SPARC Seminar Series: Dr Brendan Lee

Описание: Chronic joint pain from degenerative joint diseases such as osteoarthritis (OA) affects more than 250 million individuals around the world, a number that will continue to rise as our population ages. Available treatments show limited efficacy at slowing OA disease progression or combatting pain, so patients rely on ineffective treatments that carry significant health and dependency risks. Gene therapy represents a promising therapeutic avenue for OA because it can be delivered locally and mediates long-lasting expression of target genes.

During this SPARC seminar, Dr. Brendan Lee will discuss the translational pipeline his group developed for designing novel gene therapeutic strategies to reduce OA pain and disease progression, test them in small and large animal models, and initiate clinical trials to test their safety and efficacy in patients with OA.

To highlight this pipeline’s potential, Dr. Lee will highlight its most successful therapeutic candidate, HCAd-IL1Ra, which obtained RMAT designation from the FDA and is entering phase 2 clinical trials. Finally, Dr. Lee will discuss the need for incorporating combinatorial or repeated dosing approaches into gene therapy study designs and related preclinical studies to maximize their translational potential and their efficacy for treating complex diseases.

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