Plowshare Licenses MSUD Gene Therapy from UMass Chan
Plowshare has licensed an investigational gene therapy developed at UMass Chan Medical School to address two genetic forms of Maple Syrup Urine Disease.
Plowshare Therapies has licensed an investigational gene therapy for Maple Syrup Urine Disease (MSUD) from UMass Chan Medical School. The therapy was designed to address mutations associated with MSUD types 1A and 1B by delivering functional copies of two affected genes.
The approach was developed by researchers at UMass Chan and evaluated in preclinical models. Published findings showed that the therapy prevented newborn death, normalized growth, restored coordinated expression of the affected genes, and stabilized key biomarkers in animal models. These results are encouraging, but they do not establish that the therapy is safe or effective in people. Licensing the technology gives Plowshare responsibility for helping guide its continued development toward clinical evaluation. It is an important step in carrying promising science beyond the laboratory, but much work remains before the therapy can be studied in patients.
Plowshare brings to that work decades of clinical experience with MSUD and deep relationships within the communities most affected by the disease. We are grateful for the opportunity to help advance this research with the care, scientific discipline, and sense of responsibility that children and families deserve.