Intellia Therapeutics and ReCode Therapeutics Announce Strategic Collaboration to Develop Novel Gene Editing Therapies for Cystic Fibrosis

Rhea-AI Summary

Intellia Therapeutics (NTLA) and ReCode Therapeutics announce a strategic collaboration to develop novel genomic medicines for treating cystic fibrosis. The collaboration combines Intellia's CRISPR-based platform with ReCode's Selective Organ Targeting (SORT) lipid nanoparticle delivery to target disease-causing gene mutations in the lung. Intellia will focus on editing strategy design, while ReCode will lead preclinical and clinical development and worldwide commercialization. The partnership aims to provide life-changing therapies for CF patients.

Insights

Medical Research Analyst

The strategic collaboration between Intellia Therapeutics and ReCode Therapeutics to develop genomic medicines for cystic fibrosis (CF) represents a significant step in the application of CRISPR gene-editing technology. CF is caused by mutations in the CFTR gene and the partnership aims to correct these mutations using Intellia’s CRISPR-based platform and ReCode's SORT LNP delivery system. This approach could potentially provide a long-term solution for CF patients, particularly those with limited treatment options.

From a research perspective, the combination of CRISPR and SORT LNP is noteworthy. CRISPR technology allows for precise gene editing, while SORT LNP ensures targeted delivery to specific organs, in this case, the lungs. The efficacy of such a combination could set a precedent for treating other genetic diseases with organ-specific manifestations. Monitoring the progress of preclinical and clinical trials will be crucial for evaluating the safety and effectiveness of these therapies.

Financial Analyst

Intellia’s collaboration with ReCode has financial implications that are important for investors to consider. The agreement includes development and commercial milestone payments, along with royalties on potential sales, which could provide Intellia with a significant revenue stream if the therapies prove successful. Furthermore, the option for Intellia to lead commercialization in the U.S. for certain programs could enhance its market position and profitability in the lucrative U.S. healthcare market.

The announcement may influence Intellia’s stock performance in the short-term due to investor optimism. However, the long-term financial impact will depend on the clinical trial outcomes, regulatory approvals and the ability of both companies to effectively commercialize the therapies. Given the high costs associated with drug development, the collaboration could also mitigate financial risks by sharing the burden between the two entities.

Market Research Analyst

The market for CF treatments is competitive, with a need for innovative solutions that can address the underlying genetic cause of the disease. The collaboration between Intellia and ReCode could fill a significant unmet need in the CF market, especially for patients who do not respond to current treatments. The potential market size for effective gene therapies in CF is substantial, given the chronic nature of the disease and the patient population that requires lifelong management.

Moreover, the collaboration’s success could disrupt the broader market for genetic disorders, as it would validate the combined use of CRISPR and targeted delivery platforms for other diseases. It is essential to track the development of this partnership and the emerging data from clinical trials, as these will provide insights into the potential market adoption and impact of the therapies developed.

• Collaboration combines Intellia’s leading CRISPR-based platform, including its DNA writing technology, with ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) to extend the reach of gene editing to disease-causing targets in the lung

CAMBRIDGE, Mass. & MENLO PARK, Calif.--(BUSINESS WIRE)-- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, and ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF). CF is a genetic disease caused by mutations in the CFTR gene, leading to the accumulation of thick mucus in the lungs, digestive systems and other organs. CF can result in life-threatening infections, respiratory failure and other serious complications.

The collaboration will leverage Intellia’s proprietary CRISPR-based gene editing platform, including its DNA writing technology, and ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform to precisely correct one or more CF disease-causing gene mutations. As part of the agreement, the companies will focus initial research efforts on therapeutic approaches that address CF for patients who have limited or no treatment options available, with the opportunity to expand the scope of the collaboration in later phases. Intellia will be responsible for the design of the editing strategy and research-grade components for the investigational therapies. ReCode will lead the subsequent preclinical and clinical development. ReCode will also lead worldwide commercialization for certain programs arising from the collaboration. Intellia will be eligible to receive pre-specified development and commercial milestone payments, as well as royalties on potential sales. Intellia may also exercise an option to lead commercialization in the U.S. for certain programs.

“Intellia’s vision to realize the full promise of gene editing includes extending the reach of our industry-leading CRISPR-based platform to targets outside the liver. This collaboration with ReCode is aimed at achieving that goal as we work together to accelerate the development of potentially life-changing therapies for people with cystic fibrosis,” said Intellia President and Chief Executive Officer John Leonard, M.D. “Building on our CRISPR/Cas9 capabilities, we have made important progress advancing our proprietary DNA writing technology to enable a range of precise editing strategies. We are excited to combine our gene editing expertise and platform with ReCode’s novel lung-directed LNP delivery platform.”

“We are excited to partner with Intellia, a clear leader in the gene editing space, with the ultimate goal of bringing life-altering therapies to CF patients,” said ReCode Chief Executive Officer Shehnaaz Suliman, M.D. (MB ChB), M.B.A., M.Phil. “This collaboration provides further validation of ReCode's SORT LNP platform to deliver diverse gene editing modalities to specific cells and tissues. By combining our highly synergistic technologies and capabilities, we are excited about the potential to enable a faster path for next-generation gene editing therapeutics to CF patients.”

About Intellia Therapeutics

Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. The company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. Intellia’s ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at intelliatx.com and follow us @intelliatx.

About ReCode Therapeutics

ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues and cells implicated in disease, enabling improved efficacy and potency. ReCode’s lead programs include RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene, and RCT2100 for the treatment of the 10-13% of cystic fibrosis patients who have Class I mutations in the CFTR gene and do not respond to currently approved CFTR modulators. RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated using the SORT LNP delivery platform. ReCode is expanding its pipeline to develop potential therapies for other rare and common genetic diseases, including musculoskeletal, central nervous system, liver and infectious disease indications.

ReCode has been recognized by the San Francisco Business Times and Silicon Valley Business Journal as a Best Place to Work. For more information, visit www.recodetx.com and follow us on LinkedIn.

Intellia Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, express or implied statements regarding Intellia’s beliefs and expectations regarding: its strategy, business plans and focus; its ability to leverage its proprietary CRISPR-based gene editing platform, including its DNA writing technology, and to combine its platform with the proprietary lipid nanoparticle (“LNP”) delivery platform developed by ReCode Therapeutics, Inc. (“ReCode”) to precisely correct one or more cystic fibrosis (“CF”) disease-causing gene mutations; its ability to develop therapeutic approaches that address CF for patients who have limited or no treatment options available, and to expand the scope of the collaboration in later phases; its ability to commercialize in the U.S. the products that may result from its collaboration with ReCode; and the expected strategic benefits of any current or future collaborations, including its collaboration with ReCode.

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, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks related to Intellia’s ability to protect and maintain its intellectual property portfolio; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of Intellia’s licensors to protect and maintain their intellectual property position; uncertainties related to the development of the company’s product candidates, including product candidates to be developed in its collaboration with ReCode, and the authorization, initiation and conduct of studies and other development requirements for such product candidates; the risk that any one or more of Intellia’s or its collaborators’ product candidates (including the product candidates to be developed with ReCode) 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; and the risk that Intellia’s collaboration with ReCode or its other collaborations will not continue or will not be successful. These and other risks and uncertainties are described in greater detail in Intellia’s other filings with the Securities and Exchange Commission, including 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 other filings. Any forward-looking statements contained in this press release represent Intellia’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Intellia explicitly disclaims any obligation to update any forward-looking statements, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240215392146/en/

Intellia Contacts:

Investors:

Ian Karp

Senior Vice President, Investor Relations and Corporate Communications

ian.karp@intelliatx.com

Lina Li

Senior Director, Investor Relations and Corporate Communications

lina.li@intelliatx.com

Media:

Matt Crenson

Ten Bridge Communications

media@intelliatx.com

TBCIntellia@tenbridgecommunications.com

ReCode Contacts:

Investors:

Anne Marie Fields

Stern IR

AnneMarie.Fields@sternir.com

IR@recodetx.com

Media:

Erica Jefferson

Senior Vice President, Corporate Affairs

ejefferson@recodetx.com

Tara Cooper

The Grace Group

tara@gracegroup.us

Source: ReCode Therapeutics

FAQ

What is the collaboration between Intellia Therapeutics and ReCode Therapeutics about?

Intellia and ReCode are collaborating to develop genomic medicines for treating cystic fibrosis by combining Intellia's CRISPR-based platform with ReCode's Selective Organ Targeting (SORT) lipid nanoparticle delivery.

What is cystic fibrosis (CF) and how is it caused?

Cystic fibrosis is a genetic disease caused by mutations in the CFTR gene, leading to the accumulation of thick mucus in the lungs, digestive systems, and other organs.

What will Intellia focus on in the collaboration?

Intellia will be responsible for designing the editing strategy and providing research-grade components for the investigational therapies.

What will ReCode be responsible for in the collaboration?

ReCode will lead the subsequent preclinical and clinical development and worldwide commercialization for certain programs arising from the collaboration.

What are the potential benefits of the collaboration for CF patients?

The collaboration aims to accelerate the development of potentially life-changing therapies for people with cystic fibrosis by targeting disease-causing gene mutations in the lung.