NTLA-5001 is a novel investigational T cell receptor (TCR)-T cell therapy which leverages Intellia’s proprietary cell engineering platform
NTLA-5001 targets Wilms’ Tumor 1 (WT1), an overexpressed intracellular antigen frequently found in acute myeloid leukemia (AML) and other hematologic and solid tumors
CAMBRIDGE, MA, USA I March 01, 2022 I Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today 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. It is the company’s first ex vivo candidate developed using Intellia’s advanced lipid nanoparticle cell engineering platform, designed to improve cell performance as compared to traditional ex vivo delivery technologies.
“As Intellia’s first wholly-owned ex vivo candidate to be dosed in a patient, this NTLA-5001 milestone represents a significant step forward in our full-spectrum approach to genome editing,” said Intellia President and Chief Executive Officer John Leonard, M.D. “AML is the most common type of acute leukemia in adults, where despite recent advancements, a significant therapeutic need still exists. We look forward to advancing this investigational engineered cell therapy as a treatment for people living with this aggressive cancer of the blood and bone marrow.”
About the NTLA-5001 Clinical Program
The Phase 1/2a study will evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose of NTLA-5001 in adults who have detectable AML after having received standard first-line therapy. The study includes a dose escalation and expansion phase, with up to 54 total participants. The dose-escalation phase of the study includes two independent arms of up to three cohorts each: Arm 1 consists of adults with AML with lower disease burden, defined as those with less than 5% blasts in bone marrow, while Arm 2 consists of adults with AML with higher disease burden, defined as those with greater than or equal to 5% blasts in bone marrow. Once a dose is identified in each arm, two expansion cohorts will be opened for further safety assessment. Visit clinicaltrials.gov (NCT05066165) for more details.
About NTLA-5001
NTLA-5001 is an investigational CRISPR/Cas9-engineered T cell receptor (TCR)-T cell therapy in development for the treatment of all genetic subtypes of acute myeloid leukemia (AML). This autologous cell therapy candidate is designed for AML patients with the HLA-A*02:01 allele and whose tumors carry the Wilms’ Tumor 1 (WT1) antigen, which is widely overexpressed in AML and other cancers. NTLA-5001 is Intellia’s first wholly owned ex vivo therapeutic candidate, developed using its proprietary cell engineering platform for the treatment of cancer. Based on preclinical results, Intellia believes its proprietary cell engineering platform will result in a pipeline of more efficacious and safer cell-based cancer therapies.
About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment. It is the most common type of acute leukemia in adults in the U.S., with more than 20,000 estimated new cases in 2021. Despite currently available treatments for AML, the five-year overall survival rate for patients remains less than 30%. AML, along with other cancer types, is often characterized by overexpression of the Wilms’ Tumor 1 (WT1) antigen.
About Intellia Therapeutics
Intellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. To fully realize the transformative potential of CRISPR-based technologies, Intellia is pursuing two primary approaches. The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of genome editing to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatx.
SOURCE: Intellia Therapeutics
Post Views: 23
NTLA-5001 is a novel investigational T cell receptor (TCR)-T cell therapy which leverages Intellia’s proprietary cell engineering platform
NTLA-5001 targets Wilms’ Tumor 1 (WT1), an overexpressed intracellular antigen frequently found in acute myeloid leukemia (AML) and other hematologic and solid tumors
CAMBRIDGE, MA, USA I March 01, 2022 I Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today 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. It is the company’s first ex vivo candidate developed using Intellia’s advanced lipid nanoparticle cell engineering platform, designed to improve cell performance as compared to traditional ex vivo delivery technologies.
“As Intellia’s first wholly-owned ex vivo candidate to be dosed in a patient, this NTLA-5001 milestone represents a significant step forward in our full-spectrum approach to genome editing,” said Intellia President and Chief Executive Officer John Leonard, M.D. “AML is the most common type of acute leukemia in adults, where despite recent advancements, a significant therapeutic need still exists. We look forward to advancing this investigational engineered cell therapy as a treatment for people living with this aggressive cancer of the blood and bone marrow.”
About the NTLA-5001 Clinical Program
The Phase 1/2a study will evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose of NTLA-5001 in adults who have detectable AML after having received standard first-line therapy. The study includes a dose escalation and expansion phase, with up to 54 total participants. The dose-escalation phase of the study includes two independent arms of up to three cohorts each: Arm 1 consists of adults with AML with lower disease burden, defined as those with less than 5% blasts in bone marrow, while Arm 2 consists of adults with AML with higher disease burden, defined as those with greater than or equal to 5% blasts in bone marrow. Once a dose is identified in each arm, two expansion cohorts will be opened for further safety assessment. Visit clinicaltrials.gov (NCT05066165) for more details.
About NTLA-5001
NTLA-5001 is an investigational CRISPR/Cas9-engineered T cell receptor (TCR)-T cell therapy in development for the treatment of all genetic subtypes of acute myeloid leukemia (AML). This autologous cell therapy candidate is designed for AML patients with the HLA-A*02:01 allele and whose tumors carry the Wilms’ Tumor 1 (WT1) antigen, which is widely overexpressed in AML and other cancers. NTLA-5001 is Intellia’s first wholly owned ex vivo therapeutic candidate, developed using its proprietary cell engineering platform for the treatment of cancer. Based on preclinical results, Intellia believes its proprietary cell engineering platform will result in a pipeline of more efficacious and safer cell-based cancer therapies.
About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment. It is the most common type of acute leukemia in adults in the U.S., with more than 20,000 estimated new cases in 2021. Despite currently available treatments for AML, the five-year overall survival rate for patients remains less than 30%. AML, along with other cancer types, is often characterized by overexpression of the Wilms’ Tumor 1 (WT1) antigen.
About Intellia Therapeutics
Intellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. To fully realize the transformative potential of CRISPR-based technologies, Intellia is pursuing two primary approaches. The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of genome editing to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatx.
SOURCE: Intellia Therapeutics
Post Views: 23
