BioPharma Dive June 26, 2021
Study results offer the first clinical evidence that CRISPR gene editing inside the body can be safe and effective.
An infusion of an experimental CRISPR gene editing medicine has shown early promise as a treatment for a rare inherited condition, an encouraging finding that marks the latest, significant step forward for a technology awarded a Nobel prize last year.
Treatment with the medicine, which was developed by Intellia Therapeutics and Regeneron Pharmaceuticals, dramatically lowered levels of a misshapen protein that causes the disease transthyretin amyloidosis. Side effects in the six patients enrolled in Intellia’s study were few and mild.
The results, which were published Saturday in The New England Journal of Medicine, are the first clinical evidence that CRISPR gene...
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