Intellia Therapeutics to Present New Preclinical Data Highlighting In Vivo and Ex Vivo Genome Editing Advances at 2021 European Society of Gene & Cell Therapy Annual Congress

Rhea-AI Summary

Intellia Therapeutics (NASDAQ:NTLA) has presented key findings at the 29th Annual Congress of the European Society of Gene & Cell Therapy. The company showcased its proprietary allogeneic T cell therapy platform designed to address immune rejection in therapeutic candidates for cancer and autoimmune diseases. Significant advancements include a lipid nanoparticle-based CRISPR/Cas9 delivery system and the first reported consecutive in vivo gene insertion and knockout in non-human primates aimed at treating alpha-1 antitrypsin deficiency (AATD). This work positions Intellia at the forefront of genome editing therapeutics.

Positive

• Proprietary allogeneic T cell therapy platform shows promise for overcoming immune rejection.
• Successful demonstration of lipid nanoparticle-based CRISPR/Cas9 delivery to T cells.
• First reported consecutive in vivo gene insertion and knockout in non-human primates for AATD treatment.

Negative

• None.

• First data highlighting proprietary allogeneic cell engineering platform designed to overcome immune rejection for the development of therapeutic candidates to treat a variety of cancer and autoimmune diseases
• New data on proprietary cell engineering process utilizing lipid nanoparticle-based delivery of CRISPR/Cas9 ex vivo to T cells
• First reported consecutive in vivo gene insertion and knockout in non-human primates (NHPs) for the treatment of alpha-1 antitrypsin deficiency (AATD)
  

CAMBRIDGE, Mass., Oct. 12, 2021 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today announced the presentation of new data at the 29^th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) meeting, taking place virtually from October 19-21, 2021.

“We are excited to share for the first time important preclinical data and new developments highlighting both our allogeneic T cell therapy platform as well as the utilization of lipid nanoparticle-based delivery of the CRISPR/Cas9 gene editing components to more efficiently engineer T cells for therapeutic use,” said Intellia Chief Scientific Officer Laura Sepp-Lorenzino, Ph.D. “Additionally, we will be presenting non-human primate data from our in vivo platform where we have demonstrated our ability to both insert a functional gene and inactivate a gene to treat AATD. For AATD, a genetic disease that can cause lung dysfunction and/or liver disease and which currently has no cure, our modular delivery platform provides us the optionality for patient-tailored treatments relevant to the disease manifestation. We look forward to sharing these data with the scientific community as we continue to advance our mission of delivering breakthrough genome editing treatments for people with severe diseases.”

ESGCT Annual Congress Presentations

Oral Presentations:

Title: A Novel Strategy for Off-the-shelf T Cell Therapies Evading Host T Cell and NK Cell Rejection
Abstract number: OR18
Date/Time: Wednesday, October 20, 2021, 10:45 a.m. CEST
Location: Session 2c: Immunotherapy for cancer & CAR T cells
Presenting Author: Yong Zhang, Ph.D., associate director, Cell Therapy

Title: Consecutive Genome Editing in Non-Human Primate Achieves Durable Production of Human Alpha-1 Antitrypsin at Physiologic Levels and Reduction of the Homologous Native Protein
Abstract number: OR12
Date/Time: Wednesday, October 20, 2021, 10:15 a.m. CEST
Location: Session 2b: Gene editing I
Presenting Author: Sean Burns, M.D., vice president of Intellia’s Disease Biology and Pharmacology group

Invited Talk:
Title: Advances in CRISPR/Cas9 Therapeutic Genome Editing for In Vivo and Ex Vivo Applications
Date/Time: Friday, October 22, 2021, 11:30 a.m. CEST
Location: Session 7b: Liver and metabolic diseases II
Presenting Author: Laura Sepp-Lorenzino. Ph.D., chief scientific officer  

Poster Presentation:
Title: Lipid Nanoparticles (LNPs) as a Superior CRISPR/Cas9 Delivery Modality for Highly Efficient Multiplex Gene Editing of T Cells for Adoptive Cell Therapy
Abstract number: P205
Date/Time: Tuesday, October 19, 2021, 8:00 a.m. CEST
Presenting Author: Aaron Prodeus, Ph.D., principal scientist, Cell Therapy

Additional data collected will be included in final meeting presentations. All abstracts for the ESGCT Annual Congress will be available on ESGCT’s website here.

About Intellia Therapeutics 

Intellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. To fully realize the transformative potential of CRISPR/Cas9, 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 CRISPR/Cas9 to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding its: advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; expectations of the potential impact of the coronavirus disease 2019 pandemic on strategy, future operations and timing of its clinical trials or IND submissions; and statements regarding the timing of regulatory filings regarding its development programs.

Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; and the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission (“SEC”). All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Investors:
Ian Karp
Senior Vice President, Investor Relations and Corporate Communications
ian.karp@intelliatx.com

Lina Li
Director, Investor Relations
+1-857-706-1612
lina.li@intelliatx.com

Media:
Lisa Qu
Ten Bridge Communications
+1-678-662-9166
media@intelliatx.com
lqu@tenbridgecommunications.com 

FAQ

What is the main focus of Intellia Therapeutics' recent presentations at the ESGCT?

Intellia Therapeutics focused on advancements in its CRISPR/Cas9 technology for developing therapies for cancer and autoimmune diseases.

What significant results did Intellia report regarding alpha-1 antitrypsin deficiency (AATD)?

Intellia reported the first consecutive in vivo gene insertion and knockout in non-human primates aimed at treating AATD.

How does Intellia's proprietary cell engineering platform work?

It utilizes lipid nanoparticle-based delivery of CRISPR/Cas9 to efficiently engineer T cells for therapeutic use.

What potential does Intellia see in its allogeneic T cell therapy platform?

Intellia aims to provide off-the-shelf therapies that evade host T cell and NK cell rejection.

When and where were Intellia's findings presented?

The findings were presented during the ESGCT Annual Congress held virtually from October 19-21, 2021.