Transthyretin (ATTR) Amyloidosis
Enabling the Development of Serum [TTR] as a Biomarker for Treatment of ATTR Amyloidosis4th International ATTR Amyloidosis Meeting for Patients and Doctors — Nov 2, 2023
First-in-Human in vivo CRISPR/Cas9 Editing of the TTR Gene by NTLA-2001 in Patients with Transthyretin (ATTR) Amyloidosis with CardiomyopathyAmerican Heart Association Scientific Sessions — Nov 5, 2022
Less may be more for TTR in ATTR: model-predicted outcomes of TTR reductionsQuantitative Systems Pharmacology (QSP) Summit 2022 — Sep 15, 2022
In vivo CRISPR/Cas9 Editing of the TTR gene with NTLA-2001 in Patients with Transthyretin Amyloidosis Dose Selection ConsiderationsEuropean Association for the Study of the Liver International Liver Congress 2022 — Jun 24, 2022
NTLA-2001 for ATTR Amyloidosis: Interim Clinical Results from Ongoing Phase 1 TrialIntellia Therapeutics Sponsored Event — Feb 28, 2022
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin AmyloidosisNew England Journal of Medicine — Jun 26, 2021
In vivo CRISPR/Cas9 Editing of the TTR Gene by NTLA-2001 in Patients with Transthyretin Amyloidosis2021 Peripheral Nerve Society Meeting — Jun 26, 2021
Development of NTLA-2001, a CRISPR/Cas9 Genome Editing Therapeutic for the Treatment of ATTR2nd European Congress for ATTR Amyloidosis — Sep 1, 2019
Delivering on the therapeutic potential of CRISPR/Cas9: Development of an LNP-mediated genome editing therapeutic for the treatment of ATTR26th 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 ATTRGenome 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 ATTRAmerican Society of Gene and Cell Therapy 21st Annual Meeting — May 16, 2018
Hereditary Angioedema (HAE)
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary AngioedemaNew England Journal of Medicine — Jan 31, 2024
NTLA-2002 Interim Clinical Data Update from Ongoing First-in-Human StudyIntellia Therapeutics Sponsored Event — Jun 12, 2023
Updated Safety and Efficacy of NTLA-2002, a CRISPR/Cas9-based Gene Editing Therapy Targeting KLKB1, in a Phase 1 Study of Patients with Hereditary AngioedemaEuropean Academy of Allergy and Clinical Immunology Hybrid Congress — Jun 11, 2023
Transient exposure to NTLA-2002, an investigational CRISPR/Cas9-based gene editing therapy, leads to durable pharmacodynamic responses and attack control in patients with hereditary angioedema13th C1 Inhibitor Deficiency and Angioedema Workshop — May 6, 2023
In vivo CRISPR/Cas9 editing of KLKB1 in patients with Hereditary Angioedema: A First-in-Human StudyAmerican College of Allergy, Asthma & Immunology 2022 Annual Scientific Meeting — Nov 12, 2022
In vivo CRISPR/Cas9 editing of KLKB1 in patients with HAE2022 Bradykinin Symposium — Sep 16, 2022
CRISPR/Cas9-mediated KLKB1 Gene Editing and Serum Kallikrein Reduction by NTLA-2002 Remains Durable in Humanized Mice Following Liver Regeneration after Partial HepatectomyAmerican Academy of Allergy, Asthma & Immunology Annual Meeting — Feb 28, 2022
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 Liver Gene Knockout of KLKB1 to Treat Hereditary Angioedema23rd Annual Meeting of the American Society of Gene and Cell Therapy — May 15, 2020
Alpha-1 Antitrypsin Deficiency (AATD)
Consecutive Genome Editing in Non Human Primate Achieves Durable Production of Human Alpha-1 Antitrypsin and Reduction of the Native Protein29th Annual Congress of the European Society of Gene & Cell Therapy — Oct 20, 2021
CRISPR/Cas9-Mediated Targeted Gene Insertion of SERPINA1 to Treat Alpha-1 Antitrypsin Deficiency24th Annual Meeting of the American Society of Gene and Cell Therapy — May 11, 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
Other Diseases and Platform Innovation
Development of In Vivo, Systemic CRISPR-Based Therapeutics24th Annual Meeting of the American Society of Gene and Cell Therapy — May 11, 2021
Avoiding Unintended Genome Editing for CRISPR/Cas9 Therapeutics24th Annual Meeting of the American Society of Gene and Cell Therapy — May 10, 2021
In Vivo Genome Editing of Hematopoietic Stem and Progenitor CellsKeystone eSymposium: Precision Engineering of the Genome, Epigenome and Transcriptome — Mar 10, 2021
A Modular CRISPR/Cas9 Genome Editing Platform for Durable Therapeutic Knockout and Targeted Gene Insertion Applications16th Annual Meeting of the Oligonucleotide Therapeutics Society — Sep 29, 2020
Validation of CRISPR / Cas9 Off-Target Discovery Profiles from In Silico Prediction, Cell-Based and Biochemical-Based Assays with Targeted Off-Target Sequencing23rd 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 PrimatesKeystone 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 ExpressionEuropean Society of Gene and Cell Therapy 27th Annual Meeting — Oct 25, 2019
CRISPR/Cas9-Mediated Gene Knockout to Address Primary HyperoxaluriaEuropean 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 TherapeuticsEuropean Society of Gene and Cell Therapy 27th Annual Meeting — Oct 23, 2019
Using Lipid Nanoparticles to Efficiently Deliver CRISPR/Cas9 for Genome EditingThe 15th Annual Meeting of the Oligonucleotide Therapeutics Society — Oct 14, 2019
CRISPR/Cas9-Mediated Gene Knockout to Address Primary HyperoxaluriaAmerican 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 PrimatesAmerican 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 insertion26th Annual Congress of the European Society of Gene and Cell Therapy — Oct 18, 2018
A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome EditingCell Reports — Feb 27, 2018
Acute Myeloid Leukemia (AML)
CRISPR-Based Gene Disruption and Integration of High-Avidity, WT1-specific T Cell Receptors Improve Antitumor T Cell functionScience Translational Medicine — Feb 9, 2022
Clinical-Scale Production and Characterization of NTLA-5001 – a Novel Approach to Manufacturing CRISPR/Cas9 Engineered T Cell Therapies63rd ASH Annual Meeting & Expositions — Dec 13, 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 ALL62nd American Society of Hematology (ASH) Annual Meeting — Dec 5, 2020
Other Diseases and Platform Innovation
Potent, CRISPR-Engineered Off-The-Shelf CAR-T And TCR-T Cells Evading Host T And NK Cell Rejection29th Annual Congress of the European Society of Gene & Cell Therapy — Oct 13, 2022
Potent, CRISPR-engineered Off-The-Shelf CD30 CAR-T Cells Evading Host T and NK Cell RejectionKeystone Symposium: Precision Genome Engineering & Emerging Cellular Therapies — May 1, 2022
A Novel Strategy for Off-The-Shelf T Cell Therapy which Evades Host T Cell and NK Cell Rejection63rd ASH Annual Meeting & Expositions — Dec 11, 2021
A Novel Strategy for Off-The-Shelf T Cell Therapies Evading Host T Cell and NK Cell Rejection28th 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 Therapy28th Annual Congress of the European Society of Gene & Cell Therapy — Oct 19, 2021
Special Edition: Expanding Intellia’s Toolbox with Base EditingCold Spring Harbor Laboratory Virtual Meeting on Nucleic Acid Therapies — Mar 25, 2021
In Vivo Model Development for Genome-Edited T Cell Therapeutics23rd 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 Engineering23rd Annual Meeting of the American Society of Gene and Cell Therapy — May 12, 2020
Developing Next-Generation Engineered TCR-T Cells with CRISPRKeystone 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 KnockoutEuropean Society of Gene and Cell Therapy 27th Annual Meeting — Oct 24, 2019
