Remedium said its lead candidate is the only disease modifying gene therapy based on the clinically proven regenerative mechanism of FGF18, with the developer outlining it could potentially reverse cartilage loss in osteoarthritis (OA) via a single-injection.
“Recent placebo-controlled clinical studies evaluating repeat intraarticular injection of FGF18 demonstrated the ability to regrow cartilage in arthritic knees, while potentially preventing progression to joint replacement surgery. This scientific breakthrough is limited by the need for frequent injections, with efficacy reversing upon discontinuation.”
The biotech will partner with Exothera to advance the therapy through preclinical toxicology studies and eventually human clinical trials.
The contract development and manufacturing organization (CDMO), which specializes in gene therapy, vaccines, and oncolytic viruses, will execute a proof of concept for transient expression of AAV2-FGF18 with suspension culture, based on a HEK-293 cell line.
Exothera will also focus on identifying a high yield production process and reduce cost of goods for the manufacturing process of the therapy.
Earlier this month, Remedium announced the publication of pre-clinical data demonstrating the safety and chondrogenic activity of its gene therapy for OA. “These data are the first to be published from Remedium’s ongoing research collaboration with Tufts University studying the approach.”
It found that AAV2-FGF18 appears to have a wide therapeutic safety window, is able to induce chondrogenesis at low, locally administered doses, and works by increasing hyaline cartilage associated gene expression and promoting chondrocyte proliferation.
AAV2-FGF18 also appears safe and effective at promoting cartilage anabolism in vivo, following a single intra articular injection, according to the paper.
"Our results show the promise of treating osteoarthritis by harnessing the endogenous regenerative power of articular cartilage with a single intra-articular injection FGF18 gene therapy," said Dr Li Zeng, who is associate professor of immunology at Tufts University School of Medicine, co-author of the publication, and an expert in OA and the molecular and cellular mechanisms of the disease.
CIRM backs Genascence's OA targeted gene therapy study
California-based Genascence Corporation is another company exploring the benefits of gene therapy for musculoskeletal diseases. In February, it reported that it had been awarded $11.6m over four years from the California Institute for Regenerative Medicine (CIRM).
The funding will be used to support Genascence's Phase 1b clinical trial of GNSC-001, a recombinant AAV expressing an optimized form of interleukin-1 receptor antagonist (IL-1Ra), in knee OA, as well as phase-appropriate manufacturing activities.
The trial will enroll 50 patients with OA. The study will assess the safety and pharmacodynamics of GNSC-001 at two dose levels, as well as evaluate the effect of GNSC-001 on symptoms and biomarkers of disease progression over time.