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AAV.PHPeB
- Development stage
- Preclinical
- Lead developer
- Regel Therapeutics
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV.PHPeB is an engineered adeno-associated virus (AAV) capsid platform derived from AAV9, specifically optimized for high-efficiency crossing of the blood-brain barrier (BBB) and widespread neuronal transduction following systemic (intravenous) administration. Developed at the California Institute of Technology, it is widely used in preclinical research to deliver gene modulation tools to the central nervous system. In the development of therapies for SCN2A haploinsufficiency, such as those by Regel Therapeutics, AAV.PHPeB has been utilized to screen and characterize neuron-specific regulatory elements (REs) and to deliver payloads like dCas9-VP64. These payloads are designed to upregulate the SCN2A gene, thereby restoring functional levels of the Nav1.2 sodium channel protein to treat associated neurodevelopmental disorders, including autism and epilepsy.
- Other names
- AAV-PHP.eB
02
Targets
Ly6A (Lymphocyte antigen 6 complex locus A)AAVR (AAV receptor)
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