Selecting AAV Manufacturing Platforms

Video

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.

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#
Related Videos
Daniel Hart, PhD, on CRISPR-Mediated, In Vivo Epigenomic Activation
Luke Roberts, MBBS, PhD, on Developing Gene Therapy for Congestive Heart Failure
PJ Brooks, PhD, on Improved Newborn Screening, Non-Viral Gene Editing: New Frontiers for Neuromuscular Disease
Sowmya Viswanathan, PhD, on Translating Cell Therapies to the Clinic at ISCT 2024
Omar Nadeem, MD, on Initial Efficacy of GPRC5D-CAR in R/R Multiple Myeloma
Omer A. Abdul Hamid, MD, on Improving Gene Therapy’s Effect and Accessibility
George Tachas, PhD, on Tackling DMD Treatment From Multiple Angles
David Suhy, PhD, the cofounder and chief scientific officer of Earli
Deepak L. Bhatt, MD, MPH, MBA, on Incorporating AI into Genetic Research for Cardiovascular Disease
Jeffrey Chamberlain, PhD, on Helping Progress Cell and Gene Therapy Development
© 2024 MJH Life Sciences

All rights reserved.