Prime Medicine reports up to 80% gene correction in Wilson's Disease; on track for IND-enabling

Prime Medicine, Inc.

GalNAc-LNP delivery of Prime Editors achieved up to 80% precise correction of ATP7B H1069Q mutation in humanized mice.
In NHPs, up to 51% precise editing of liver cells with surrogate H1069Q Prime Editor; no off-target edits detected.
IND-enabling activities for Wilson's Disease program to start in 4Q 2024; IND/CTA filing expected 1H 2026.
GSD1b program data: up to 85% precise liver cell editing in NHPs, durable to 44 weeks, no safety concerns.
Universal liver-targeted LNP platform shows modularity; can be adapted to other rare and non-rare liver indications.

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