Genascence Launches Phase 1b Trial for Knee OA Gene Therapy

Genascence Corporation, a biotechnology firm specializing in gene therapy for musculoskeletal diseases, has launched a Phase 1b clinical trial for its GNSC-001 treatment targeting knee osteoarthritis (OA). The trial, named DONATELLO, is currently enrolling patients at ten medical centers across the United States, with an aim to include around 50 participants by early 2024.

GNSC-001 is a novel genetic medicine that utilizes a recombinant adeno-associated viral vector to deliver an optimized version of IL-1Ra, a protein that naturally inhibits the inflammatory effects of interleukin-1 (IL-1). IL-1 is a significant contributor to OA's pathogenesis, leading to inflammation, joint pain, and cartilage breakdown. The therapy is intended to provide a long-lasting suppression of IL-1 with just a single injection, potentially offering a new approach to managing this debilitating condition.

Thomas Chalberg, the CEO of Genascence, highlighted the importance of this trial, stating that OA is a condition that can cause significant pain and disability over many years, and currently, there are no treatments that can slow the progression of the disease. The DONATELLO trial represents a significant advancement in the development of gene therapy for OA and other prevalent musculoskeletal diseases.

Lachy McLean, Genascence's chief medical officer, expressed optimism about the potential of GNSC-001 to change the treatment landscape for OA patients. The company anticipates sharing preliminary data from the trial after six months, which is expected to be in the final quarter of 2024.

The DONATELLO trial is a randomized, double-blinded, placebo-controlled study that will assess the safety, tolerability, and pharmacodynamics of GNSC-001 when administered as a single intra-articular injection. The study is designed to provide valuable insights into the therapy's effectiveness in managing knee OA, a condition that affects over 30 million Americans and is associated with a significant economic burden due to medical costs and loss of productivity.

Knee OA is a progressive joint disease characterized by the degradation of cartilage and changes in bone structure, leading to pain and reduced joint function. The prevalence of OA is increasing due to an aging population and the rise in obesity rates. Current treatments, such as NSAIDs, opioids, and steroid injections, only manage pain without addressing disease progression.

Genascence was established in 2017 and has licensed technology from renowned U.S. research institutions, including the Mayo Clinic, University of Florida, and NYU Langone Health. The company's leadership has extensive experience in developing and manufacturing gene therapies and biological medicines, positioning Genascence at the forefront of innovative treatments for musculoskeletal diseases.