ASC Therapeutics, U Mass Medical School, and the Clinic for Special Children Announce Podium Presentation of Safety and Efficacy in Murine and Bovine Models for Novel Gene Therapy in Maple Syrup Urine Disease at the 25th ASGCT Meeting
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Saturday, May 21, 2022
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The partnership designed a dual-function AAV9 gene replacement vector that was administered via intravenous (IV) injection.
Key Points:
- The partnership designed a dual-function AAV9 gene replacement vector that was administered via intravenous (IV) injection.
- Surviving mice grew and behaved similar to normal littermates and had normal or nearly normal biochemical markers with unrestricted diet for 16 weeks.
- As compared to MSUD patients, MSUD calves exhibit a similar phenotype and are closely matched for size and metabolic rate.
- In doing so, it has built a reputation as a world-class research institution and as a leader in primary care education.