ReCode Therapeutics, Inc.

The programme utilises Intellia’s CRISPR-based gene editing platform and DNA-writing technology. Credit: Natali _ Mis/Shutterstock. ReCode Therapeutics is set to secure up to $15m in additional funding from the Cystic Fibrosis Foundation to enhance its gene correction research programme. The initiative, conducted in collaboration with Intellia Therapeutics , aims to develop new treatments for cystic fibrosis (CF), including for patients with genotypes that are non-responsive or intolerant to existing cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The programme utilises Intellia’s clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing platform and deoxyribonucleic acid (DNA)-writing technology along with the company’s selective organ targeting (SORT) lipid nanoparticle (LNP) delivery platform aiming at gene correction. The investment will propel the development of up to three gene correction programmes which are designed to address CF mutations not covered by current therapies. It will also enhance ReCode’s existing RCT2100 clinical programme, an investigational inhaled messenger ribonucleic acid (mRNA) therapy, currently in a Phase Ib multi-dose study for CF patients. ReCode Therapeutics CEO Shehnaaz Suliman stated: “This funding will support our gene correction approach, enabling us to significantly accelerate research in collaboration with Intellia and to advance the development of a potentially transformative treatment for CF. “Along with RCT2100, our inhaled mRNA programme, we are building a therapeutic franchise to expand disease-modifying treatment options for all people with CF.” In February 2024, the company announced a strategic collaboration with Intellia Therapeutics to develop gene editing therapies for CF. CF is a debilitating genetic disease leading to persistent lung infections and respiratory failure, stemming from mutations in the CFTR gene. The company’s lead programmes, RCT2100 and RCT1100, target CFTR gene mutations unresponsive to approved modulators and primary ciliary dyskinesia, respectively. Both are inhaled mRNA-based therapies formulated with ReCode’s SORT LNP delivery platform. In January 2023, Asklepios BioPharmaceutical entered a research partnership with ReCode to explore a single-vector gene-editing platform.

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, announced today that it will receive new funding from the Cystic Fibrosis Foundation (CF Foundation) to support ReCode’s gene correction research program with the goal of developing and commercializing new treatments for people with cystic fibrosis (CF), including those with genotypes that do not respond to, or are intolerant to, approved CFTR modulators. The gene correction program is being developed with Intellia Therapeutics, utilizing Intellia’s proprietary CRISPR-based gene editing platform, including its DNA-writing technology. “We are grateful to the CF Foundation for their ongoing support of our efforts to develop novel treatments for people living with cystic fibrosis,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “This funding will support our gene correction approach, enabling us to significantly accelerate research in collaboration with Intellia and to advance the development of a potentially transformative treatment for CF. Along with RCT2100, our inhaled mRNA program, we are building a therapeutic franchise with the goal of expanding disease-modifying treatment options for all people with CF.” Key Highlights: The CF Foundation has agreed to invest up to $15 million in new funding to support gene correction therapeutic research, which is currently underway as part of a collaboration agreement with Intellia Therapeutics announced earlier this year. The programs leverage Intellia’s proprietary CRISPR-based gene editing platform, including its DNA writing technology, and ReCode’s next-generation Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform with the aim to correct the underlying genetic cause of CF and offer a potentially transformative therapy. The funding from the CF Foundation will support development activities for up to three different gene correction programs. These programs will initially focus on developing novel therapies for CF mutations that are not addressed or adequately treated by existing therapies. The funding is in addition to the CF Foundation's prior investment in ReCode to support the company’s ongoing RCT2100 clinical program investigational inhaled mRNA therapy, which is currently enrolling a Phase 1b multi-dose study in people with CF. 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. An estimated 105,000 people have been diagnosed with CF across 94 countries, according to the CF Foundation. The absence or dysfunction of the CFTR protein results in a defect in airway hydration, which leads to excessive mucus buildup in the lungs and other organs. It also results in a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, approximately 10% of people in the CF community have genetic mutations that do not enable them to benefit from these life-changing therapeutics. 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 RCT2100 for the treatment of the 10% of people with cystic fibrosis who have genetic mutations in the CFTR gene that do not respond to currently approved CFTR modulators and RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene. 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 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 and Instagram .

BETHESDA, Md.--( BUSINESS WIRE )--The Cystic Fibrosis Foundation has agreed to invest up to $15 million in ReCode Therapeutics to support their gene editing collaboration with Intellia Therapeutics. The funding is intended to be used to develop a gene editing therapy that could be delivered into the lung cells of people with cystic fibrosis. ReCode is focused on developing a version of its lipid nanoparticles that is optimized for delivery to lung stem cells, the ideal target for a permanent gene editing therapy. Similar to a pill capsule that protects medicines, lipid nanoparticles shield and direct genetic material to cells without damaging the material along the way. Lung stem cells are important because they create new lung cells to replace damaged or dying ones. Delivering genetic-based therapies to specific cells in the lungs remains a significant challenge in medicine. While ReCode concentrates on the delivery system, Intellia is working to develop the gene editing therapy being delivered. Intellia’s therapy is designed to target permanent or near-permanent correction of a specific nonsense mutation (also called a “stop” mutation) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Other rare mutations that are not eligible for CFTR modulators will be explored as the editing technology matures. “Gene editing is a promising, complex technology, which will take many years to develop into a therapy for people with CF,” said Steven Rowe, MD, executive vice president and chief scientific officer of the Cystic Fibrosis Foundation. “By investing in this research now, we are laying the foundation to develop what we hope will be a transformative treatment for people with CF, especially those who can’t take CFTR modulators.” The companies are initially targeting the lungs for gene correction because CF severely impacts the lungs and can cause respiratory failure — a leading cause of death in people with the disease. Eventually, a systemic treatment will need to be developed to correct CF in other organs impacted by the disease, such as the pancreas. The funding for the gene editing program is in addition to a separate $15 million investment in ReCode that the Foundation made to develop a messenger RNA therapy , which is being tested in Phase 1 and 2 clinical trials. Messenger RNA therapy is another potential treatment that could help all people with CF, especially those who aren’t eligible for or cannot tolerate CFTR modulators. About the Cystic Fibrosis Foundation The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org .