Intellia Therapeutics Presents New In Vivo and Engineered Cell Therapy Data at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy
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
“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
Intellia’s First Demonstration of Targeted Gene Insertion in NHPs
In a follow-up to Intellia’s presentation at the 2018
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
Data Update from Intellia’s Acute Myeloid Leukemia Program
Intellia and its research collaborator, IRCCS Ospedale San Raffaele, will provide an update on the company’s lead engineered cell therapy program in AML. Researchers will present new in vitro data showing that CRISPR/Cas9 editing resulted in >98% knockout of the endogenous T cell receptor (TCR), while achieving transfer of various Wilms’ Tumor 1 (WT1)-specific TCRs into >95% of isolated T cells. Researchers generated and tested a library of TCRs with different epitope specificities and human leukocyte antigen (HLA) restrictions, building on the data reported at the 2018 ESGCT meeting. Several lead TCRs restricted to the HLA-A*02:01 allele, a frequently expressed allele in the western hemisphere, show the desired T cell characteristics, including high affinity, epitope specificity and killing of a panel of primary leukemic blast cells that expressed the WT1 antigen.
Intellia and OSR are collaborating to develop best-in-class CRISPR-edited T cells directed to a specific epitope of WT1, a tumor-associated antigen overexpressed across a wide range of different tumor types and a known driver of leukocyte blasts in hematological cancers. Intellia’s first cell therapy tumor target is WT1 for the treatment of AML and other potential hematological malignancies, as well as for solid tumors.
Today’s presentation, titled “Exploiting Clonal Tracking of WT1-Specific T Cells to Generate a
Additional In Vivo Data to be Presented at the 2019 ASGCT Meeting
Intellia’s third oral presentation at the 2019 ASGCT Meeting will take place later this week, on
The data shows the continued progression of Intellia’s modular platform capability using CRISPR to knock out liver gene targets. The data being presented includes results from an ongoing collaboration with researchers at the
This press release contains "forward-looking statements" of Intellia within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding our ability to advance and expand the CRISPR/Cas9 technology to develop human therapeutic products, as well as our CRISPR/Cas9 intellectual property portfolio; our ability to achieve stable or effective genome editing; our ability to perform genomic editing, such as knock-out and insertion, to treat disease by modulating, replacing or correcting genetic function; our ability to effectively administer one dose or multiple doses of our CRISPR/Cas9-based product candidates; the potential timing and advancement of our preclinical studies, including continuing non-human primate studies for our Transthyretin Amyloidosis (“ATTR”) program and other studies for our other programs (such as, alpha-1 antitrypsin deficiency (“AATD”), primary hyperoxaluria type 1 (“PH1”), and acute myeloid leukemia (“AML”)), and clinical trials; our ability to replicate results achieved in our preclinical studies in any future studies, including human clinical trials; the potential development of ex vivo cell therapeutics of all types using CRISPR/Cas9 technology, including using Wilms’ Tumor 1 (“WT1”) to develop cell therapies for the treatment of AML; our ability to commence IND-enabling studies of a lead TTR development candidate in 2019 and subsequently submitting an Investigational New Drug application; our intent to present additional data for our liver programs, organs beyond the liver, additional insertion/repair data, and preclinical data in support of our in vivo programs, including TTR and PH1, as well as our ex vivo programs on immuno-oncology, including AML, during 2019; our ability to nominate a development candidate for an ex vivo program, as well as a second in vivo indication, in 2019; the intellectual property position and strategy of Intellia, Intellia’s licensors and other third parties from which Intellia derives rights; actions by government agencies; the impact of our collaborations on our development programs; the potential timing of regulatory filings regarding our development programs; the potential commercialization opportunities, including value and market, for our product candidates; our expectations regarding our uses of capital, expenses, future accumulated deficit and other 2019 financial results; and our ability to fund operations into 2021.
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain our intellectual property position; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will be predictive of future results in connection with future studies; and the risk that Intellia’s collaborations with
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Source: Intellia Therapeutics, Inc.