

• We fund and collaborate with academic HD researchers around the world.
• CHDI staff scientists manage our own internal therapeutic programs through our collaborations at contract research organizations.
• We work in partnership with biotech and pharmaceutical companies to develop potential therapeutics.
• We act as a ‘collaborative enabler’ for any research group that wants to work on HD by making resources—such as clinical data & biosamples, animal models, assays, reagents, antibodies—available to the HD research community.
• We work to build clinical infrastructure and capacity so that as soon as promising therapeutics are ready the wider HD research community can quickly progress to clinical trials.

Essentially, CHDI is a science management organization. We have assembled a team of scientists and other professionals working in offices in New York, Los Angeles, and Princeton, and our science directors and project managers work closely with a network of more than 700 researchers in academic and industrial laboratories around the world in the pursuit of novel therapeutics, providing strategic scientific direction and management to ensure that our common goals remain in focus.

CHDI’s overall strategy is to de-risk therapeutic approaches and develop them to the stage that pharmaceutical company partners will view them as a worthwhile investment and take them on to full clinical development, including licensure and marketing to get therapeutics to HD patients.

Since we are nonprofit we have no competitors, and our bottom line is ensuring the shortest possible time to getting effective therapeutics to HD patients. As such, a big part of CHDI’s remit is to collaboratively enable any researcher that is interested in working on HD by lowering the barrier to entry through the provision of HD domain knowledge, reagents, protocols, animal models or funding. In our role as a collaborative enabler, CHDI seeks to bring the right partners together to identify and address critical scientific issues to increase the understanding of HD and hasten the development of therapeutic approaches to clinical evaluation as rapidly as possible.

CHDI also manages and funds Enroll-HD, a worldwide HD clinical research platform and observational study launched in 2012 that has now enrolled more than 28,000 participants at 155 clinical sites in 23 countries in Europe, North America, Latin America, and Australasia. Enroll-HD supports the design of clinical trials and enables faster and smarter recruitment, makes all clinical data and biosamples collected available to the wider HD research community, and helps improve clinical care for HD. Visit the Enroll-HD website to learn more.

CHDI also works closely with colleagues at the HD advocacy organizations around the world to forge links between HD families and researchers/clinicians to speed the drug development process.