Application Granted: 3/9/2021
Inventors: Brian P. Head Ph.D., Professor Department of Anesthesiology UC San Diego (Tenure) and VA Research Career Scientist; Piyush M. Patel M.D., Professor Department of Anesthesiology UC San Diego; Hemal Patel Ph.D., Professor Department of Anesthesiology and Vice-Chair for Research UC San Diego; and David M. Roth Ph.D., M.D., Professo Department of Anesthesiology UC San Diego and VA Medical Center

Caveolin-1 (Cav-1) is a cholesterol-binding and membrane-scaffolding protein that plays a critical role in cellular functions such as synaptic signaling, endocytosis, vesicular trafficking, and mitochondrial regulation. This patent introduces a novel gene therapy approach for neurodegenerative diseases, including Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease), as well as for ischemic and traumatic brain injuries. The therapy enables neuron-specific overexpression of Cav-1 through a synapsin (Syn) promoter of the human Cav-1 gene sequence (termed SynCav1). In a significant advancement, the SynCav1 gene therapy has been licensed and is now advancing toward an Investigational New Drug (IND) filing, marking a key milestone on the path toward clinical and regulator development.

“SynCav1 evokes neuroprotective and pro-survival signaling pathways in various neurodegenerative disease models such as AD and ALS, independent of decreasing the mutant monogenic component attributed to the disease. The ability of SynCav1 gene therapy to afford neuroprotection in various disease indications regardless of etiology, makes it an attractiv therapeutic candidate for both familial and sporadic forms.”

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