![]() ![]() HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. Intellia will present data updates on its potential hereditary angioedema (HAE) therapy, which uses the company’s modular lipid nanoparticle (LNP)-based CRISPR/Cas9 delivery system to knock out the prekallikrein B1 ( KLKB1) gene and reduce kallikrein activity. “CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema” Presentation date/time: Tuesday, May 12, 2020, from 3:45-4 p.m. Session: CAR T and Other Engineered T Cells Targeting Hematological Malignancies Presenter: Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy Researchers will also present data showing a significant decrease in AML tumor burden in mice treated with the WT1-specific TCRs, which are incorporated into Intellia’s lead NTLA-5001 candidate for the treatment of AML. This presentation will focus on Intellia’s ongoing research collaboration with IRCCS Ospedale San Raffaele to apply CRISPR/Cas9 technology to develop engineered T cell therapies to address intractable cancers, such as AML. “Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia” Presenter: Aaron Prodeus, Ph.D., senior scientist, Cell Therapy Intellia plans to submit an IND for NTLA-5001 for the treatment of AML in the first half of 2021. Intellia will focus its initial clinical investigation of NLTA-5001 on acute myeloid leukemia (AML), while exploring in ongoing preclinical studies its application in solid tumors. NTLA-5001 utilizes a T cell receptor (TCR)-directed approach to target the Wilms’ Tumor 1 (WT1) antigen, obtained through the ongoing research collaboration with IRCCS Ospedale San Raffaele, which is over-expressed in many hematologic and solid tumors. These platform advances support NTLA-5001, which is Intellia’s first wholly owned engineered T cell therapy development candidate. “Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering”Īs a follow-on to data presented at Keystone Symposia’s Engineering the Genome Conference this past February, Intellia’s ASGCT presentation will focus on process improvements in its CRISPR/Cas9-based engineering to deliver T cell therapies with high levels of editing, achieving robust levels of expansion, desirable memory phenotypes, improved function and reduced translocations. Intellia’s data include important updates about the company’s progress and platform development activities: Abstracts are available on the ASGCT website. (NASDAQ: NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced that five abstracts were accepted for the 23 rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), taking place virtually from May 12-15, 2020. ![]() CAMBRIDGE, Mass., Ap(GLOBE NEWSWIRE) - Intellia Therapeutics, Inc. ![]()
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