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Genascence Starts Enrollment in Knee Osteoarthritis Gene Therapy Phase Ib Trial

NEW YORK – Genascence on Wednesday said it is recruiting patients at 10 US sites for a Phase Ib clinical trial of GNSC-001, an investigational gene therapy for knee osteoarthritis.

Investigators plan to enroll about 50 patients in the study, dubbed DONATELLO. In the randomized, placebo-controlled, dose-ranging trial, Genascence will assess the safety, tolerability, and pharmacodynamics of a single injection of GNSC-001 in patients with knee osteoarthritis, a progressive disease that leads to pain and loss of joint function.

GNSC-001, the firm's lead program, uses a recombinant adeno-associated viral vector to deliver a coding sequence for an optimized form of interleukin-1 receptor antagonist (IL-1Ra), a protein that inhibits IL-1 signaling. IL-1 is thought to be one of the key mediators in the pathogenesis of osteoarthritis, resulting in inflammation, joint pain, and cartilage destruction.

"Osteoarthritis is incapacitating, causing years of pain and disability for people living with the disease, and there are no currently available treatments to slow down disease progression," said Genascence Founder and CEO Thomas Chalberg said in a statement.

Palo Alto, California-based Genascence expects to complete enrollment in Q1 2024 and report initial six-month data from the trial in Q4.