CHDI Foundation is exclusively dedicated to collaboratively developing therapeutics that will substantially improve the lives of individuals with Huntington’s disease. Even with this clarity of mission, there is a practical limit to the number of initiatives that can be orchestrated in parallel. Therefore, CHDI must prioritize where our resources will be most effective in HD drug discovery and development. This requires a transparent strategic framework that ensures alignment among the various stakeholders, from biology to chemistry, early discovery to translational to clinical, as well as the important integration of CHDI staff with the broader community in the academic, biopharmaceutical, and regulatory sectors.

Our preclinical research programs are organized into four Thematic Areas, each covering a broad area of R&D activity that, in concert, will offer the greatest likelihood of delivering effective therapeutics to HD families as soon as possible. Our Thematic Areas cover basic research and assessing /prosecuting therapeutic opportunities for targeting 1) mHTT DNA, RNA, & Protein; 2) HTT CAG tract somatic instability & DNA mismatch repair (MMR) pathway enzymes 3) mHTT Therapeutic Gene Editing and 4) ‘Non-MMR’ Genome-Wide Association (GWA) HD disease modifier genes.

Additionally, we expend significant resources dedicated to ‘Transversal Capabilities’: activities that apply to – and impact – all Thematic Areas, these being: Chemistry; Biomarkers Discovery and development; Human Genetics & Phenotyping; HD Brain ‘Deep Phenotyping’; Therapeutic Indexing in Preclinical Systems (TIPS) and External Partnership enablement. We also continue to monitor areas of interest such as cell-based therapies, innovative delivery methods, transformative technologies, and findings from human based studies.

If you would like more information on preclinical activities or would like to discuss other areas of HD biology then please contact .