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FORNY20-FORNY2020

KVAL: Gene therapy for osteoarthritis and other inflammatory conditions

Alternative title: Genterapi mot artrose og andre inflammatoriske sykdommer

Awarded: NOK 0.50 mill.

Project Number:

329243

Project Period:

2021 - 2022

Funding received from:

Organisation:

Location:

RNA therapy for osteoarthritis and other inflammatory conditions Osteoarthritis (OA) is the most common joint disease leading to pain, physical incapacities, reduced quality of life for hundred millions of people worldwide. The prevalence of OA is on the rise and there is no disease modifying treatment. The only available treatments are pain relieving options like painkillers and physiotherapy. The final outcome is often total joint replacement. RNA therapy might become a game changer for these patients. microRNAs are small RNA molecules that can turn certain genes on and off, which makes them powerful therapeutic agents. Osteoarthritis is a complex disease where several factors and drivers are involved. This project investigated inflammation as one of the drivers behind OA. The project aim to tackle the disease using specific microRNAs that has shown strong inhibitory effects against inflammation and other important immune responses. In this way one might be able to protect the cartilage and the whole joint from the destructive inflammatory response and prevent joint degradation. In this project both in vitro and vivo studies were performed with the chosen microRNAs. Results showed that the microRNAs downregulated central immune genes, which support and strengthen the commercial potential for the microRNAs. Based on positive results in the animal study an international PCT patent application was submitted. In the project period industry partnering was performed, and although the project is at an early stage it has attracted industry interest due to the high unmet need in OA.

OA is a global disease that is on the rise, thus the need for effective disease-modifying treatments is high. Intra-articular administration of 5’ isomiR is a novel gene therapeutic strategy with a potential to stop the degenerative process in patients with OA. Results from this project have brought the science one step closer to a commercialization and have developed the drug candidate further. The intended drug would have a considerable impact on both the individual patient and on society. On the individual level it would reduce pain, enhance the patients' mobility, activity and quality of life. On the societal level it would save the society considerable amount of money and keep its members active and at work.

Funding scheme:

FORNY20-FORNY2020