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Intellia Fixed 95-98% T-Cells Inside the Body Using CRISPR Gene Therapy

crispr/cas9 t cell receptor tcr intellia therapeutics

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#1 Engadin

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Posted 07 May 2019 - 09:30 PM





Intellia and its research collaborator, IRCCS Ospedale San Raffaele presented new in vitro data showing that CRISPR/Cas9 editing resulted in over 98% knockout of the endogenous T cell receptor (TCR), while achieving transfer of various Wilms’ Tumor 1 (WT1)-specific TCRs into over 95% of isolated T cells.

Intellia Therapeutics is a leading genome editing company focused on developing 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, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients’ diseased cells.


Intellia scientists advance CRISPR-mediated targeted gene insertion in non-human primates


Achieved normal circulating human Factor IX protein levels in 
non-human primates using targeted gene insertion


High rate and specificity of acute myeloid leukemia cell killing progressed
using genome-edited, Wilms’ Tumor 1-targeting T cells


CAMBRIDGE, Mass., April 29, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc.(NASDAQ:NTLA), will present today new data, including the first demonstration of targeted gene insertion with CRISPR/Cas9 in the liver of non-human primates (NHPs), at the 22ndAnnual Meeting of the American Society of Gene and Cell Therapy (ASGCT), taking place April 29-May 2, 2019, in Washington, D.C. 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.”


Intellia’s First Demonstration of Targeted Gene Insertion in NHPs


In a follow-up to Intellia’s presentation at the 2018 European Society of Gene and Cell Therapy (ESGCT) meeting of the first robust demonstration of CRISPR-mediated insertion of transgenes in the liver of mice using Factor 9 (F9) as a model gene, the company will present at ASGCT an advancement of its modular hybrid delivery system, which combines Intellia’s lipid nanoparticle (LNP) platform with an adeno-associated virus (AAV). F9 is a gene that encodes FIX, a blood-clotting protein that is missing or defective in hemophilia B patients.


In a collaboration between Intellia and Regeneron, researchers delivered F9 DNA via a proprietary bi-directional insertion template to demonstrate targeted gene insertion in NHPs, resulting in circulating human FIX protein levels within the normal range of human FIX protein levels (3-5 μg/mL; source: Amiral et al., Clin. Chem., 1984). Researchers observed circulating human FIX protein levels of ~3-4 μg/mL at day 14 after a single dose in an ongoing study, with levels sustained through 28 days (~3-5 μg/mL).


Today, researchers additionally will share updated results from an ongoing durability study, first reported in October, that the circulating supratherapeutic human FIX protein levels achieved in mice with Intellia’s hybrid LNP-AAV delivery system have remained stable through 10 months of observation. Further, researchers will show that they can regulate FIX protein levels in mice by varying LNP doses, AAV doses or insertion site.


Today’s presentation, titled “CRISPR/Cas9-Mediated Targeted Insertion of Human F9 Achieves Therapeutic Circulating Protein Levels in Mice and Non-Human Primates,” will be made by Hon-Ren Huang, associate director, Vector Biology, Intellia. This presentation will be accessible through the Events and Presentations page of the Investor Relations section of Intellia’s website at www.intelliatx.com



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