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YOLT-101 Phase I interim data show 52% LDL reduction in investigator-led trial
Interim Phase I results from an investigator-led trial at Renji Hospital and Shanghai Jiao Tong University demonstrated that YOLT-101 achieved 50% LDL-C reduction and 76% PCSK9 reduction in the highest-dose cohort. The data supported regulatory approvals in both China and the United States.
- Phase I trial enrolled patients with heterozygous familial hypercholesterolemia.
- Highest-dose patients showed 50.4% LDL reduction and 75.8% PCSK9 reduction at 4 months.
- Safety profile favorable with mild infusion-related reactions and temporary liver enzyme elevations.
- First clinical validation of in vivo base editing for cardiovascular disease.