GLEN BURNIE, Md.—CavoGene LifeSciences divulged today that it has licensed the rights for the development and commercialization of SynCav1, an investigational gene therapy for patients with neurodegenerative diseases, such as Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease (AD), traumatic brain and spinal cord injury, and age-related cognitive decline, from the University of California, San Diego (UC San Diego). SynCav1 is a novel gene therapy intervention that restores and augments pro-growth signaling, axonal and dendritic growth, and formation of new functional synapses in animal models.

 

SynCav1 was developed by Brian P. Head, PhD, a Veterans Affairs’ research scientist and associate adjunct professor in UC San Diego School of Medicine’s Department of Anesthesiology; and co-inventors Hemal H. Patel, PhD, professor and vice-chair for research; Piyush M. Patel, MD, professor; and David M. Roth, MD, PhD, professor. The data demonstrate that SynCav1 improves motor function, maintains body weight and extends survival in ALS rodent models, increases structural and functional neuroplasticity, and improves learning and memory in aged mice and in an AD mouse model.

 

“Cav-1 is a neuronal cell membrane scaffolding protein, analogous to a coat hanger in one’s closet,” said Head. “By recruiting and organizing synaptic receptors and associated signaling components together in close proximity, Cav-1 allows for the enhancement of functional synapses and neuroplasticity. In addition, we believe that this novel gene therapy could also work in combination to enhance the efficacy of compounds that activate synaptic signaling complexes.”

 

SynCav1 represents a new paradigm that uses a single genetic intervention, namely neuron-targeted Caveolin-1 overexpression (e.g., SynCav1), to re-establish polarized plasma membrane signaling microdomains (termed membrane/lipid rafts, MLRs), with improved neurotrophin and synaptic signaling and enhanced neuroregeneration and innervation. Clinically, SynCav1 has the therapeutic potential to improve cognitive function, and to improve the quality of life for those afflicted with neurodegenerative disorders.

 

CavoGene has contracted with CSSi LifeSciences to manage the preclinical, GMP, regulatory and clinical development of SynCav1.

 

Jim Sergi, President of CSSi LifeSciences, stated, “We are excited by the opportunity to leverage our experience and subject matter expertise to advance the commercial development of this potential disease modifying therapy.”