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First disease targeted by Intellia's LNP-based gene editing therapy?
5
Resolution / Starting Odds
Cystic fibrosis • 25%
Sickle cell anemia • 25%
Hemophilia • 25%
Other • 25%
Official press release from Intellia Therapeutics
Story
Intellia Therapeutics Achieves Milestone in LNP-based Gene Editing with First Successful Redosing
Jun 25, 2024, 07:54 PM
Intellia Therapeutics has successfully demonstrated the ability to redose LNP-based gene editing medicine in humans, marking the first demonstration of this kind. This significant milestone in clinical gene editing shows that sequential edits can be made and certain edits can be reversed. In the study, three patients were initially given a subtherapeutic dose of 0.1 mg/kg, followed by a therapeutic dose of 55 mg. The treatment was found to be effective and well-tolerated. This breakthrough in CRISPR/Cas9-LNP could expand the addressable market for CRISPR-based therapies and may influence future programs targeting conditions like cystic fibrosis.
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