Genascence has commenced its Phase Ib clinical trial designed to evaluate GNSC-001, a gene therapy for the treatment of knee osteoarthritis (OA).

The randomised, double-blinded, placebo-controlled, dose-ranging trial, named DONATELLO, is currently enrolling patients across ten clinical centres in the US.

It aims to assess the tolerability, safety, and pharmacodynamics of a single intra-articular injection of GNSC-001 in patients with knee OA, a debilitating joint disease and the primary cause of disability.

Approximately 50 patients are expected to be enrolled in the clinical study, to complete enrolment by the first quarter of 2024.

GNSC-001 is a genetic medicine utilising a recombinant adeno-associated viral vector to express an optimised form of Interleukin-1 receptor antagonist (IL-1Ra), a protein that inhibits interleukin-1 (IL-1) signalling.

IL-1 is a significant factor in OA pathogenesis, leading to inflammation, joint pain, and cartilage destruction. The GNSC-001 therapy aims to provide long-term relief with a single injection into the affected joint.

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Genascence chief medical officer Lachy McLean said: “Our GNSC-001 gene therapy has the potential to transform the treatment landscape for individuals suffering from OA.

“We look forward to sharing initial six-month data from the DONATELLO clinical trial in the fourth quarter of 2024 and continue advancing our clinical programme so we can bring a new option to patients as quickly as possible.”

Knee OA leads to cartilage destruction and bone structural changes, resulting in pain and loss of joint function.

Genascence founder and CEO Thomas Chalberg said: “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.

“We are excited that our Phase IB DONATELLO clinical trial is now fully up and running at ten clinical sites across the US. This is a critical next step toward the development of the first gene therapy for prevalent musculoskeletal diseases like OA.”

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

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