gene-editing therapy
Coverage of gene-editing therapy in the Nexus archive.
- ‘A leap of faith’: Nashville teen makes history with gene-editing treatment for sickle cell disease
A 13-year-old Nashville boy, Rickey Buggs, underwent a 42-day gene-editing treatment at TriStar Centennial Children’s Hospital under Dr. Haydar Frangoul, potentially offering a cure for his sickle cell disease. The therapy, described as transformative, marks a significant advancement for patients without family matches for traditional treatments.
- Bad cholesterol slashed 62% by single dose of gene-editing drug in small trial
An experimental gene-editing drug, VERVE-102, showed a 62% reduction in LDL cholesterol in a Phase I trial with 35 patients, with no serious adverse events reported. The treatment demonstrated sustained effectiveness over 18 months, though a temporary mild liver enzyme increase was observed.
- STAT+: Eli Lilly says Verve’s gene editor lowers cholesterol levels in early study
Eli Lilly reported a 62% reduction in LDL cholesterol levels in a Phase 1 trial using its gene-editing therapy, VERV-102, acquired from Verve Therapeutics. The therapy showed no serious adverse events, marking a significant safety improvement over Verve's previous shelved candidate. The treatment is positioned as a potential one-time solution for preventing heart disease.