In vivo
"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
"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
"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
"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
"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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