“We presented the first clinical data in history supporting precision editing of a disease-causing gene within the body following a single, systemic dose of Crispr/Cas9,” Intellia said in a statement.
“These data open a new era of medicine – one that holds the potential of curing genetic disease.”
Intellia recently traded at $166.26, up 13%. It has more than doubled (up 148%) over the past six months.
The Crispr results “beat our most optimistic expectations,” Truist analyst Joon Lee wrote in a commentary cited by Bloomberg.
The analyst called the second quarter “transformative,” even as Intellia reported a loss of $1.01 a share, widened from 61 cents a year earlier and sharper than the 63-cent loss expected by Capital IQ’s analyst consensus.
As for Intellia’s treatment, “the interim data from our ongoing Phase 1 trial of NTLA-2001 support its potential to halt and even reverse the relentless progression of ATTR amyloidosis with one dose,” said Intellia Chief Executive John Leonard.
“More broadly, they offer proof of concept for our LNP-based platform for systemic in vivo delivery.
“With these results, we believe we are unlocking the treatment of diseases that originate in the liver and introducing a rapid and reproducible development path that accelerates our in vivo portfolio.”
Other gene-editing stocks gained, too. Editas Medicine (EDIT) - Get Free Report, which was upgraded to outperform by Evercore ISI, recently traded at $53.76, up 17%; and Crispr Therapeutics (CRSP) - Get Free Report was at $131.90, up 5.9%