Ciliary Motility Disorders

– Study will evaluate the safety and tolerability of RCT2100, an inhaled mRNA-based therapy for the 10-13% of CF patients who have Class I mutations in the CFTR gene – – Second clinical-stage candidate from ReCode’s SORT LNP delivery platform is designed to deliver CFTR mRNA to target cells and instruct them to make a functional version of the CFTR protein – MENLO PARK, CA, USA I February 21, 2024 I 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 that the first cohort of healthy volunteer participants have been dosed in a Phase 1 clinical study of RCT2100, an inhaled mRNA-based investigational therapy for cystic fibrosis (CF) that focuses specifically on the 10-13% of patients with nonsense mutations that do not respond to or experience side effects with, approved CFTR modulators. “The initiation of the clinical study of RCT2100, which is the second candidate developed using our novel Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform, represents an important milestone for the CF community,” stated David Lockhart, Ph.D., chief scientific officer and president, ReCode Therapeutics. “While progress has been made for many people with CF, those who do not benefit from existing modulators report feeling left behind and continue to face profound physical and emotional hardship from the disease. RCT2100 is designed to target the underlying cause of CF so that more people with CF may benefit, including those with rare mutations.” CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and impacts approximately 130,000 people worldwide. Despite advancements in CFTR modulator treatments, no available therapies exist to restore function for Class I mutations in the CFTR gene since those mutations result in no CFTR protein being produced that can be modulated. Formulated using ReCode’s proprietary SORT LNP delivery platform, RCT2100 is an inhaled therapy designed to deliver CFTR mRNA to target cells and instruct them to make a functional version of the CFTR protein. The Phase 1 double-blind, placebo-controlled, first-in-human study is designed to assess the safety and tolerability of a single ascending dose of inhaled RCT2100 administered via nebulizer. The study is being conducted in New Zealand and ReCode anticipates enrolling approximately 32 healthy adults who will receive a single dose of either placebo or RCT2100. For more information, please visit clinicaltrials.gov . ReCode plans to submit an Investigational New Drug application (IND) to the U.S. Food and Drug Administration for a Phase 1 trial of RCT2100 in CF patients in the first half of 2024. About Cystic Fibrosis Cystic fibrosis (CF) is a progressive genetic disease that causes persistent lung infections and respiratory failure. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and impacts approximately 130,000 people worldwide. The absence or dysfunction of the CFTR protein results in a defect in airway hydration, which leads to excessive mucus buildup in the lungs. It also creates a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, no available therapies exist to restore function for Class I mutations in the CFTR gene since no significant CFTR protein is produced. 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 novel 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 . SOURCE: ReCode Therapeutics

MENLO PARK, Calif.--(BUSINESS WIRE)-- 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 that company management will participate in the upcoming February conferences: William Blair Private Company Focus Conference Format: Fireside Chat Date: February 22, 2024 Time: 12:00 p.m. ET Location: Virtual Evercore ISI 2024 Emerging Biotech Conference Format: Company Presentation Date: February 28, 2024 Time: 4:00 p.m. ET Location: Virtual 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 and follow us on LinkedIn.

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. and MENLO PARK, Calif., Feb. 15, 2024 (GLOBE NEWSWIRE) -- 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 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. 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