By Kamal Choudhury Jan 27 (Reuters) – Intellia Therapeutics said on Tuesday that the U.S. drug regulator has lifted a clinical hold on one of its late-stage gene therapy trials, allowing the company to resume testing its experimental drug for a rare nerve disease. Shares of the drug developer rose 10% in morning trading. The […]
Health
Intellia gets FDA nod to resume one gene therapy trial after safety pause
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By Kamal Choudhury
Jan 27 (Reuters) – Intellia Therapeutics said on Tuesday that the U.S. drug regulator has lifted a clinical hold on one of its late-stage gene therapy trials, allowing the company to resume testing its experimental drug for a rare nerve disease.
Shares of the drug developer rose 10% in morning trading.
The Food and Drug Administration removed the hold on a study testing Intellia’s CRISPR-based therapy, nexiguran ziclumeran, in patients with hereditary transthyretin amyloidosis with polyneuropathy, or ATTRv-PN, a condition in which a misshapen protein builds up in nerves and causes progressive damage. The company said it plans to restart enrolling patients at the soonest.
The regulator had paused two related studies in October after a patient developed severe liver‑related side effects.
Jones Trading analyst Debanjana Chatterjee said the regulator’s decision “removes one element of uncertainty and indicates that constructive FDA engagement is ongoing.”
A separate trial testing the same therapy in patients whose hearts are affected by the disease, known as cardiomyopathy (ATTR‑CM) remains on hold. The patient who experienced the liver complications was enrolled in that heart disease study and later died in early November.
Intellia said it is still working with the FDA on that program and would provide an update once a path forward is agreed upon.
Chatterjee noted that clarity on an FDA-acceptable mitigation plan for the heart disease trial “will be central to how the story evolves from here,” given the significantly larger commercial opportunity in that patient population.
Current treatments for the heart‑related form of the disease include Alnylam Pharmaceuticals’ injectable drug Amvuttra, Pfizer’s blockbuster Vyndaqel and BridgeBio Pharma’s Attruby.
Intellia has expanded the nerve disease trial to enroll about 60 patients, up from 50, and will compare a one‑time infusion of the therapy against a placebo.
(Reporting by Kamal Choudhury in Bengaluru; Editing by Shailesh Kuber)