Addressing Unmet Needs With Gene Therapy

“We've really tried to focus on diseases... with a high unmet medical need... and with the highest likelihood of success. With the broad platform modalities we have built within Ultragenyx, with small molecules, enzyme replacement therapy, gene therapy, and more recently messenger RNA, we can really focus in on these diseases... with high unmet medical need... and then choose which modality is the best way to take on these diseases.”

Ultragenyx Gene Therapy, part of Ultragenyx Pharmaceuticals, is targeting diseases with high unmet medical needs for their gene therapy development program. They are exploring adeno-associated virus vector (AAV)-directed gene therapy in indications such as glycogen storage disease (GSD), ornithine transcarbamylase (OTC) deficiency, Wilson disease, and Duchenne muscular dystrophy (DMD).

Their lead programs are phase 3 studies of DTX401 for the treatment of GSD type 1a (NCT03517085) and DTX301 for the treatment of OTC deficiency (NCT02991144). UX053, an mRNA therapy, also recently received orphan drug designation for the potential treatment of GSD type 3 and will be evaluated in a phase 1/2 trial beginning enrollment in the second half of 2021.

GeneTherapyLive spoke with Eric Crombez, MD, senior vice president and chief medical officer, Ultragenyx Gene Therapy, to learn more about the indications the company has chosen to target. He also discussed the HeLa platform that their DMD program will be using and the company’s discussions with the FDA.

REFERENCE

Ultragenyx receives Orphan Drug Designation from FDA and European Commission for UX053, an investigational mRNA-therapy for the treatment of Glycogen Storage Disease Type III. News release. Ultragenyx Pharmaceuticals. July 27, 2021. https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-receives-orphan-drug-designation-fda-and-european#