Improving AAV Production With HeLa Platform


Sam Wadsworth, PhD, chief scientific officer of Ultragenyx Pharmaceuticals, discussed the company’s platform for efficiently producing AAV vectors.

“The HeLa platform is the most advanced platform that we have. It is a highly engineered system for manufacturing AAV gene therapy vectors using HeLa cells. The elements of the platform are finely tuned to work in concert to produce AAV vectors at the scale and quality required for our products."

Ultragenyx Pharmaceuticals has expanded into the field of gene therapy over the last couple of years while developing and evolving its HeLa gene therapy platform. The platform has allowed for the production of greater quality and quantity of AAV vectors.

The company is working on several gene therapies using this platform, including DTX401 for patients with Glycogen Storage Disease Type 1a and DTX301 for patients with ornithine transcarbamylase deficiency. Both programs are currently in phase 1/2 studies (NCT03517085 and NCT02991144, respectively).

The company also recently received orphan drug designation for their mRNA therapy UX053 for the treatment of Glycogen Storage Disease Type III. A phase 1/2 trial is expected to begin enrollment in the second half of 2021.1

GeneTherapyLive spoke with Sam Wadsworth, PhD, chief scientific officer, Ultragenyx Pharmaceuticals, to learn more about the company’s platform and the advantages it provides over other methods of generating AAV vectors. He also discussed a recent partnership with Daiichi Sankyo granting access to Ultragenyx's gene therapy technologies that was initiated in 2020.2

1. 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.
2. Ultragenyx enters into strategic partnership with Daiichi Sankyo for gene therapy manufacturing technology. News release. Ultragenyx. March 31, 2020. Accessed August 6, 2021.
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