Here’s a shocking statistic: in 2020, 7.6% of the world’s population was living with the most common form of arthritis – osteoarthritis (OA). That amounts to 595 million people globally and 32.5 million adults in the U.S.

That’s a lot of people with painful knees, hips, and other joints. Although pain medication can help with alleviate symptoms, there is no cure for this progressive condition.

The California Institute for Regenerative Medicine (CIRM) has funded four teams working toward possible therapies for osteoarthritis. One of these teams just released some good news about the results of an early trial called DONATELLO. This particular trial was testing a therapy for osteoarthritis in the knee, which is the most common location for the condition.

The Palo Alto-based company, Genascence, is using a gene therapy approach to block a molecule called IL-1 that causes osteoarthritis. Their gene therapy produces a molecule called IL-1Ra that binds IL-1 present in the joint and will, hopefully, prevent the disease from getting worse or possibly even reverse it. They call their gene therapy GNSC-001.

Genascence conducted a Phase 1b trial, which was looking at whether the approach was safe and whether IL-1Ra remained present in the knee joint twelve months following injection. The company recently announced that they were successful with both outcomes.

“These results from the DONATELLO trial reflect the kind of innovation CIRM was created to support,” said Lisa Kadyk, PhD, CIRM Fellow, Clinical Development at CIRM, which supported the DONATELLO clinical trial with a $12 million award. “By harnessing the power of gene therapy, GNSC-001 represents a novel and potentially disease-modifying approach to treating osteoarthritis. We are encouraged by the 12-month safety and biomarker data and proud to have supported this important step toward a more effective, long-term treatment for people living with knee OA.”

In their statement, Genascence said that they had met with the U.S. Food and Drug Administration to discuss a larger Phase 2 trial to bring their potential therapy closer to the millions of people in California and worldwide who are waiting for a cure. We always caution that a successful Phase 1 trial still requires several additional steps to prove that a therapy is safe and effective before it reaches patients. But it is heartening to see progress, especially for diseases that impact so many people’s lives.

Written by guest contributor Amy Adams