Gene therapy sector needs tech to meet regulatory requirements

Manufacturing rules are a challenge for gene therapy firms as researchers say industry needs better tech to meet regulatory requirements.

Gareth Macdonald

April 19, 2022

2 Min Read
Gene therapy sector needs tech to meet regulatory requirements
Image: Stock Photo Secrets

Manufacturing rules are a major challenge for gene therapy firms according to researchers, who say industry needs better tech to meet regulatory requirements.

The call for better technology came in a new study published in the journal Molecular Therapy earlier this month, in which progress seen in the cell therapy space was compared with the slower growth of the commercial gene therapy market.

“Cell-based therapies have revolutionized the treatment of some blood cancers… Gene therapy, on the other hand, has yet to reach its full potential and therefore remains a top priority in genomic medicine.”


Image: Stock Photo Secrets

Part of the problem, the authors say, is that gene therapy developers focus all their planning and resources in early phase research rather than on the broader product development lifecycle.

“Some companies believe that, after having achieved a robust proof-of-concept, clinical development and downstream interaction with regulatory agencies will be a straightforward process.

“The reality, however, can be quite different. The entire process from concept to clinical grade viral vector under good manufacturing practice standards is a very demanding one.”

To overcome these challenges the gene therapy sector needs better manufacturing technologies, particularly when moving from lab to commercial-scale. Similarly, the say, more accurate measuring technologies are required.

“There is a constant need for technological improvements to overcome challenges in maintaining productivity, scaling up production, or undergoing tests using advanced analytics to meet regulatory requirements.”

Pathfinder challenge

The study comes less than a year after the European Innovation Council – an EC-backed body that supports the commercialization of high-risk, high-impact technologies- also concluded the gene therapy sector needed better manufacturing technologies and analytics.

According to the EIC “With the first gene therapies on the market and dozens more in trials, the race is on to improve the production and manufacturing processes to deploy gene therapies at scale.

“Technological solutions are sought to effectively control challenges in the production of viral vectors at the large scales needed to reach the clinical trial step. Speed is critical, yet excessive speed can put product quality, safety, and efficiency at risk.”

To try and stimulate the development of such solutions the EIC made gene therapy technologies one of its “pathfinder challenges” – funding opportunities intended for researchers working on cutting edge systems.

The EIC also identified the development of more effective vector delivery

You May Also Like