As Gene Therapy Becomes a Reality, Spark Therapeutics Looks at Challenges That Still Remain

According to a story from BioPharma Dive, the advent of gene therapy development at Spark Therapeutics has been five years in the making. At this juncture, it has been nearly a year since the company got the first gene therapy for a heritable disorder approved by the US Food and Drug Administration (FDA). In a recent interview, BioPharma Dive sat down with the company’s CEO, Jeff Marrazzo, to discuss the hurdles and challenges that lie ahead in the future.

Marrazzo says that a lot of time has been spent on determining manufacturing best practices for gene therapy. A big challenge has been confirming the quality and integrity of the finished product. He says that in process of developing its first gene therapy that the company has been a part of 41 assays. An assay is essentially a test that serves to identify the ingredient of a product and the quality of those ingredients. Thankfully for Spark it is unlikely that they will have to do such extensive testing for future products going forward.

A lot of information submitted to the FDA explained in minute detail the company’s manufacturing process. The company has developed several manufacturing innovations during the development of Luxturna, the company’s first gene therapy, and going forward will be a test of the reproducibility of the process.

Getting the process started also meant having the right people with the relevant expertise and experience in the room. This meant finding experts who understood the gene therapy concept as well as scientists who were familiar with more conventional drugs such as enzymes and monoclonal antibodies that are commonly manufactured at massive scales. These scientists are more familiar with the operational knowledge necessary to get mass production of complex medical products going effectively.

Another challenge was working with the FDA to come up with useful testing standards, such as endpoints, for gene therapy, particularly for indications that had no prior available therapies. Luxturna for example, which is intended to treat Leber’s congenital amaurosis, was developed using entirely novel endpoints. This required close collaboration with the agency.

While there is still more that needs to be “ironed out” in regards to manufacturing gene therapies, the work of Spark Therapeutics has been significant in bringing these remarkable therapies to reality.


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