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PR009
- Development stage
- Preclinical
- Lead developer
- Eli Lilly
- Modality
- Gene Therapies
- Administration
- Intrathecal
01
Overview
PR009 (LY3884965) is an investigational recombinant adeno-associated virus (rAAV) gene therapy designed to deliver a functional human triggering receptor expressed on myeloid cells 2 (hTREM2) gene packaged in a microglia-tropic capsid. Developed by Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company, PR009 is designed as a disease-modifying, one-time treatment for adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) associated with colony-stimulating factor 1 receptor (CSF1R) mutations (ALSP-CSF1R). By increasing TREM2 expression in brain microglia, PR009 aims to compensate for the loss of CSF1R function, thereby restoring microglial survival, health, and function. The therapy is currently in preclinical development.
02
Targets
TREM2 (Triggering receptor expressed on myeloid cells 2)
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