Dimension Therapeutics receives positive opinion for EU ODD of DTX401
Dimension Therapeutics announced that the European Medicines Agency, or EMA, Committee for Orphan Medicinal Products has issued a positive opinion recommending DTX401 for designation as an orphan medicinal product for the treatment of Glycogen Storage Disease Type Ia, or GSDIa. The positive opinion for DTX401 is a key milestone for Dimension's leading and unique program advancing the opportunity for AAV gene therapy for patients with limited treatment options to manage their disease. Designed to address the underlying genetic defect, DTX401 has been shown in multiple in vivo preclinical models to deliver stable expression and activity of the enzyme glucose-6-phosphatase-alpha. DTX401 has also been shown to improve G6Pase-alpha activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. Dimension has completed candidate selection for its DTX401 program and is currently in IND-enabling studies.