(Reuters) -Intellia Therapeutics has temporarily stopped dosing and patient screening in two late-stage studies of its gene therapy after a participant developed serious liver injury, the company said on Monday, sending its shares down over 44% in premarket trading.
The patient, who received the therapy called Nexiguran ziclumeran (nex-z) in a study for transthyretin amyloidosis with cardiomyopathy (ATTR-CM), showed sharp spikes in liver enzymes and elevated bilirubin, triggering a protocol-defined safety pause, the company said.
The individual has been hospitalized and is under medical care.
ATTR-CM is a rare and deadly heart disease in which faulty transthyretin proteins accumulate in the heart and can cause the organ to fail.
The development marks a setback for the company, which is developing treatments based on the CRISPR technology. Nex-z is being developed as a one-time treatment that works by turning off the gene responsible for producing the transthyretin protein.
Intellia said it is consulting outside experts, weighing risk‑reduction steps and speaking with regulators about a plan to restart enrollment “as soon as appropriate.”
The current treatment landscape for ATTR-CM includes Alnylam Pharmaceuticals’ injectable drug Amvuttra, Pfizer’s blockbuster Vyndaqel and BridgeBio Pharma’s Attruby.
(Reporting by Padmanabhan Ananthan in Bengaluru; Editing by Shailesh Kuber)
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