NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

Summary

This is an early phase 1, open-label, single-center, dose-escalation, pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT006 in homozygous familial hypercholesterolemia (HoFH) patients with LDLR mutations. NGGT006 is an adeno-associated viral (AAV) vector carrying codon-optimized human LDLR gene, driving the expression of LDLR protein with normal function and promoting the clearance of low-density lipoprotein cholesterol (LDL-C).

Timeline

Start

2023-10-29

Primary completion

2024-11-30

Completion

2028-11

Publications

• Zheng T, Gao G, Xu C, Li R, Shao J, Wang X, Han X, Li J, Liu P, Xiong Y, Song L, Wang J, Wen S, Zhuo X, Wang G, Jiang L, Qu G, Chen T, She J, Luo Y, Yuan Z, Wu Y. AAV gene therapy for homozygous familial hypercholesterolemia: a phase 1 trial. Nat Med. 2026 Jun;32(6):2137-2146. doi: 10.1038/s41591-026-04441-3. Epub 2026 Jun 4.

Drugs

Indications