Intellia Therapeutics (NTLA) - Get Intellia Therapeutics, Inc. Report shares surged on Monday after the biotechnology company along with Regeneron Pharmaceuticals (REGN) - Get Regeneron Pharmaceuticals, Inc. Report reported positive results from its first clinical trial using a new, Nobel Prize-winning Crispr technology to treat Transthyretin Amyloidosis.
Intellia shares were up more than 45% 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.
Transthyretin Amyloidosis, also known as ATTR and Familial Transthyretin Amyloidosis, is a rare condition characterized by abnormal build-up of a protein called amyloid in the body's organs and tissues.
"These are the first ever clinical data suggesting that we can precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of Crispr,” said Intellia CEO John Leonard, adding that the results “support our belief that NTLA-2001 has the potential to halt and reverse the devastating complications of transthyretin amyloidosis with a single dose.”
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 45.62% at $129.78. The shares ended the trading day Friday up 2.29% at $88.23.
Regeneron in April reported positive progress in a Phase 3 trial of its Regen-COV antibody cocktail, which it is testing to prevent COVID-19.
Regeneron shares were up 0.73% at $554.56.