By Dhirendra Tripathi
Investing.com – Intellia Therapeutics (NASDAQ:NTLA) rose more than 60% Monday after a phase-I study of a gene-editing treatment for a disease called transthyretin amyloidosis showed positive results.
Shares of Regeneron (NASDAQ:REGN) Pharmaceuticals, Intellia's partner in the study, gained 3%. Two other companies working on the same gene-editing technology also rallied in premarket trading, with CRISPR Therapeutics soaring 8% and Editas Medicine (NASDAQ:EDIT) rising 16%.
The stock soared because of the wider implications the gene-editing treatment holds and not just for the patients suffering from transthyretin amyloidosis.
The Intellia data showed deep reduction in the disease-causing protein after single infusion of NTLA-2001, an investigational CRISPR therapy for transthyretin amyloidosis.
NTLA-2001 is the first investigational CRISPR therapy candidate to be administered systemically, or through a vein, to edit genes inside the human body. CRISPR is a Nobel Prize-winning gene-editing technology.
Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease that occurs when a person is born with mutations in the TTR gene.
The mutation causes the liver to produce damaged proteins that then build up to cause serious complications in multiple tissues, including the heart, nerves and digestive system.
There are an estimated 50,000 people worldwide living with ATTRv amyloidosis (hereditary ATTR) and between 200,000 and 500,000 people with ATTRwt amyloidosis (wild-type ATTR).