Intellia seeks FDA approval for first in vivo CRISPR therapy after Phase 3 success
Intellia Therapeutics is pursuing FDA approval for what could become the first in vivo CRISPR gene editing therapy, following a Phase 3 trial showing its treatment for hereditary angioedema reduced attacks by 87% compared to placebo. The company anticipates a potential market launch in 2027. Hereditary angioedema is a rare genetic disorder causing recurrent swelling episodes. If approved, this would mark a milestone for CRISPR-based medicines administered directly inside the body, rather than editing cells outside the body.
Key facts
- Intellia is seeking FDA approval for an in vivo CRISPR gene editing therapy.
- The treatment targets hereditary angioedema.
- Phase 3 trial showed 87% reduction in attacks vs. placebo.
- Potential launch is projected for 2027.
- This would be the first in vivo CRISPR therapy approved.
- Hereditary angioedema is a rare genetic disorder.
- The therapy is administered directly inside the body.
- The trial results cleared the way for regulatory submission.
Entities
Institutions
- Intellia Therapeutics
- FDA
Sources
- Quartz —