Copyright The Boston Globe
Intellia Therapeutics Inc. shares dropped after the company said a patient died after suffering liver damage in a clinical trial for the company’s gene-editing treatment. The patient, who was in his early 80s, died Wednesday, the company said. He was hospitalized Oct. 24 after after reporting abdominal pain and showing elevated liver enzymes. Intellia shares sank 23 percent in Friday trading. They’d risen almost 6 percent on the year through Thursday’s close. After the company said that the patient was hospitalized last month, its stock fell the most on record. The Cambridge-based biotech company is studying the use of Crispr, the Nobel Prize-winning gene-editing technology, to treat heart and nerve conditions. The company paused clinical trials last month after the patient was hospitalized. The patient death could make it more difficult for the company to persuade US regulators to allow the trials to resume, said William Blair analyst Myles Minter. Advertisement “We need to see a mechanistic explanation from Intellia and a clear regulatory path forward to be more constructive on the stock,” Minter said in a note. Intellia is still looking into the cause of the patient’s liver damage. The patient, who was in a study to treat a heart condition, had “complicating comorbidities” and the company is considering measures to limit the risks. Liver problems are a known side effect of gene therapy because of the way in which the medicine is delivered. The safety issue gained widespread attention earlier this year after two teenagers died from acute liver failure after receiving a gene therapy from Sarepta Therapeutics Inc. For the heart condition, known as ATTR-CM, Intellia is trying to stand out from competitors with already approved drugs, including Pfizer Inc., Alnylam Pharmaceuticals Inc. and BridgeBio Pharma Inc., by creating a one-time treatment. Advertisement
