Biopharmaceutical company ASC Therapeutics disclosed on Tuesday that it will co-develop the first in-vivo gene therapy for Maple Syrup Urine Disease (MSUD) in partnership with the University of Massachusetts Medical School (UMMS).
MSUD is reportedly a severe genetic disease with liver transplantation as the only treatment currently available. The rare genetic disorder affects the degradation of the branched-chain amino acids (BCAA) leucine, isoleucine and valine and their ketoacid derivatives.
Led by Guangping Gao, PhD, director of the Horae Gene Therapy Center at UMMS and Dan Wang, PhD, assistant professor of RNA therapeutics, ASC will develop multiple animal models for MSUD, as well as conduct pre-clinical testing using animal models and proprietary AAV constructs.
In conjunction, ASC Therapeutics receives an exclusive option on the selected constructs and intellectual property resulting from this partnership.
The partnership will jointly conduct further IND-enabling studies, regulatory and manufacturing activities to obtain US FDA's IND clearance and advance this programme into clinical stage.
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