Benzinga | Dec 1, 2021 08:07AM EST

Ultragenyx Reports First Patient Dosed In Phase 1/2 Study Of UX053 For Treatment Of Glycogen Storage Disease Type 3

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that the first patient has been dosed in its Phase 1/2 study of UX053, an investigational messenger RNA (mRNA) therapy in development for the treatment of Glycogen Storage Disease Type III (GSDIII).

"Initiation of this trial paves the way for UX053 to potentially become the first medicine for people living with GSDIII, who currently are burdened with managing a complex metabolic disorder with diet alone," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "We believe mRNA is a promising treatment modality for rare diseases like GSDIII because this technology can deliver large genes to targeted cells and achieve a high uniformity of protein expression. We can also individualize mRNA therapies based on the needs of each patient by adjusting the dose level and dose frequency."

UX053 is Ultragenyx's most advanced investigational mRNA therapy. The company is developing a preclinical pipeline of mRNA therapies through its long-term collaboration with Arcturus Therapeutics Inc., a clinical-stage messenger RNA medicines company. More information about Ultragenyx's broad portfolio of rare disease medicines can be found here: https://www.ultragenyx.com/our-research/our-pipeline/