BIO
Navigating the ever-evolving gene therapy regulatory landscape
In the last decade, the rapid rise of gene therapies has taken over the world of medicine, presenting a revolutionary way to treat genetic disorders.
These groundbreaking therapies have the potential to change lives and offer hope to patients around the world.
But as with any new medicinal innovation, the path to market is littered with complexities and challenges. Of the current 3,905 gene therapies in development in 2023, only 60 made it to clinical trials by Q31.
To achieve the FDA’s ambitious goal of 10-20 new cell and gene therapy approvals a year by 2025, developers and manufacturers must find ways to overcome the following challenges:
- Regulatory bodies adapting on the go: Traditional regulatory bodies have struggled to keep pace with continuous advancements and expanding knowledge into gene therapies. This means that they are often ‘building the plane as it flies’ when implementing this knowledge into regulations to ensure the safety of these therapies.
- Cautious approaches lead to stringent requirements: Gene therapies are inherently complex and there is limited availability of long-term clinical data. This has meant that regulators have adopted a more cautious stance in their evaluation to safeguard against any unforeseen effects on patient health. This vigilance in scrutinising gene therapies may potentially impede the approval timeline and is something that developers must factor into their processes.
- Unique analytical conditions add further complexity to regulatory requirements: In both clinical trials and commercial gene therapy use, developers and manufacturers must provide comprehensive analytical data and substantiation to regulatory bodies to demonstrate compliance. As a relatively new modality, gene therapies differ from traditional drug development methods, meaning that the regulatory standards remain subject to change as we learn more about them. This therefore makes it more challenging for companies to provide the required analytical data to regulators.
- Complex gene therapy manufacturing requirements lead to difficulty in scaling up: As complex formulations, gene therapies necessitate specialised manufacturing procedures, often introducing viral vectors and helper genes/plasmids into the production cell line, for example. These present challenges when scaling up production, and can often result in bottlenecks that impede progress toward critical milestones and regulatory approval.
In our latest piece with Pharma Focus America, our experts Kai Lipinski, PhD, Chief Scientific Officer, ReciBioPharm, Xiaojun Liu, PhD, Director AAV Process Development, ReciBioPharm and Jing Zhu, Vice President, Nucleic Acid & Viral Vector Technology, ReciBioPharm discuss these challenges further and explore the solutions to overcoming them. The article can be found on page 10 of the latest issue.