What gene therapy manufacturers can gain from collaboration

In recent years, the biopharmaceutical industry has found itself at a crossroads – being able to offer advanced and truly innovative treatments, such as gene therapies, but facing the complex challenge of creating manufacturing standards for the biotechnology sector from the ground up. Solving this issue will improve industry best practice, increase public confidence and create a more efficient pathway to patients; but it necessitates enhanced collaboration between companies.

Challenges in gene therapy manufacturing

Photo: courtesy of PTC Therapeutics

The biotech industry is facing a bottleneck in product manufacturing, with more than 1,000 gene therapies currently being developed as potential clinical candidates,¹ and not nearly enough capacity in manufacturing facilities to accommodate them. Growth has been rapid since Europe’s first gene therapy approval in 2012,² and the first US Food and Drug Administration (FDA) approval of a gene therapy in 2017.³ Any sector would struggle to keep up with expansion of this speed, but the inherent difficulty of working with and producing biologics adds another layer of complexity.

At PTC Therapeutics, we have opened a gene therapy manufacturing Center of Excellence. We started this process by examining our manufacturing practices, and asked ourselves: where are the bottlenecks and what can we do to speed up the process from start to finish? It became clear that a level of control was required to ensure our products were manufactured to a high quality in a timely manner, especially given the intense regulatory scrutiny of pharmaceutical products and facilities.