Genomic and Biologic Therapies for ADRD (U01 Clinical Trial Optional)

The National Institutes of Health, under the Department of Health and Human Services, has issued solicitation RFA-NS-28-012 to support the translation of genome editing and biologic therapies for Alzheimer's Disease-Related Dementias (ADRD). This includes Frontotemporal Dementia, Lewy Body Dementias, Vascular Contributions to Cognitive Impairment and Dementia, and Multiple Etiology Dementias. The program funds the entire translational pipeline, from therapeutic lead optimization and drug product development to IND-enabling studies and early first-in-human clinical evaluations. Key activities include optimizing editing constructs and delivery systems, demonstrating in vivo efficacy, conducting safety pharmacology and toxicology studies, and developing scalable manufacturing processes that align with regulatory expectations. The agency intends to commit 6 million dollars in both fiscal years 2028 and 2029, with an expected three awards and a maximum project period of five years. Individual annual budget caps are set at 1.3 million dollars in direct costs, increasing to 2.5 million dollars for projects involving clinical trials. Awards are governed by the Public Health Service Act and federal regulations 42 CFR Part 52 and 2 CFR Part 200. Evaluation is based on an Overall Impact Score considering significance, innovation, approach, investigator expertise, and environment, with a specific focus on the Target Product Profile and the use of quantitative Go/No-Go milestones. Eligible applicants include small businesses and other for-profit organizations. Requirements for submission include active registration in the System for Award Management, a Unique Entity Identifier, and eRA Commons accounts. Awardees must adhere to strict cybersecurity incident reporting protocols, notifying the agency within 48 hours of discovery, and must register applicable clinical trials with ClinicalTrials.gov. The solicitation features multiple application cycles with deadlines extending through October 10, 2028, and earliest start dates ranging from December 2027 to July 2029.