In vivo

Consecutive Genome Editing in Non Human Primate Achieves Durable Production of Human Alpha-1 Antitrypsin and Reduction of the Native Protein
29th Annual Congress of the European Society of Gene & Cell Therapy
Oct 20, 2021

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
New England Journal of Medicine
Jun 26, 2021

In vivo CRISPR/Cas9 Editing of the TTR Gene by NTLA 2001 in Patients with Transthyretin Amyloidosis
2021 Peripheral Nerve Society Meeting
Jun 26, 2021

CRISPR/Cas9-Mediated Targeted Gene Insertion of SERPINA1 to Treat Alpha-1 Antitrypsin Deficiency
24th Annual Meeting of the American Society of Gene and Cell Therapy
May 11, 2021

Development of In Vivo, Systemic CRISPR-Based Therapeutics
24th Annual Meeting of the American Society of Gene and Cell Therapy
May 11, 2021

Avoiding Unintended Genome Editing for CRISPR/Cas9 Therapeutics
24th Annual Meeting of the American Society of Gene and Cell Therapy
May 10, 2021

Recording: In Vivo Genome Editing of Hematopoietic Stem and Progenitor Cells
Keystone eSymposium: Precision Engineering of the Genome, Epigenome and Transcriptome
Mar 10, 2021

In Vivo Genome Editing of Hematopoietic Stem and Progenitor Cells
Keystone eSymposium: Precision Engineering of the Genome, Epigenome and Transcriptome
Mar 10, 2021

NTLA-2002: CRISPR/Cas9-Mediated Gene Knockout of KLKB1 for Hereditary Angioedema
American Academy of Allergy, Asthma & Immunology Annual Meeting
Feb 27, 2021

CRISPR/Cas9 Mediated Targeted Gene Insertion of SERPINA1 to Treat Alpha 1 Antitrypsin Deficiency (AATD)
Alpha-1 Foundation’s 20th Gordon L. Snider Critical Issues Workshop: The Promise of Gene-Based Interventions of Alpha-1 Antitrypsin Deficiency
Dec 12, 2020

A Modular CRISPR/Cas9 Genome Editing Platform for Durable Therapeutic Knockout and Targeted Gene Insertion Applications
16th Annual Meeting of the Oligonucleotide Therapeutics Society
Sep 29, 2020

CRISPR/Cas9 Mediated Liver Gene Knockout of KLKB1 to Treat Hereditary Angioedema
23rd Annual Meeting of the American Society of Gene and Cell Therapy
May 15, 2020

Validation of CRISPR / Cas9 Off-Target Discovery Profiles from In Silico Prediction, Cell-Based and Biochemical-Based Assays with Targeted Off-Target Sequencing
23rd Annual Meeting of the American Society of Gene and Cell Therapy
May 12, 2020

In Vivo Delivery of CRISPR/Cas9 to the Liver Using Lipid Nanoparticles Enables Gene Knockout Across Multiple Targets in Rodent and Non-Human Primates
Keystone Symposia: Engineering the Genome
Feb 9, 2020

In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression
European Society of Gene and Cell Therapy 27th Annual Meeting
Oct 25, 2019

CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria
European Society of Gene and Cell Therapy 27th Annual Meeting
Oct 24, 2019

In Silico, Biochemical and Cell-based Integrative Genomics Identifies Precise CRISPR / Cas9 Targets for Human Therapeutics
European Society of Gene and Cell Therapy 27th Annual Meeting
Oct 23, 2019

Using Lipid Nanoparticles to Efficiently Deliver CRISPR/Cas9 for Genome Editing
The 15th Annual Meeting of the Oligonucleotide Therapeutics Society
Oct 14, 2019

Development of NTLA-2001, a CRISPR/Cas9 Genome Editing Therapeutic for the Treatment of ATTR
2nd European Congress for ATTR Amyloidosis
Sep 1, 2019

CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria
American Society of Gene and Cell Therapy 22nd Annual Meeting
May 2, 2019

CRISPR/Cas9-Mediated Targeted Insertion of Human F9 Achieves Therapeutic Circulating Protein Levels in Mice and Non-Human Primates
American Society of Gene and Cell Therapy 22nd Annual Meeting
Apr 29, 2019

Supra-therapeutic levels of transgene expression achieved in vivo by CRISPR/Cas9 mediated targeted gene insertion
26th Annual Congress of the European Society of Gene and Cell Therapy
Oct 18, 2018

Delivering on the therapeutic potential of CRISPR/Cas9: Development of an LNP-mediated genome editing therapeutic for the treatment of ATTR
26th Annual Congress of the European Society of Gene and Cell Therapy
Oct 18, 2018

Development of NTLA-1001: First-in-Class, LNP-CRISPR/Cas9 Mediated Genome Editing Therapeutic for the Treatment of ATTR
Genome Engineering: The CRISPR-Cas Revolution at Cold Spring Harbor Laboratory Presentation
Aug 23, 2018

Rescue of Amyloid Deposition Phenotype After Single-Treatment CRISPR/Cas9 Gene Editing in a Humanized Mouse Model of ATTR
American Society of Gene and Cell Therapy 21st Annual Meeting
May 16, 2018

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
Cell Reports
Feb 27, 2018

 

Engineered Cell Therapy

Clinical-Scale Production and Characterization of NTLA-5001 – a Novel Approach to Manufacturing CRISPR/Cas9 Engineered T Cell Therapies
63rd ASH Annual Meeting & Expositions
Dec 13, 2021

A Novel Strategy for Off-The-Shelf T Cell Therapy which Evades Host T Cell and NK Cell Rejection
63rd ASH Annual Meeting & Expositions
Dec 11, 2021

A Novel Strategy for Off the shelf T Cell Therapies Evading Host T Cell and NK Cell Rejection
29th Annual Congress of the European Society of Gene & Cell Therapy
Oct 20, 2021

Lipid Nanoparticles (LNPs) as a Superior CRISPR/Cas9 Delivery Modality for Highly Efficient Multiplex Gene Editing of T Cells for Adoptive Cell Therapy
29th Annual Congress of the European Society of Gene & Cell Therapy
Oct 19, 2021

Special Edition: Expanding Intellia’s Toolbox with Base Editing
Cold Spring Harbor Laboratory Virtual Meeting on Nucleic Acid Therapies
Mar 25, 2021

NTLA 5001, a T Cell Product Candidate with CRISPR Based Targeted Insertion of a High Avidity, Natural, WT1 Specific TCR, Shows Efficacy in In Vivo Models of AML and ALL
62nd American Society of Hematology (ASH) Annual Meeting
Dec 5, 2020

In Vivo Model Development for Genome-Edited T Cell Therapeutics
23rd Annual Meeting of the American Society of Gene and Cell Therapy
May 14, 2020

Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering
23rd Annual Meeting of the American Society of Gene and Cell Therapy
May 12, 2020

Developing Next-Generation Engineered TCR-T Cells with CRISPR
Keystone Symposia: Engineering the Genome
Feb 10, 2020

Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In-Locus Insertion Combined with Endogenous TCR Knockout
European Society of Gene and Cell Therapy 27th Annual Meeting
Oct 24, 2019

 

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