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GNSC-001 is a first-in-class gene therapy designed to offer long-term relief of musculoskeletal diseases such as osteoarthritis.
The FDA granted fast track designation (FTD) to GNSC-001, a first-in-class gene therapy that could potentially offer a treatment for patients with osteoarthritis (OA) of the knee, according to a news release from Genascence Corporation.1
As a genetic medication, GNSC-001 is a recombinant adeno-associated vector (AAV) that expresses an optimized form of interleukin-1 receptor antagonist (IL-1Ra), a naturally occurring protein that blocks the signaling of interleukin-1 (IL-1).1
IL-1 is widely considered one of the key mechanisms behind the pathogenesis of OA. It can cause inflammation, joint pain, and cartilage destruction. Following a single injection into the affected knee, GNSC-001 is designed to offer long-term, sustained inhibition of IL-1.1
"Osteoarthritis is incapacitating, causing years of pain and disability for people living with the disease. Current treatment options are short-term, and while they can provide temporary relief of symptoms, they do not slow down or reverse disease progression,” Thomas Chalberg, PhD, founder and CEO of Genascence, said in the news release.1
Phase 1 clinical trial data of GNSC-001 for the treatment of OA indicated that it is well-tolerated in patients, according to Genescence. The findings showed that a single intra-articular injection resulted in elevated IL-1Ra expression over baseline in synovial fluid for the length of the study, which lasted 12 months.1
Furthermore, patients treated with GNSC-001 reported improvement of pain and function scores, with a limited amount of disease progression, both positive indicators of the effectiveness of the drug.1
"Fast track designation from the FDA underscores the serious unmet medical need in patients with widespread, debilitating diseases like OA," Chalberg said. “We believe GNSC-001 has potential to deliver transformative results, and we are excited to advance our clinical program so we can help patients suffering from this disabling disease."1
OA, a degenerative joint disease, is characterized by the destruction of cartilage and structural changes in the bone within the joint, contributing to pain and loss of joint function. The disease affects millions of Americans and is a leading cause of disability in conjunction with an increasingly aging population and the increasing prevalence of obesity.1
In addition to GNSC-001, other potential treatments for OA are in the clinical trial pipeline, signifying advancements made in the treatment of the debilitating disease. Recently, the FDA granted FTD to MM-II, a non-opioid product, for the treatment of OA-related knee pain. The product uses proprietary suspension of liposomes to relieve joint pain in patients.2
As new products are evaluated for their safety and efficacy and become poised to enter the drug market following their approval, pharmacists’ role in the management of patients with OA and the recommendation of proper therapies will be paramount. Beyond therapy recommendations, early detection of OA—and prompt treatment initiation—is essential to preventing joint destruction.3
Especially in retail settings, pharmacists should make themselves available to patients for counseling and discussion regarding their OA management and treatment strategies. Pharmacists can aid patients with selecting proper medications, but they can also monitor disease progression and offer educational resources, making them a valued member of a patient’s care team.3
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