Key points from article :
A CRISPR/Cas9 drug by Regeneron and Intellia showed impressive results in patients with ATTR amyloidosis.
In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body.
Phase 1 trial looked at patients who had polyneuropathy as a result of the disease.
A dose of NTLA-2001 cut protein levels in the blood by 87% in three patients treated with a 0.3mg/kg dose.
In three patients treated at 0.1mg/kg, levels fell by 52%.
An encouraging safety profile, with no serious adverse events in the first six patients by day 28.
“NTLA-2001 is the first CRISPR/Cas9-based candidate to be administered systemically, via IV infusion, for precision editing of a gene in a target tissue in humans,” - Intellia.
Will move onto the phase 2 later this year, and also start testing in cardiomyopathy.
“...new possibilities in treating and potentially curing life-threatening and historically difficult-to-address diseases,” - George Yancopoulos, Regeneron’s chief medical officer.
