Fierce Biotech October 18, 2023
Intellia Therapeutics has received the green light to study its in vivo CRISPR-based gene editing therapy in the U.S., positioning it to start a phase 3 trial of the Regeneron-partnered candidate by the end of the year.
Massachusetts-based Intellia worked with sites in Europe and New Zealand to study the drug candidate, NTLA-2001, in a phase 1 trial. Having shown the candidate may safely reduce levels of misfolded TTR, a protein that drives the rare, life-threatening disease ATTR amyloidosis, the biotech has secured the FDA’s blessing to include U.S. sites in an upcoming phase 3 trial.
Intellia is aiming to start the global phase 3 trial by the end of the year, keeping it on track to achieve the timeline...
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