CHDI Foundation is a privately-funded, not-for-profit biomedical research organization devoted to a single disease – Huntington’s disease. Our mission is to develop drugs that will slow the progression of Huntington’s disease and 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 academic 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 work with academic HD researchers at universities around the world
- Our staff scientists manage our own internal drug programs through our collaborations at contract research organizations
- We work in partnership with biotech and pharmaceutical companies to develop potential drugs
- We act as a ‘collaborative enabler’ for any research group that wants to work on HD by making resources (reagents, antibodies, mouse models, clinical samples, etc.) freely available to the HD research community.
- We work to build clinical capacity so that as soon as promising potential drugs are ready we, and the wider HD research field, can quickly progress to clinical trials.
Essentially, CHDI is a science management organization. CHDI has assembled a team of scientists and other professionals working in offices in New York City, Los Angeles, and Princeton, NJ, and our science directors and project managers work closely with a network of more than 600 researchers in academic and industrial laboratories around the world in the pursuit of these novel therapies, 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 good investment and take them on to full clinical development, including licensure and marketing to get drugs to HD patients. We’ve recently had some notable progress in this regard; an anti-sense oligonucleotide therapeutic approach that CHDI helped develop with Ionis Pharmaceuticals was recently the subject of substantial investment from the pharmaceutical company Roche, and we are currently working with Pfizer to evaluate their phosphodiesterase 10 inhibitor in humans.
As a not-for-profit entity 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 Huntington’s disease 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 is also the sponsor of a worldwide Huntington’s disease observational study and registry called Enroll-HD that has participants in the US, Canada, Latin America, Europe, Australia/New Zealand; the aim is to recruit 20,000 HD family members around the world to build a research platform that will enable faster and smarter clinical trial recruitment.
We also work closely with colleagues at the patient advocacy organizations (like the HDSA, HDA, HSC etc.) around the world to forge links between patients/families and researchers/clinicians to speed the drug development process.