ARTICLE SUMMARY:
Pioneers working on gene therapies for osteoarthritis and other chronic joint conditions won the prestigious OREF Award for 2024 at this year’s AAOS. One of the winners, Christopher Evans, PhD, discusses 30 years of research leading up to the founding of Genascence, which is now in clinical trials for a gene therapy for OA.
Osteoarthritis, one of the most common and debilitating chronic diseases, affects more than 30 million Americans, a number that is expected to increase exponentially as the population ages.
Therapeutic options are limited.
Current approaches start with nonpharmacological treatments such as walking aids, weight loss, and physical therapy, followed by acetaminophen and NSAIDS. When these fail, intra-articular injections of steroids and hyaluronic acid can provide temporary relief from pain, but the data does not support their efficacy and the former may accelerate disease progression. Beyond that, options are slim, and none of the treatments modify underlying causes of disease or stop its progression. The ultimate treatment to date is total joint replacement surgery.
Genascence, a start-up founded in 2017, aims to break that logjam, transforming the treatment of musculoskeletal (MSK) diseases with single-dose gene therapies. Its technology is based on pioneering research conducted by three scientists who began studying the effectiveness of gene therapy on MSK conditions more than 30 years ago, at a time when no one else was doing so and the nascent gene therapy field was focused almost exclusively on cancer and single-gene Mendelian conditions.
