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Gene Therapy Shown to Build Knee Cartilage

DNA illustration

(DennisM2, Flickr.

13 Mar. 2023. Findings from research with lab animals shows a single gene therapy injection delivered with a benign virus helps build new knee cartilage cells and tissue. Results of the study, by a team from Remedium Bio Inc. in Needham, Massachusetts and Tufts University medical school in Boston appear on 10 Mar. in the the journal Cartilage.

The study tests an experimental treatment for osteoarthritis in development by Remedium Bio, code-named RMD1101. Arthritis is a common condition associated with aging, with all forms of arthritis affecting some 58.5 million people in the U.S. or about a quarter (24%) of adults, according to Centers for Disease Control and Prevention. Osteoarthritis occurs when articular cartilage for protecting joints wears down due to wear and tear over the years, with symptoms developing slowly and becoming worse over time in most people. Symptoms from inflammation in osteoarthritis include stiffness, pain, loss of flexibility, swelling, tenderness, and bone spurs that in some cases can develop around the joints.

Remedium Bio is a three year-old biotechnology enterprise developing gene therapies for age-related and immune conditions. RMD1101 is the company’s lead product, designed to treat inflammatory conditions affecting the joints, beginning with osteoarthritis. The treatment is a synthetic form of the fibroblast growth factor 18 gene that codes for the FGF18 protein expressed in a number of human cells and tissues, but in this case is critical for connective tissue and bone development. Remedium Bio delivers RMD1101 with a newer engineered adeno-associated virus, a small and benign microbe that has become the workhorse for delivering gene therapies, called AAV2.

Measured thickness of articular cartilage

In the study, researchers from Remedium and Tufts University medical school first tested RMD1101 in lab cultures that shows the synthetic FGF18 gene promotes expression of other genes coding for collagen and hyaluronic acid needed for production of cartilage. The team then injected the knees of lab rats with either a single dose of the FGF18 gene therapy delivered with AAV2, or two injections per week of FGF18 protein over six weeks, or a saline-solution placebo. The researchers measured development of cartilage by the thickness of articular cartilage throughout the knee structure of the rats, as well as watching for adverse reactions.

Results show the gene therapy and protein injections encourage similar cartilage growth in the animals’ knee joints, across the medial meniscus — the band of cartilage across the knee joint — and on the tibial plateau, the top of the tibia bone in the shin below the knee. However, over the two-month study period, swelling in the rats’ knee joints — a sign of adverse effects — occurs most often in recipients of the multiple protein injections or higher-dose gene therapy. The lower-dose gene therapy recipients show little knee swelling, suggesting a safer option.

“Our results,” says Tufts University immunologist Li Zeng, a co-author of the paper in a Remedium Bio statement released through Cision, “show the promise of treating osteoarthritis by harnessing the endogenous regenerative power of articular cartilage with a single intra-articular injection FGF18 gene therapy.” Zeng is a scientific adviser to the company.

Remedium Bio joins at least one other company experimenting with gene therapies to treat osteoarthritis. In Feb. 2023, Science & Enterprise reported on Genascence Crop. in Palo Alto, California that gained state funding for a clinical trial of its gene therapy for osteoarthritis.

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