(Bloomberg) -- Intellia Therapeutics Inc. and Regeneron Pharmaceuticals Inc.'s experimental gene-editing therapy cut levels of an abnormal liver protein in interim, early-stage trial results, a step toward showing Crispr technology can provide lasting treatment.
Six patients who received a high dose of the Crispr-based therapy saw faulty protein levels drop an average of 93% for as long as six months, the companies said Monday in a statement. The abnormal protein causes rare transthyretin amyloidosis that damages the heart and nerves, sometimes leading to death.
Companies began pursuing drugmaking with Crispr -- a way to remove or replace defective genes in cells -- years before two gene-editing pioneers won the Nobel Prize in 2020. While other therapies for the liver condition from Alnylam Pharmaceuticals Inc. and Pfizer Inc. require repeated dosing, a Crispr fix has the potential to be permanent.
“Is this going to be life-long?” Intellia Chief Executive Officer John Leonard said in an interview. “Nobody's lived his or her entire life in this study yet, but with each passing month we're more confident that's the way it's going to turn out to be.”
Read More: Crispr, the Tool Giving DNA Editing Promise and Peril
So far, the ongoing trial has looked at the experimental treatment, NTLA-2001, in patients whose nerves were affected by the disease; the companies are expanding the trial to include people suffering heart damage. Later-stage trials will focus first on patients with cardiac involvement, Leonard said.
TTR amyloidosis may affect more than 400,000 people in the world. Only Pfizer's drugs -- Vyndaquel and once-daily Vynadamax -- treat the condition's effects on both the heart and the nerves. Alnylam's Onpattro is currently limited to the nerve indication, while Ionis Pharmaceuticals Inc. and AstraZeneca Plc are developing eplontersen for treatment of both forms of the disease.
In Intellia and Regeneron's Crispr study, reductions in abnormal protein levels ranged as low as 52% in patients who received the lowest dose of 0.1 milligram per kilogram. Among patients who received 0.3 milligrams per kilogram, average levels of the abnormal protein dropped by at least 87% four weeks after infusion. The protein levels remained at low levels for as long as a year for some patients.
Intellia and Regeneron said they'll continue trials using a dose similar to the highest in the study, 1 milligram per kilogram. All four dose levels were generally well tolerated, with the most common side effects including headaches, infusion-related reactions, back pain, rash and nausea, the companies said.
Intellia said the results validate its approach in transthyretin amyloidosis and support work in other diseases.
The study “gives us a platform for where to build additional complexity as we move our other tools forward,” Intellia Chief Scientific Officer Laura Sepp-Lorenzino said in an interview.
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