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In Vivo Gene Editing

All articles tagged with #in vivo gene editing

Cellectis to cut 70% of staff as it abandons CAR-T for in vivo gene editing
biotech22 minutes ago

Cellectis to cut 70% of staff as it abandons CAR-T for in vivo gene editing

Paris-based Cellectis is eliminating up to 70% of its workforce as it pivots from allogeneic CAR-T cell therapies to in vivo gene editing. The company scrapped its clinical-stage lasme-cel and eti-cel programs in September 2026, citing a changed commercial landscape. It is now focusing on two preclinical candidates: HEAL-101 for severe hypertriglyceridemia and HEAL-201 for severe hypercholesterolemia. Third-quarter financial results show a net loss of $101.1 million for the nine-month period, with cash reserves expected to fund operations into late 2028.

In vivo CRISPR therapy nears approval after striking Phase 3 results in HAE
biotech6 months ago

In vivo CRISPR therapy nears approval after striking Phase 3 results in HAE

Intellia Therapeutics’ Phase 3 study of lonvo-z, its in vivo CRISPR-based treatment for hereditary angioedema, showed an 87% reduction in attack rates versus placebo after a single dose, with over 60% of patients attack-free, prompting an FDA rolling submission and positioning lonvo-z as the second approved CRISPR medicine and the first in vivo gene-editing therapy.