Intellia Therapeutics (NTLA) shares surged on Monday after the biotechnology company along with Regeneron Pharmaceuticals (REGN) reported positive results from its first clinical trial using a new, Nobel Prize-winning Crispr technology to treat Transthyretin Amyloidosis.
Intellia shares were up nearly 50% in premarket trading after the company and Regeneron reported positive interim data from an ongoing phase 1 clinical study of genome editing candidate NTLA-2001, a single-dose treatment for Transthyretin Amyloidosis that utilizes so-called Crispr technology.
The gene-sequencing technology inactivates the transthyretin gene in liver cells to prevent the production of misfolded transthyretin protein, which accumulates in tissues throughout the body and causes the debilitating and often fatal complications of the genetic disease.
The study of six patients, which received the therapy via intravenous infusion, showed significant reductions in levels of a harmful liver protein associated with the genetic disease, the companies said.
Intellia expects to begin a single-dose expansion cohort in part 2 of the phase 1 trial later in 2021. At last check, its shares were up 48.6% at $132. The shares ended the trading day Friday up 2.29% at $88.23.