One of the pioneers in commercializing one-shot-and-done gene therapies in the eye is pivoting to the knee.
Uncloaking Genascence Corp., CEO Tom Chalberg said Wednesday that the Palo Alto company has dosed nine patients across three cohorts of an early-stage clinical trial of its gene therapy for treating osteoarthritis in the knee. The company spun out of research from the Mayo Clinic, the University of Florida and the University of Minnesota.
Chalberg, who is a Genascence founder with Christopher Evans, Steven Ghivizzani and Paul Robbins, founded Avalanche Biotechnologies Inc. in 2006 and took that company public. He left the company, which would become Adverum Biotechnologies Inc., after inconclusive clinical trial results for its first treatment for a blinding eye disease, then built specialized eyeglass maker SightGlass Vision Inc. before selling it earlier this year to a joint venture of vision giant Cooper Cos. and EssilorLuxottica.
The knee is fresh ground for gene therapy as well, which has focused on eye diseases and other single-gene conditions that could require replacing a defective gene with a correct copy. The promise for patients and the potential of high payouts to companies for fixing chronic conditions has drawn dozens of companies into the space but with mixed results and concerns for patient safety after some deaths have been reported in trials.
Genascence leaders believe their treatment can be safe and effective against osteoarthritis. Early data from the Mayo-sponsored, U.S. Defense Department-funded Phase I trial of GNSC-001 indicated the treatment was safe and tolerable for patients who endure osteoarthritis pain.
The therapy is designed to deliver a coding sequence to block or dampen IL-1, a network of inflammation-promoting cytokines, or small proteins.
The company now will push the therapy ahead to a Phase II company-sponsored study.
"These are important clinical milestones in the development of GNSC-001 as a potential therapy for OA," Chalberg said in a statement. "Osteoarthritis patients have limited treatment options and nothing is currently available that is able to slow down progression of this disabling disease."
