Allo WT1-TCR / Intellia Therap - LARVOL DELTA

Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2 | N=6 | Terminated | Sponsor: Intellia Therapeutics | N=54 ➔ 6 | Trial completion date: Sep 2025 ➔ Aug 2022 | Active, not recruiting ➔ Terminated | Trial primary completion date: Nov 2023 ➔ Jul 2022; Pivoting to an allogeneic version of this program currently in preclinical development.

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • HLA-A

Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2 | N=54 | Active, not recruiting | Sponsor: Intellia Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • HLA-A

Intellia Therapeutics Announces First Quarter 2022 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "Intellia is initiating IND-enabling activities for NTLA-6001. At the 2022 Keystone Symposia’s Precision Genome Engineering Conference, Intellia presented preclinical data leading to the development of NTLA-6001....NTLA-5001 for AML: Continue to enroll patients in Phase 1/2a study in 2022."

Enrollment status • Acute Myelogenous Leukemia • Hematological Malignancies • Hodgkin Lymphoma • Oncology

Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-5001 for the Treatment of Acute Myeloid Leukemia (GlobeNewswire) - "Intellia Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Intellia’s ex vivo investigational T cell receptor (TCR)-T cell therapy, NTLA-5001, for the treatment of acute myeloid leukemia (AML)."

Orphan drug • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Intellia Therapeutics Announces First Patient Dosed in Phase 1/2a Clinical Trial of NTLA-5001 for the Treatment of Acute Myeloid Leukemia (GlobeNewswire) - "Intellia Therapeutics...announced that the first patient has been dosed with NTLA-5001, the company’s ex vivo CRISPR/Cas9 genome editing candidate for the treatment of acute myeloid leukemia (AML). NTLA-5001 is an autologous T cell receptor (TCR)-T cell therapy designed to target the Wilms’ Tumor (WT1) antigen, which is found in AML and many other hematologic and solid tumors."

Trial status • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Intellia Therapeutics Announces Fourth Quarter and Full-Year 2021 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "In the fourth quarter of 2021, Intellia initiated screening of patients in the Phase 1/2a study of NTLA-5001 for patients with AML. The Company has begun enrolling patients and expects to dose its first patient in the coming weeks. Later this year, the Company plans to provide guidance around timing of the first expected data readout, with the goal of demonstrating clinical proof of concept for its TCR-based platform."

P1/2 data • Trial status • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Intellia Therapeutics Highlights Strategic Priorities and Anticipated Development Milestones for 2022 (GlobeNewswire) - "In the fourth quarter of 2021, Intellia initiated screening of patients in the Phase 1/2a study of NTLA-5001 for patients with AML. The Company expects to dose its first patient in the coming weeks and enroll patients throughout the year. Later this year, the Company plans to provide guidance around timing of the first expected data readout, with the goal of demonstrating clinical proof-of-concept for its TCR-based platform."

Trial status • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Clinical-Scale Production and Characterization of Ntla-5001 - a Novel Approach to Manufacturing CRISPR/Cas9 Engineered T Cell Therapies (ASH 2021) - "In addition, NTLA-5001 drug product displayed cytotoxic functionality when exposed to cell lines presenting the target WT1 peptide. The NTLA-5001 clinical-scale manufacturing process is a controlled and robust platform for the generation of minimally differentiated T cells with high rates of editing and transgene expression."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • Wilms Tumor • CD4 • CD8 • HLA-A • SELL • TRB • WT1

Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2; N=54; Recruiting; Sponsor: Intellia Therapeutics; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • HLA-A

Intellia Therapeutics to Highlight Ex Vivo Genome Editing and CRISPR/Cas9 Manufacturing Advances at 2021 American Society of Hematology (ASH) Annual Meeting (GlobeNewswire) - "...'The data will feature our novel allogeneic technology designed to overcome rejection by host T and NK cells without the need for host immune suppression, as well as highlight our clinical-scale manufacturing process developed for NTLA-5001, our TCR-based T cell therapeutic candidate for the treatment of acute myeloid leukemia'."

Commercial • Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Intellia Therapeutics Announces Third Quarter 2021 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "Intellia intends to initiate patient screening by year-end for a Phase 1/2a study evaluating NTLA-5001 in adults with persistent or recurrent AML who have previously received first-line therapy."

Trial initiation date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Phase 1/2a, Single Dose Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2; N=54; Not yet recruiting; Sponsor: Intellia Therapeutics

Clinical • New P1/2 trial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • HLA-A

Intellia Therapeutics Announces U.S. FDA Acceptance of Investigational New Drug Application for NTLA-5001, its CRISPR/Cas9-Engineered TCR-T Cell Candidate for Acute Myeloid Leukemia (GlobeNewswire) - "Intellia Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has accepted the investigational new drug (IND) application for NTLA-5001, the company’s first wholly-owned ex vivo CRISPR genome editing candidate for the treatment of cancer....In addition to the U.S., Intellia has also submitted a regulatory application to the U.K. for NTLA-5001."

European regulatory • IND • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Intellia Therapeutics Announces Second Quarter 2021 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "Upcoming Milestones: AML: Initiate patient screening in the Phase 1 study of NTLA-5001 by year-end."

Trial status • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology

Intellia Therapeutics Highlights Strategic Priorities and Anticipated Development Milestones for 2021 (GlobeNewswire) - "Intellia plans to submit an Investigational New Drug (IND) or equivalent regulatory application for NTLA-5001 in mid-2021. The first-in-human trial is expected to evaluate the safety and activity of NTLA-5001 in patients with persistent or recurrent AML..."

IND • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology

Intellia Therapeutics Presents New Preclinical Data Supporting Its CRISPR/Cas9-Engineered TCR-T Cell Treatment for Acute Myeloid Leukemia at the 62nd ASH Annual Meeting (GlobeNewswire) - "Intellia Therapeutics, Inc...is presenting new preclinical data in support of NTLA-5001...at the 62nd American Society of Hematology (ASH) Annual Meeting, taking place virtually from December 5-8, 2020...Specifically, NTLA-5001’s lead TCR-T cells resulted in significantly higher anti-tumor activity in mouse models of acute leukemias than that observed in mice treated with cells engineered using the standard process...Intellia plans to submit an Investigational New Drug (IND) application or equivalent for NTLA-5001 in the first half of 2021...the first-in-human trial planned to evaluate safety and activity in patients with persistent or recurrent AML who have previously received first-line therapies."

IND • New trial • Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology

[VIRTUAL] NTLA5001, 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 (ASH 2020) - "This can be achieved with CRISPR/Cas9-mediated replacement of the endogenous TCR α and β chains, by knocking out the TRAC and TRBC genes and inserting the tgTCR into the TRAC locus. NTLA-5001 is being advanced into clinical development for AML immunotherapy. Given the expression of WT1 in many solid tumors, engineered WT1 TCR-T cells are being further explored in those indications."

IO Biomarker • Preclinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Oncology • Solid Tumor • Wilms Tumor • CD8 • IL15 • WT1

Intellia Therapeutics Announces Third Quarter 2020 Financial Results (GlobeNewswire) - "AML: Submit an IND or IND-equivalent for NTLA-5001 in 1H 2021."

IND • Acute Myelogenous Leukemia • Amyloidosis • Hematological Malignancies • Oncology

Intellia Therapeutics Announces Second Quarter 2020 Financial Results (GlobeNewswire) - "Intellia continues to advance Investigational New Drug application (IND)-enabling activities and remains on track to submit an IND or IND-equivalent for NTLA-5001 in the first half of 2021. Additional efforts are underway to evaluate potential use of the WT1-targeted TCR construct to treat solid tumors."

Clinical • IND • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology • Solid Tumor

Intellia Therapeutics reports progress on CRISPR/Cas9 AML cancer therapy using proprietary cell engineering process at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (GlobeNewswire) - "Intellia researchers are presenting new data in support of NTLA-5001, the company’s engineered cell therapy candidate for the treatment of acute myeloid leukemia (AML)...'The data being presented today validate Intellia’s approach of reducing AML tumor cell blasts, and our plans to enter the clinic with NTLA-5001 next year'....The company confirmed plans last week to submit an IND or IND-equivalent for NTLA-5001 for the treatment of AML in the first half of 2021."

IND • New trial • Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Intellia Therapeutics announces first quarter 2020 financial results (GlobeNewswire) - "NTLA-5001 for the treatment of acute myeloid leukemia (AML) is Intellia’s first T cell receptor (TCR)-T cell development candidate, which targets the Wilms’ Tumor 1 (WT1) antigen…The Company is on track to submit an IND or IND-equivalent for NTLA-5001 in the first half of 2021."

NDA • Acute Myelogenous Leukemia • Genito-urinary Cancer • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • Wilms Tumor

Intellia Therapeutics announces fourth quarter and full-year 2019 financial results (GlobeNewswire) - “Plans to submit an IND application for NTLA-5001, a WT1-directed TCR-T cell therapy, for the treatment of acute myeloid leukemia in 1H 2021.”

IND

Intellia Therapeutics presents new data from its engineered cell therapy and in vivo programs at Keystone Symposia’s Engineering the Genome Conference (GlobeNewswire) - "...we are moving ahead with IND-enabling studies and manufacturing for NTLA-5001, to enable a regulatory submission in the first half of 2021....The company is presenting data demonstrating that the selection of a natural, high-affinity TCR, in combination with CRISPR-enabled engineering and targeted insertion, results in an engineered T cell capable of specific and potent killing of primary AML blasts."

IND • Preclinical

Intellia Therapeutics highlights recent progress and anticipated 2020 milestones (GlobeNewswire) - "Intellia today announced NTLA-5001 as its first engineered T cell therapy development candidate, utilizing its T cell receptor (TCR)-directed approach to target the Wilms’ Tumor 1 (WT1) intracellular antigen for the treatment of acute myeloid leukemia (AML)....The Company expects to present preclinical data in support of NTLA-5001 at an upcoming scientific meeting in the first quarter of 2020 and plans to submit an IND application in the first half of 2021."

IND • Preclinical

Newly added product (GlobeNewswire) - Preclinical, Oncology, AML

Pipeline update