VIDEO: Gene therapy requires a delicate balance between efficacy and safety

BySuber S. Huang, MD, MBA

ByMichela Cimberle

Fact checked byChristine Klimanskis, ELS

ROME — In this Healio Video Perspective from the FLORetina-ICOOR meeting, Suber S. Huang, MD, MBA, speaks about adverse events in gene therapy.

One difference between gene therapy and drug-based therapy is that gene therapy is meant to be permanent.

“This is a major leap forward because it is a cure, not a treatment. At the same time, if there are adverse events, potential complications, they cannot be easily reduced or removed,” Huang said.

Estimating the dose has been shown in studies to be critically important to keep the balance between efficacy and safety.

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Sources/Disclosures

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Source: Huang S. Adverse events in gene therapy 2023. Presented at: FLORetina-ICOOR; Nov. 30-Dec. 3, 2023; Rome.

Disclosures: Huang reports being the former chair of the data monitoring committee for Regenxbio gene therapy studies.

FLORetina-ICOOR meeting

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VIDEO: Gene therapy requires a delicate balance between efficacy and safety

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