U.K.’s University of Sheffield Establishes New Gene Therapy Innovation Center

Rare Daily Staff

The United Kingdom’s University of Sheffield unveiled plans for the Sheffield Gene Therapy Innovation and Manufacturing Center, one of three cutting-edge hubs in the United Kingdom dedicated to advancing the clinical development of new genetic treatments.

The Sheffield Hub, which is the first in the north of England, is part of $24.7 million (£18 million) network created by LifeArc and the Medical Research Council, with support from the Biotechnology and Biological Sciences Research Council. Additional funding came from The Law Family Charitable Foundation and University of Sheffield alumni.

Sheffield has emerged as one of the leading players in cell and gene therapy and this national network of partners, facilities, and training programs will allow it to keep pace with translational discoveries for new and potentially life changing treatments.

The university said while the United Kingdom has a world-class genetics research base, to date, academics have found it difficult to get access to the clinical materials, facilities, and expertise required to progress gene therapy research into clinical trials.

The Gene Therapy Innovation and Manufacturing Center is planned for a site on the University of Sheffield’s Innovation District close to existing translational research facilities and will contribute to an ongoing program of regional investment and regeneration. The Center will manufacture commonly used vectors including both lentivirus and adeno-associated virus that are needed for genetic therapy trials, while positioning the United Kingdom for significant bioprocessing innovation work with the potential to radically increase yields and reduce productivity barriers in future years.

The hub network will also design and share commercially ready platforms, using common cell-lines, plasmids, and reagents to reduce costs, facilitate simplified licensing agreements, and streamline regulatory reviews. A key aim is to smooth the transition between small-scale supply for early clinical trials through to larger-scale manufacture for patient trials, and beyond.

The state-of-the-art center will bring together academic institutions, NHS trusts, non-profit and industry partners across the north of England, Midlands and Wales enabling academic-led clinical trials of novel gene therapies. The center will deliver essential translational and regulatory support alongside extensive training and skills programs to enable upskilling and address shortage of skills in Good Manufacturing Practice manufacturing.

“The Gene Therapy Innovation and Manufacturing Centre will tackle major challenges in gene therapy development for some of the most devastating diseases,” said Mimoun Azzouz, director of the Gene Therapy Innovation and Manufacturing Center and chair of translational neuroscience at the University of Sheffield. “Gene therapies are pioneering medical advances that have the potential to offer much-needed, novel, effective treatments for many rare and incurable diseases that cannot be treated by conventional drug compounds. This is a momentous milestone for revolutionary medical advances not only for Sheffield and South Yorkshire, but also for the U.K.”

Photo: Artist impression of the new Gene Therapy Innovation Manufacturing Centre