In vivo CRISPR therapy nears approval after striking Phase 3 results in HAE
TL;DR
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.
Topics:businessbiotech#biotech#crispr#fda-rolling-submission#hereditary-angioedema#in-vivo-gene-editing#lonvo-z
- Intellia says CRISPR-based treatment for rare disease reduced swelling attacks in pivotal trial statnews.com
- Intellia Therapeutics says its Crispr-based treatment succeeds in pivotal trial CNBC
- Intellia Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for Lonvoguran Ziclumeran (lonvo-z) as a One-Time Treatment for Hereditary Angioedema Intellia Therapeutics
- Intellia races in vivo CRISPR therapy to FDA after phase 3 data paint ‘compelling’ picture Fierce Biotech
- Intellia CRISPR drug succeeds in late-stage study against rare swelling disorder BioPharma Dive
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