On April 28, 2020 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported that five abstracts were accepted for the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper), taking place virtually from May 12-15, 2020. Abstracts are available on the ASGCT (Free ASGCT Whitepaper) website (Press release, Intellia Therapeutics, APR 28, 2020, View Source [SID1234556686]).

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Intellia’s data include important updates about the company’s progress and platform development activities:

Oral Presentations

"Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering"
As a follow-on to data presented at Keystone Symposia’s Engineering the Genome Conference this past February, Intellia’s ASGCT (Free ASGCT Whitepaper) 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. These platform advances support NTLA-5001, which is Intellia’s first wholly owned engineered T cell therapy development candidate. 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. 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. Intellia plans to submit an IND for NTLA-5001 for the treatment of AML in the first half of 2021.

Presenter: Aaron Prodeus, Ph.D., senior scientist, Cell Therapy
Abstract number: 35
Session: CAR T-Cell Therapies I
Presentation date/time: Tuesday, May 12, 2020, from 11:45 a.m.-12 p.m. ET

"Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia"
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. 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.

Presenter: Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy
Abstract number: 78
Session: CAR T and Other Engineered T Cells Targeting Hematological Malignancies
Presentation date/time: Tuesday, May 12, 2020, from 3:45-4 p.m. ET

"CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema"
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. 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. This presentation will include results from the company’s ongoing collaboration with researchers at Regeneron and also will build on initial data released at Keystone Symposia’s Engineering the Genome Conference in February. Intellia expects to nominate a development candidate for HAE in the first half of 2020.

Presenter: Jessica Seitzer, director, Genomics
Abstract number: 1320
Session: Gene Regulation and Delivery Technologies
Presentation date/time: Friday, May 15, 2020, from 10:30-10:45 a.m. ET

Poster Presentations

"Validation of CRISPR/Cas9 Off-Target Discovery Profiles from In Silico Prediction, Cell-Based and Biochemical-Based Assays with Targeted Off-Target Sequencing"
This presentation will highlight Intellia’s approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.

Presenter: Nishit Patel, Ph.D., senior associate scientist, Informatics, Computational Biology and Genomics
Abstract number: 203
Session date/time: Tuesday, May 12, 2020, from 5:30-6:30 p.m. ET

"In Vivo Model Development for Genome-Edited T Cell Therapeutics"
Researchers will present improved murine models used to assess safety, efficacy and persistence of therapeutic modalities in cell-based therapies. Together with biotechnology company Taconic Biosciences, Intellia developed two in vivo mouse models, which allow for enhanced monitoring of graft-versus-host disease and human natural killer cytotoxicity.

Presenter: Yong Zhang, Ph.D., principal scientist, Cell Therapy
Abstract number: 1174
Session date/time: Thursday, May 14, 2020, from 5:30-6:30 p.m. ET

Workshop and Symposia

"Building a Modular CRISPR/Cas9 Platform for Human Therapeutic Applications"
Intellia will participate in the ASGCT (Free ASGCT Whitepaper) Translational Science Genome Editing Pre-Meeting Workshop, which will include an overview on important factors to consider in building a modular CRISPR/Cas9 platform for human therapeutic applications.

Presenter: Jessica Seitzer, director, Genomics
Session: Gene Editing Workshop
Presentation date/time: Monday, May 11, 2020, from 4:10-4:30 p.m. ET

Industry Sponsored Symposia

Intellia will participate in an industry lunchtime panel discussion during ASGCT (Free ASGCT Whitepaper).

Panelist: Laura Sepp-Lorenzino, Ph.D., chief scientific officer
Panel date/time: Wednesday, May 13, 2020, from 12-1:30 p.m. ET