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Stock Watch: Intellia Edits Out The Competition

Significant Gene Editing Efficacy Gives Edge Over Traditional Gene Therapy

 
• By Andy Smith

Exciting early results in gene editing contrast with issues around traditional gene therapies, including non-responders, safety and questions over the duration of efficacy.

Stock Watch Image, Andy Smith
ANDY SMITH OFFERS A LIFE SCIENCE INVESTOR'S PERSPECTIVE ON BIOPHARMA BUSINESS

At the recent European Academy of Allergy and Clinical Immunology Congress,Intellia Therapeutics, Inc. announced an update of the first part of the Phase I/II study of its CRISPR/Cas9 gene editing product NTLA-2002 for the prevention of inflammatory attacks in hereditary angioedema (HAE) patients. (Also see "Stock Watch: Not So Fast, Gene Editors" - Scrip, 5 July, 2021.)

The aim of NTLA-2002 is to inactivate the kallikrein B1 gene because C1 esterase inhibitor deficiency in HAE patients leaves the kallikrein protein unchecked, resulting in excessive inflammation. At worst,...

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