AAV gene therapy for homozygous familial hypercholesterolemia: a phase 1 trial - PubMed
4 hours ago
- #familial hypercholesterolemia
- #phase 1 trial
- #gene therapy
- AAV gene therapy NGGT006 was developed to treat homozygous familial hypercholesterolemia (HoFH) by expressing LDLR in the liver.
- In preclinical studies, NGGT006 lowered LDL-C levels and reduced aortic plaque size in animal models.
- A phase 1 trial involved three HoFH patients receiving escalating doses, with no severe vector-related adverse events reported.
- All patients experienced temporary liver enzyme elevations, managed with sirolimus and methylprednisolone therapy.
- The highest-dose patient achieved a sustained reduction in LDL-C from 11 mmol/L to below 1.8 mmol/L within 3 weeks post-treatment.