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SynCav1 is an experimental, systemically delivered gene therapy that uses an AAV-PhP.eB vector to deliver the caveolin-1 gene under a synapsin promoter for neuron-specific expression. It crosses the blood-brain barrier to upregulate the neuroprotective protein caveolin-1 throughout the brain and spinal cord. Rather than clearing toxic protein aggregates, SynCav1 strengthens intrinsic neuronal resilience by preserving membrane lipid rafts, stabilizing synaptic structures, and protecting mitochondria. It is being investigated as a neuron-centric therapeutic candidate for TDP-43 proteinopathy-associated neurodegenerative diseases including ALS, frontotemporal dementia, and Alzheimer's disease.
SynCav1 is an experimental, systemically delivered gene therapy that uses an AAV-PhP.eB vector to deliver the caveolin-1 gene under a synapsin promoter for neuron-specific expression. It crosses the blood-brain barrier to upregulate the neuroprotective protein caveolin-1 throughout the brain and spinal cord. Rather than clearing toxic protein aggregates, SynCav1 strengthens intrinsic neuronal resilience by preserving membrane lipid rafts, stabilizing synaptic structures, and protecting mitochondria. It is being investigated as a neuron-centric therapeutic candidate for TDP-43 proteinopathy-associated neurodegenerative diseases including ALS, frontotemporal dementia, and Alzheimer's disease.
SynCav1 is an experimental gene therapy that employs a modified adeno-associated virus serotype PhP.eB (AAV-PhP.eB) to deliver the caveolin-1 (Cav-1) gene under a synapsin promoter, enabling neuron-specific expression. Following systemic delivery, the vector crosses the blood-brain barrier and upregulates caveolin-1 expression throughout the brain and spinal cord. Caveolin-1 is a master neuroprotective protein that organizes critical signaling pathways and preserves membrane lipid rafts (MLRs) — specialized subcellular structures essential for neuronal communication. Mechanistically, SynCav1: (1) prevents pathological TDP-43 from mislocalizing to membrane lipid rafts; (2) stabilizes MLR-associated GluN2A expression, a component of NMDA receptors critical for synaptic plasticity; (3) preserves synaptic ultrastructure; (4) mitigates TDP-43-induced mitochondrial hyper-fragmentation and excessive mitochondrial fission signaling. Rather than directly clearing toxic protein aggregates, SynCav1 strengthens intrinsic neuronal resilience, allowing neurons to withstand disease-related metabolic stress even when toxic proteins are present.
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