SynCav1

SynCav1 is an experimental **gene therapy** utilizing an adeno-associated virus serotype 9 (AAV9) vector driven by the synapsin promoter to achieve neuron-targeted overexpression of **Caveolin-1 (Cav-1)**, a key scaffolding protein that organizes membrane/lipid rafts (MLRs) essential for neurotrophin signaling, synaptic plasticity, and neuroprotection. Developed initially at UC San Diego, it has demonstrated robust preclinical efficacy in preserving hippocampal-dependent memory, synaptic integrity, and neuronal activity in symptomatic Alzheimer's disease (AD) mouse models (PSAPP and APPKI), while also delaying disease onset, extending survival by ~10-19%, and maintaining motor function in amyotrophic lateral sclerosis (ALS) models (hSOD1G93A mice and rats) independent of targeting mutant SOD1. By restoring MLR-localized signaling (e.g., preserving PAC1R and ADNP expression), SynCav1 counteracts neurodegeneration, downregulates pathogenic pathways (AD, PD, ALS), and upregulates cognition/synaptic pathways, positioning it as a broad neuroprotective candidate for CNS disorders.[1][2][3][5]

Other names

SynCav-1SynCav1SynCav 1