Axon Spheroid-Induced CNS Disorders
Lead Innovator: Jaime Grutzendler, MD
IP: Patents Pending
Related: OCR8237
Pathological Neuronal Branching: Plaque-Associated Axonal Spheroids (PAAS)
- Axon Conduction Disorders
- Alzheimer's Disease
- Other diseases of nerve conduction
- Standard of Care: None
- Novelty: no drugs target PAAS
- In Vivo Model: mouse model recapitulates PAAS pathology of human post-mortem brain samples (Fig. 1)
- Axonal Spheroid-enriched Targets
- Target 1: endolysosomal resident enzyme
- Target 2: plasma membrane integral protein
- Intervention Strategies: small molecules, siRNA/antisense, antibody, PROTACS
- In Vivo Assessment of Efficacy: brain interhemispheric axonal conduction
Validity of Therapeutic Hypothesis
- Human/Mouse: human and mouse PAAS morphology (Fig.1)
- Mouse: diminished PAAS (observed) results in normalization of axonal conductance (two targets, two modalities; Fig.2a: Target 1/ Fig.2b:Target 2)
Anticipated Clinical Assessment of Efficacy of PAAS-directed Therapy
- Neuropsychological and computer-assisted measurements of cognitive processing speed/reaction times.