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Intellia Therapeutics Presents Preclinical Data Demonstrating Advancements in its CRISPR-Engineered Allogeneic Platform at the 2022 Keystone Symposia’s Precision Genome Engineering Conference

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Intellia Therapeutics (NTLA) announced promising preclinical data supporting the development of NTLA-6001, an allogeneic CAR-T therapy targeting CD30-expressing hematologic cancers. Presenting at the Keystone Symposia, findings showed that their proprietary engineering platform produced immune-evading T cells, crucial for potent tumor responses. The preclinical data indicated NTLA-6001's potential in treating relapsed or refractory classical Hodgkin lymphoma. The candidate is progressing towards IND-enabling activities, reinforcing Intellia’s commitment to innovative genomic therapies.

Positive
  • NTLA-6001 shows promise in treating CD30-expressing hematologic cancers with complete tumor regression observed in preclinical models.
  • The proprietary allogeneic platform created T cells that evade immune recognition, retaining essential characteristics for effective tumor targeting.
  • Advancing NTLA-6001 towards IND-enabling activities supports the company's pipeline for CAR-T therapies.
Negative
  • None.
  • Data highlight proprietary allogeneic cell engineering platform capable of creating immune-evading T cells and deployable for TCR-T and CAR-T cell therapy
  • Findings support recent development candidate initiation of NTLA-6001, an investigational allogeneic CAR-T therapy, for the treatment of CD30-expressing hematologic cancers

CAMBRIDGE, Mass., May 02, 2022 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today announced the presentation of new preclinical data from its differentiated allogeneic cell engineering platform at Keystone Symposia’s Precision Genome Engineering Conference, taking place April 27 – May 1, 2022, in Keystone, Colorado. The data presented support the development of NTLA-6001, Intellia’s allogeneic CAR-T development candidate targeting CD30 for the treatment of CD30-expressing hematologic cancers, including relapsed or refractory classical Hodgkin lymphoma (cHL).

“We are pleased to present promising preclinical data that led to the nomination of Intellia’s wholly owned allogeneic development candidate, NTLA-6001, for CD30-expressing hematologic lymphomas. NTLA-6001 is the first candidate using our differentiated allogeneic platform, which leverages a novel combination of sequential, LNP-delivered gene edits to yield T cells shielded from immune rejection,” said Intellia Chief Scientific Officer Laura Sepp-Lorenzino, Ph.D. “Our approach to engineering T cells aims to solve key immunological challenges to allogeneicity, while retaining cell attributes necessary for potent and durable tumor killing. We look forward to advancing NTLA-6001 toward IND-enabling activities.”

The data shared at Keystone demonstrated that Intellia’s proprietary allogeneic solution created T cells that not only avoided immune recognition by host CD4 and CD8 T cells, but also were protected from NK cell-mediated killing in in vitro and in vivo mouse models. Furthermore, allogeneic T cells engineered sequentially with LNPs retained high viability, cell expansion, memory phenotype, cytotoxic and cytokine secretion characteristics. Intellia’s allogeneic platform can be deployed for TCR-T and CAR-T cell therapy.

As part of these platform advancement efforts, Intellia evaluated multiple CD30 CAR constructs in a series of in vitro and in vivo experiments. The most potent CAR construct showed complete tumor regression and protection from tumor rechallenge in a T cell lymphoma model. This lead allogeneic CAR-T cell candidate, NTLA-6001, is now in preclinical development for cHL and certain CD30+ T cell lymphomas. CD30, the target for NTLA-6001, is a cell surface protein that is often overexpressed in a variety of hematologic cancers, making it an important candidate for CAR-T cell therapy.

The presentation is available on Intellia’s website at www.intelliatx.com.

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.

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 concerning: its ability to successfully extend its leadership and harness the full potential of genomic medicines to bolster its genome editing capabilities and pipeline; its ability to generate additional data demonstrating the capability of NTLA-6001 to avoid immune recognition while retaining cell attributes for potent and durable tumor killing; its ability to deploy its platform technology, including its allogeneic cell engineering platform, to develop additional TCR-T and CAR-T cell therapies; its advancement of its development candidates, including NTLA-6001 for CD30+ lymphomas; its ability to generate data to initiate clinical trials and the timing of CTA and IND submissions, including for NTLA-6001; its ability to maintain and expand its related intellectual property portfolio; and its expectations of the potential impact of the coronavirus disease 2019 pandemic, including the impact of the Delta and Omicron variants on strategy, future operations and timing of its clinical trials, including potential clinical trials for NTLA-6001.

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, including uncertainties related to regulatory approvals to conduct clinical trials; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; the risk that clinical study results will not be positive and the risk that it will not be able to deploy its allogeneic cell engineering platform to TCR-T and CAR-T cell therapies. 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 and quarterly report on Form 10-Q, 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
+1-857-449-4175
ian.karp@intelliatx.com

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

Media:
Matt Crenson
Ten Bridge Communications
+1-917-640-7930
media@intelliatx.com
mcrenson@tenbridgecommunications.com


FAQ

What is NTLA-6001?

NTLA-6001 is an investigational allogeneic CAR-T therapy developed by Intellia Therapeutics targeting CD30-expressing hematologic cancers.

What were the results of the preclinical studies for NTLA-6001?

Preclinical studies showed that NTLA-6001 produced T cells that successfully avoided immune recognition and demonstrated complete tumor regression in mouse models.

What is the next step for NTLA-6001?

Intellia plans to advance NTLA-6001 towards IND-enabling activities to initiate clinical trials.

When was the preclinical data for NTLA-6001 presented?

The preclinical data for NTLA-6001 was presented at the Keystone Symposia from April 27 to May 1, 2022.

What cancers is NTLA-6001 targeting?

NTLA-6001 targets CD30-expressing hematologic cancers, including relapsed or refractory classical Hodgkin lymphoma.

Intellia Therapeutics, Inc

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