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Drug Development/Novel Targets: Novel therapeutic agents (Milestone 6.D)

In Progress

Timeline Start - End

2017 - 2024

Research Implementation Area

Drug Development - Novel Targets

Initiate drug discovery efforts to develop novel therapeutic agents against at least 6 novel therapeutic targets (a minimum of three targets for presymptomatic and early stage disease and a minimum of three for advanced disease).

Success Criteria

  • Complete preclinical development, through IND filing, of at least 12 therapeutics agents against at least 3 novel targets (at least one novel target should be for presymptomatic disease).

  • Invest in the expansion of therapeutic modalities including natural products, gene therapy, antisense oligonucleotides, cell therapy, etc.

Summary of Key Accomplishments

Fourteen new drug candidates developed with support from NIA’s translational funding initiatives have received Investigational New Drug (IND) status from the FDA to initiate human trials. These drug candidates are targeting 11 novel therapeutic targets and three amyloid-beta-related targets. One of these drug candidates is a vaccine, one is a gene therapy drug, and the remaining 12 are small molecules/pills. Eight of these drug candidates are in or are anticipated to start Phase 1 trials, and six have advanced to Phase 2 testing. An additional 18 new therapeutic agents are being developed, targeting multiple aspects of the disease. Of these, three therapeutic agents are expected to receive IND status in 2022. Among these, two therapeutic agents are immunotherapies, one is a vaccine, and one is stem-cell therapy.

The key accomplishments summary is current as of July 2022. 

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