Optimization of Genome Editing Therapeutics for Alzheimer’s Disease-Related Dementias (ADRD) (U01 – Clinical Trials Not Allowed)
Funding Opportunity Number: RFA-NS-24-009
Deadline: September 6, 2023
Award Amounts up to $3,000,000.
Purpose
This Notice of Funding Opportunity (NOFO) supports the optimization of promising genome editing-based therapeutic leads for Alzheimer’s Disease-Related Dementias (ADRD), towards IND-enabling studies. Specifically, it supports the characterization and optimization of therapeutic lead(s) that show promise as potential genome editing therapeutics, as evidenced by convincing proof-of-concept studies in appropriate models. At the end of the funding period, successful projects will have optimized a genome editing therapeutic candidate with demonstrated bioactivity, manufacturability, biodistribution, in vivo efficacy, and/or target engagement (measurement of proximal downstream effects) and optimal dosing, combined with other properties consistent with the desired clinical application.
For more information, please see the funding opportunity website.