gene editing

Stable2 articles2 sourcesDernière mise à jour: 28.04.2026

Sujets associésthalassaemia gene therapy CS-101 base editing ShanghaiTech University beta-thalassaemia haemoglobin tBE

Derniers articles

Science·28.04.2026Résumé IA

Chinese Scientists Develop Gene Therapy CS-101 Curing Five Beta-Thalassaemia Patients

Scientists at ShanghaiTech University have developed CS-101, a base-editing gene therapy that successfully cured five Chinese patients with beta-thalassaemia. The treatment restored haematopoietic function, raising total haemoglobin and fetal haemoglobin levels, freeing patients from lifelong blood transfusions. China has 30 million thalassaemia gene carriers while the global total reaches 350 million.

SCMP Economy

Intellia's CRISPR Treatment Shows 87% Reduction in Hereditary Angioedema Attacks

En développement

Science·27.04.2026Résumé IA

Intellia's CRISPR Treatment Shows 87% Reduction in Hereditary Angioedema Attacks

Intellia Therapeutics' CRISPR-based treatment for hereditary angioedema met its goals in a Phase 3 trial, reducing attacks by 87% compared with placebo. The one-time infusion therapy edits genes directly in the liver and achieved 62% of patients being attack-free six months after treatment. The company has started a rolling FDA application and plans to launch in the first half of next year if approved.

CNBC