Though gene therapies, like small molecule drugs, need to be shown to be safe and effective in order to gain regulatory approval, there are notable differences between these therapeutic modalities.

Photo: Peter Marks, U.S. Food and Drug Administration Director of the Center for Biologics Evaluation and Research
Now, in a new editorial, the U.S. Food and Drug Administration regulator who oversees the division responsible for gene therapies is raising questions about whether the pace of development has been slowed by applying a framework to these genetic medicines that was developed for traditional therapies.
U.S. Food and Drug Administration Director of the Center for Biologics Evaluation and Research Peter Marks, writing in an editorial in the journal Expert Opinion on Biologicial Therapy, argues greater attention needs to be paid to how to translate scientific advances into practical ones that can benefit people to fully realize the potential of gene therapies.
Marks said there are aspects of the framework for the clinical development of small molecule drugs that can be appropriately applied to the development of gene therapies, such as understanding the non-clinical aspects of the product, the use of good manufacturing practices, and the demonstration of safety and efficacy.
But there are other aspects of gene therapies that may be better suited for different models. For instance, Marks noted that gene therapies use a vector backbone to deliver a transgene. “Considering the ‘device-like’ quality of this vector backbone allows one to consider whether, within specific limits, one could reuse information related to the vector backbone to expedite the development of multiple gene therapy products,” he wrote.
One bottleneck for the development of gene therapies today is manufacturing small batches of product. Often gene therapies originate in academic labs or small companies, which have adequate capabilities to produce material for clinical trials. But when these products are transferred to contract manufacturers who produce materials in accordance with good clinical manufacturing practices, it can be expensive and time consuming. He suggests this could be addressed by standardizing processes and reusing well-characterized gene therapy vectors to streamline the movement from one gene therapy to another to address different diseases.
Other points Marks touched on included advances in the use of novel endpoints and clinical trial designs, enhanced communication with regulatory authorities, and harmonization of global regulations, all of which could accelerate the availability of these therapies.
None of these ideas are particularly radical and Marks, who has worked closely with the National Institutes of Health on initiatives like the Platform Vector Gene Therapy Pilot project and the Bespoke Gene Therapy Consortium, has been at the forefront of considering such ideas and moving the conversation forward.
It is nevertheless encouraging to see a regulator at the forefront raising these issues with an eye toward ensuring more people are able to benefit from scientific breakthroughs enabling new, life-saving medicines.

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