VIDEO: Novel hemophilia B gene therapy approach shows promise

BySavannah Demko

Fact checked byMindy Valcarcel, MS

Lindsey A. George, MD, discussed “exciting” preclinical data on gene editing for hemophilia B, which was presented in a session at ASH Annual Meeting and Exposition.

“Their data are quite compelling. They already have an ongoing lead-in trial,” George, assistant professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania, and director of clinical in vivo gene therapy and attending hematologist at the Children’s Hospital of Philadelphia, told Healio in a video interview.

“We might expect in the next year to have, at least, very, very preliminary data from their initiation of treated patients,” she continued. “I think this is a really exciting next-generation approach to hemophilia B gene therapy.”

Reference:

Sabin L, et al. Novel approaches for gene-based therapies: Targeted gene insertion of factor 9 as a potential durable treatment for hemophilia B. Presented at: ASH Annual Meeting and Exposition; Dec. 9-12, 2023; San Diego.

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Sources/Disclosures

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Source:

Healio Interviews

Disclosures: George reports consulting for Regeneron and Spark Therapeutics; receiving research funding/royalties from AskBio; and a role on the Board of Directors or advisory committee for Regeneron. She also has a leadership/fiduciary role at STRM.BIO.

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VIDEO: Novel hemophilia B gene therapy approach shows promise

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