Articles by tag: gene-editing

CRISPR-Cas3 treatment of Duchenne muscular dystrophy: deletion of exons 45-55

Japanese geneticists used CRISPR-Cas3 to delete long DNA segments in Duchenne muscular dystrophy. The method is suitable for 60% of patients. Details in the article.

CRISPR Therapy HAE: Phase 3 Data from Intellia at EAACI 2026

Intellia presented the long-awaited results of phase 3 of CRISPR therapy lonvoguran ziclumeran for hereditary angioedema. Reduction of attacks by 87%, 62% of patients without therapy. Learn more.

Intellia CRISPR therapy: phase 3 data on edema — breakthrough

Intellia presented long-awaited phase III data of CRISPR therapy lonvo-z for hereditary angioedema: 87% reduction in attacks, 62% of patients attack-free. Learn about the breakthrough in in vivo genome editing.

CRISPR therapy in vivo: Intellia study 87% efficacy in HAE

Phase 3 HAELO: single CRISPR infusion reduced hereditary angioedema attacks by 87% vs placebo. First in vivo therapy on the verge of registration. Details, insights, forecasts.

AI model Pythia predicts CRISPR results: breakthrough in gene editing

Neural network Pythia from scientists in Zurich and Ghent predicts DNA repair after CRISPR, enabling precise gene insertion into non-dividing cells. Learn how AI is changing gene therapy.

Intellia CRISPR therapy: FDA filing for lonvo-z for HAE

Intellia has filed the world's first application for an in vivo CRISPR therapy, lonvo-z, for hereditary angioedema (HAE). 87% reduction in attacks, 62% attack-free. Risk, market, and forecast analysis. Read.

VERVE-102 gene editing reduces cholesterol by 62% — analysis

Phase 1 results of VERVE-102: sustained LDL-C reduction up to 62% in patients with hypercholesterolemia. Analysis of market implications and forecasts. Read more.

evoCAST: gene insertion without DNA cuts — breakthrough 2026

The new evoCAST tool inserts whole genes without double-strand breaks. Efficiency increased 400-fold. Learn how this will change gene therapy.

Personalized CRISPR therapy saved an infant: in vivo breakthrough

A child with the ultra-rare CPS1 mutation survived thanks to personalized CRISPR therapy in 6 months. Learn about the breakthrough in genetic engineering and N-of-1 platforms.

In vivo gene editing: phase 3 of HAE therapy successful

First ever successful phase 3 in vivo gene editing: lonvo-z therapy reduces HAE attacks by 87%. Learn about the breakthrough and discontinuation of prophylaxis.

Pig kidney transplant to human: 69 genome edits

Surgeons transplanted a genetically modified pig kidney with 69 edits into a patient. The organ worked for 271 days — learn how this addresses the donor crisis.

In vivo gene editing: CRISPR therapy for hereditary angioedema

Single-dose CRISPR therapy NTLA-2002 reduced hereditary angioedema attacks by 87% in phase III. Learn how this will change treatment and pave the way to a cure.

Blood cells created from stem cells in the laboratory

Australian scientists have for the first time obtained hematopoietic stem cells in vitro. The technology promises to replace bone marrow transplantation and genetically correct blood cells. A breakthrough in hematology.