Intellia Therapeutics will showcase the first demonstration of targeted gene insertion with CRISPR/Cas9 in the liver of non-human primates during its presentation at American Society of Gene and Cell Therapy.
Intellia Therapeutics (NASDAQ:NTLA) will showcase the first demonstration of targeted gene insertion with CRISPR/Cas9 in the liver of non-human primates during its presentation at American Society of Gene and Cell Therapy (ASGCT).
As quoted in the press release:
Researchers also will present today new in vitro data from Intellia’s lead engineered cell therapy program in acute myeloid leukemia (AML). Later this week at the 2019 ASGCT meeting, Intellia will present new data from its primary hyperoxaluria (PH1) program.
“The data we are presenting at ASGCT reflects our rapid progress with Intellia’s modular CRISPR/Cas9 platform across a variety of in vivo and engineered cell therapeutic applications,” said Intellia President and Chief Executive Officer John Leonard, M.D. “Today’s presentation of our most recent targeted gene insertion data depicts Intellia’s second successful demonstration of CRISPR-mediated gene editing in non-human primates, both in collaboration with Regeneron Pharmaceuticals, Inc. The first was through gene knockout in our transthyretin amyloidosis program and, now, we have used our targeted insertion approach with Factor 9 as a model gene. We also continue to make strides with our collaborators at IRCCS Ospedale San Raffaele toward developing engineered cell therapies for a variety of intractable cancers, such as acute myeloid leukemia.”