Selecting AAV Manufacturing Platforms


The chief scientific officer and chief medical officer of Ultragenyx Gene Therapy discussed the company’s future research in gene therapies.

“Our gene therapy pipeline, which is really a part of a much broader Rare Disease pipeline within Ultragenyx, includes both early and late-stage programs. Originally, we were really focused on the liver, with liver directed gene therapy really focused on inborn errors of metabolism.”

Ultragenyx Pharmaceuticals has expanded into the field of gene therapy via their evolving HeLa platform. The platform has been an integral part of improving efficiency in gene therapy manufacturing.

The company is targeting several diseases with gene therapies, including glycogen storage disease Type 1a and ornithine transcarbamylase deficiency, which they are developing DTX401 (NCT03517085) and DTX301 (NCT02991144), respectively.

Their mRNA therapy, UX053, which is being developed for the treatment of glycogen storage disease Type 3, also recently received orphan drug designation. The therapy will be evaluated in a phase 1/2 trial which will begin enrollment in the second half of 2021.

GeneTherapyLive spoke with Sam Wadsworth, PhD, chief scientific officer, and Eric Crombez, MD, senior vice president and chief medical officer, Ultragenyx Gene Therapy, to learn more about future targets for the company’s gene therapies. They also discussed the manufacturing plant set to open in 2023 that they believe will further improve their efficiency.

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.
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