Intellia Therapeutics Demonstrates Sustained and Durable Genome Editing with CRISPR/Cas9 in One-Year Animal Study
September 26 2017 - 7:30AM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on the development of potentially curative
therapeutics using CRISPR/Cas9 technology, today presented the full
12-month data of its completed long-term mouse study, demonstrating
robust and durable in vivo genome editing post single-dose,
intravenous administration using its proprietary lipid nanoparticle
(LNP) delivery system.
The data presented at the 13th Annual Meeting of the
Oligonucleotide Therapeutics Society in Bordeaux, France,
demonstrated robust editing and durability:
- Achieved and maintained approximately 97 percent reduction in
serum TTR protein levels through one-year, following a single
dose
- Attained approximately seventy percent editing at the target
DNA site in the liver through one year
- The transient nature of LNP delivery was confirmed with 99
percent clearance of mRNA within 10 hours and of sgRNA within 72
hours in the liver
- The genome editing treatment was well tolerated with no adverse
events during the course of the 12-month study
“These results show the long-term persistence of edited TTR
genes in the mouse liver, indicating the potential for sustained
efficacy and tolerability of our CRISPR/Cas9 platform as we move to
higher species,” said David Morrissey, Ph.D., senior vice
president, Platform and Delivery Technology. “These data validate
Intellia’s approach as we remain on track with our non-human
primate studies, which will permit us to designate our development
candidate and perform IND-enabling activities in 2018 for the
development of potential therapies in humans.”
About Intellia TherapeuticsIntellia
Therapeutics is a leading genome editing company focused on the
development of proprietary, potentially curative therapeutics using
the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9
technology has the potential to transform medicine by permanently
editing disease-associated genes in the human body with a single
treatment course. Our combination of deep scientific, technical and
clinical development experience, along with our leading
intellectual property portfolio, puts us in a unique position to
unlock broad therapeutic applications of the CRISPR/Cas9 technology
and create a new class of therapeutic products. Learn more about
Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; Follow us
on Twitter @intelliatweets.
Intellia Contacts:
Media
Contact:Jennifer Mound Smoter Senior Vice President,
External Affairs & Communications
+1 857-706-1071
jenn.smoter@intelliatx.com |
Investor
Contact: Lindsey Trickett Vice President, Investor
Relations +1 857-285-6211 lindsey.trickett@intelliatx.com |
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