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
"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