Preclinical Results from Osteoarthritis Gene Therapy Studies Published by GeneQuine and Collaborators in Leading Arthritis Journal [ 14 Sep 2018 ]

A scientific paper reporting preclinical small and large animal data of the gene therapy approach for osteoarthritis developed by GeneQuine and Baylor College of Medicine was published in “Arthritis & Rheumatology”, a leading peer-reviewed journal in the field. The data suggest symptomatic and disease-modifying efficacy in osteoarthritis treatment.

Preclinical Results from Osteoarthritis Gene Therapy Studies Published by GeneQuine and Collaborators in Leading Arthritis Journal

The approach was originally developed at Baylor College of Medicine (Houston, TX) and was exclusively licensed by GeneQuine Biotherapeutics (Hamburg, Germany). Based on the technology, GeneQuine has been developing drug candidates for osteoarthritis in humans, horses and dogs. In December 2017, Flexion Therapeutics Inc. (Nasdaq: FLXN) acquired the human drug candidate FX201 (formerly known as GQ-203) in a milestone-based deal worth up to $64 million. GeneQuine has retained the rights to the veterinary drug candidates GQ-201 (horses) and GQ-202 (dogs).

The technology is based on helper-dependent adenoviral vectors expressing interleukin-1 receptor antagonist. In conjunction with Baylor College of Medicine and Cornell University (Ithaca, NY), GeneQuine conducted preclinical studies in mouse and horse osteoarthritis animal models. The results of these studies, which suggest symptomatic as well as disease-modifying efficacy, have now been published in “Arthritis & Rheumatology”, one of the leading peer-reviewed journals in the field (read the article on Wiley Online Library).

Alan Nixon, BVSc, MS, professor and director of comparative orthopedics laboratory at Cornell University, and first author of the publication commented: “Interleukin-1 is widely regarded as the principal degrading agent in osteoarthritis, and blocking its action with interleukin-1 receptor antagonist is a key therapeutic strategy in this chronic debilitating disease. The positive results in these animal models heralds the possibility of long term osteoarthritis control in clinical cases.”

Kilian Guse, PhD, Chief Executive Officer of GeneQuine and senior author of the publication said: “These are very significant findings as we have demonstrated in small and large animal models that the gene therapy approach has the potential for symptomatic and disease-modifying efficacy in osteoarthritis. These data pave the way for further development of the technology, which Flexion is advancing to the clinic.”

The study also establishes helper-dependent adenoviral vectors as a suitable vector technology for joint gene therapy, which is of particular significance for GeneQuine as all of the company’s drug candidates are based on this promising vector type. After the recent acquisition of its lead program by Flexion, GeneQuine is now focusing on the development of a second gene therapy program for osteoarthritis (GQ-303). Furthermore, GeneQuine is expanding the application of the vector technology to other diseases in the musculoskeletal space. (Image by