Fierce Biotech

Intellia races in vivo CRISPR therapy to FDA after phase 3 data paint ‘compelling’ picture

A phase 3 trial of Intellia Therapeutics’ in vivo gene-editing therapy lonvoguran ziclumeran (lonvo-z) has hit its primary ...
Stocktwits on MSN

NTLA stock surges 35% overnight: Retail mood peaks ahead of first-ever phase 3 readout for gene-editing therapy

The Haelo Phase 3 trial marks the first late-stage test of an in-vivo CRISPR gene-editing treatment. ・Earlier mid-stage studies showed a 96% reduction in monthly attack rates, with 97% of patients ...

Intellia Therapeutics Reports Positive Phase 3 Results in Hereditary Angioedema, Marking a Global First for In Vivo Gene Editing

Phase 3 HAELO trial of lonvoguran ziclumeran (lonvo-z) met primary and all key secondary endpoints; favorable safety and tolerability data ...
Healio

Advanced gene editing ‘promising’ for sickle cell disease, but access challenges remain

Two papers published in The New England Journal of Medicine highlight the potential of gene editing for treatment of sickle ...
BioSpace

Intellia heads to FDA after notching first late-stage win for in vivo gene editor

With Phase 3 data in hand, Intellia Therapeutics is seeking approval for its in vivo CRISPR gene editing therapy for ...
BioPharma Dive

Intellia CRISPR drug succeeds in late-stage study against rare swelling disorder

The findings position Intellia to bring to market the first “in vivo” gene editing medicine, though the therapy’s commercial ...
MPR

Gene Editing Candidate Lonvo-z Meets Endpoints in Phase 3 HAE Study

Lonvo-z is an in vivo CRISPR gene editing candidate that is designed to inactivate the kallikrein B1 gene, thereby permanently lowering kallikrein and bradykinin levels.
BioSpace

Gene therapy leaders aim to help more Baby KJs with novel regulatory models

As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” ...

Compact CRISPR system unlocks targeted in-body gene editing, with up to 90% efficiency

A research team has discovered an enhanced CRISPR gene-editing system that could enable targeted delivery inside the human body—a key step toward broader clinical use. Researchers identified a ...

New gene-editing tool allows scientists to cut out cancerous DNA without harming healthy cells

Researchers from Wageningen University & Research and Van Andel Institute used a variant of CRISPR, a gene-editing tool, to ...