Scott Jeffers, PhD, on The Importance of Precise Reproducibility of AAVs

“The purity of the viral vector is super important. It needs to be ultra pure and ready to go to be injected into people, in order to be safe and effective and potent, and to make sure that that it's doing what it's supposed to do. But the purification process needs to also be controlled really well. We need it to be reproducible. We need to do it over and over again. It needs to do the same thing every time we do it. We need to know that it's doing it in the same way, as well as being able to monitor that.”

Manufacturing adeno-associated virus (AAV) vector-based gene therapy is a complex and expensive process that needs to be precisely reproducible and controlled. New platforms and technologies are constantly being pursued to help streamline and improve gene therapy manufacturing. GenSight Biologics recently turned to Verdot Biotechnologies’ FlexiPro system to improve the purification of its gene therapy product, Lumevoq (GS010).

Lumevoq is being evaluated in the phase 3 REFLECT clinical trial (NCT03293524) in participants with Leber hereditary optic neuropath. GenSight recently presented long-term follow-up data from REFLECT that demonstrated sustained improvements in best corrected visual acuity.¹

Scott Jeffers, PhD, chief technology officer, GenSight, also gave a talk about how the company has improved viral purification during AAV manufacturing with the FlexiPro system at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland.² CGTLive® spoke with Jeffers to learn more about the changes in manufacturing. He emphasized the importance of having a system that can yield accurate and precise reproducibility of its products.

REFERENCES

1. GenSight Biologics confirms sustained efficacy and safety of bilateral LUMEVOQ® injections four years after one-time administration. News release. GenSight Biologics. March 20, 2024. https://www.gensight-biologics.com/2024/03/20/gensight-biologics-confirms-sustained-efficacy-and-safety-of-bilateral-lumevoq-injections-four-years-after-one-time-administration/
2. Increased Reproducibility in a Critical Downstream Processing Step Allows for Speed to Commercialization of Gene Therapy Product to Treat Leber's Hereditary Optic Neuropathy (LHON). Presented at: Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #1031