Potential Gene Therapy for ALS and Similar Diseases Licensed to CavoGene LifeSciences

The University California San Diego (UC San Diego) has licensed the right to develop and commercialize an investigational gene therapy for amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease, called SynCav1, to CavoGene LifeSciences.

The therapy may also benefit people with traumatic brain and spinal cord injuries, and cognitive decline disorders.

Caveolin-1 (Cav-1) is a protein found in the membranes of cells, and in neurons (nerve cells) is one of the key players for synapses — the junctions between nerve cells that allow them to communicate —and, consequently, neuronal communication.

SynCav1 is a gene therapy that targets Cav-1 expression to nerve cells, using a specific “controller” sequence that is only present in neurons, the so-called synapsin promoter.

UC researchers have shown that SynCav1 can enhance survival and growth signaling of primary nerve cells, as well promoting growth in axons and dendrites, specialized projections of nerve cells — dendrites bring information to the cell body and axons carry information away from it.

In a subsequent study, they showed that SynCav1 improved synapses and cognitive behavior in old mice, suggesting its potential as a therapy for neurodegenerative disorders.

In an animal model of ALS, researchers found that SynCav1 preserved the animals’ body weight, improved their motor function — the animals performed better on the running wheel — and delayed disease onset by one month. The therapy also extended survival, and showed evidence of improving their learning and memory.

These preclinical findings support the potential of SynCav1 to improve cognitive function and quality of life of ALS patients and others affected by neurodegenerative disorders.

“Cav-1 is a neuronal cell membrane scaffolding protein, analogous to a coat hanger in one’s closet,” Brian Head, PhD, an associate adjunct professor at the UC San Diego School of Medicine and a co-inventor of SynCav1 said in a press release.

“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,” added Head, who is also a Veterans Affairs‘ research scientist.

CSSi LifeSciences, contracted by CavoGene, will manage the preclinical, good manufacturing practices, and all regulatory and clinical development aspects of SynCav1, the release states. The terms of these agreements were not disclosed.

“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,” said Jim Sergi, president of CSSi LifeSciences.