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– Phase 1b study will evaluate the safety and tolerability of multiple ascending doses of RCT2100 in people with cystic fibrosis – MENLO PARK, CA, USA I September 26, 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 patient has been dosed in a Phase 1b study evaluating RCT2100, an investigational inhaled mRNA therapy for people with cystic fibrosis (CF). CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, impacting approximately 130,000 people worldwide. Although recent advancements in CFTR modulator therapies have improved outcomes for many people with CF, those with certain mutations remain underserved because these mutations prevent the production of functional CFTR protein, making current treatments ineffective. “The initiation of our Phase 1b study of RCT2100 in cystic fibrosis marks a significant milestone for us and is a step closer to offering new hope to those who cannot benefit from existing CF therapies,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “We are immensely grateful for the ongoing support and partnership of the CF patient community and are focused on accelerating enrollment to bring this potential new treatment to those in need as quickly as possible.” Formulated using ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform, RCT2100 is designed to deliver CFTR mRNA directly to target cells in the lungs, instructing them to produce a functional version of the CFTR protein that is missing in some individuals with CF. By providing this CFTR mRNA, the therapy aims to address the root cause of CF, potentially restoring CFTR protein function in the lungs rather than just managing the symptoms. ReCode recently received global regulatory authorization to proceed with the RCT2100 Phase 1b study in the United States (U.S.), United Kingdom (UK), and Europe. The study is currently enrolling at sites in the Netherlands, with future expansion planned for France, the U.S. and the UK. For more information, please visit CF-Clinical-Studies.com . 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 Cystic Fibrosis 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. It also creates a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, approximately 10% of the CF community have genetic mutations that do not 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 cystic fibrosis patients 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 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 and Instagram . 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 presented preclinical data from its mRNA-based cystic fibrosis (CF) program at the North American Cystic Fibrosis Conference (NACFC), taking place September 26-28, 2024, in Boston, Massachusetts. These preclinical data demonstrate that RCT2100 significantly restored CFTR function in human bronchial epithelial (HBE) cells derived from people with CF, achieving up to 135% of the efficacy seen with an elexacaftor/tezacaftor/ivacaftor equivalent in cells with an F508del mutation on one allele and a minimal function mutation on the other allele. Additionally, in vivo studies using a G551D CF ferret model revealed notable improvement in mucociliary clearance (MCC) post-treatment, highlighting RCT2100's potential to address the medical needs of approximately 10% of people with CF not eligible for current CFTR modulator therapy. "These preclinical data underscore the potential of RCT2100 as a novel therapeutic approach for people with cystic fibrosis who do not respond to existing CFTR modulators,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “In the studies, RCT2100, an inhaled mRNA-based therapy, successfully restored CFTR function in patient-derived cells in vitro and improved mucociliary clearance, which is critical for improving patient outcomes, in a ferret model of CF lung disease. These findings support our goal of developing innovative therapies that address the needs of the entire cystic fibrosis community, especially those still awaiting effective treatment options.” “The results seen in this preclinical study are significant in that they demonstrate the ability of RCT2100 to improve CFTR function in HBE cells at a level previously only seen with triple combination CF drug modulator treatments,” said Marco Weinberg, Ph.D., Head of Research at ReCode Therapeutics. “In a separate study, the use of RCT2100 demonstrated that when used in the G551D CF ferret model, treatment resulted in a notable improvement in mucociliary clearance. This is significant in that treatment with RCT2100 resulted in an increase in MCC after two weeks, similar to that seen with existing modulator therapy after only two doses administered once per week. Combined, these data highlight the potential of RCT2100 as a treatment option for a group of CF patients that do not respond to current therapy.” ReCode is pleased to welcome NACFC participants to booth #816 in the Exhibit Hall. 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 Cystic Fibrosis 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. It also creates a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, approximately 10% of the CF community have genetic mutations that do not 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 RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene, and 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. 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 and Instagram .

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 the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for RCT1100 for the treatment of primary ciliary dyskinesia (PCD). RCT1100 is a novel investigational therapy being developed to treat PCD caused by pathogenic mutations in the DNAI1 gene. PCD is a rare and progressive genetic disease characterized by the dysfunction of cilia, which are microscopic hairlike structures that line the respiratory tract. Defective ciliary function results in the inability to clear mucus from the airways. As a result, bacteria and viruses become trapped in the airways, leading to chronic infections and compromised respiratory function, with a small percentage of patients requiring a lung transplant. Mutations in the DNAI1 gene account for approximately seven percent of PCD cases. “PCD is a rare genetic lung disorder that currently has no FDA-approved treatments; therefore, receiving this designation is an important recognition for the PCD patient community," said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. "We are actively enrolling PCD patients in our single-dose study across the U.S. and UK and look forward to sharing an update when enrollment is completed.” ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform was used to develop RCT1100, an inhaled mRNA therapy designed to deliver DNAI1 mRNA to target airway cells. The DNAI1 mRNA is translated into the DNAI1 protein inside cells, which is intended to establish proper ciliary function by addressing the underlying cause of the disease. In preclinical studies, the company’s DNAI1 mRNA led to efficient production of the DNAI protein and rescue of ciliary function in relevant models of PCD. ReCode is currently enrolling patients in Part B of a Phase 1, open-label study designed to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered with a nebulizer in patients with PCD caused by pathogenic mutations in the DNAI1 gene. For more information, please visit clinicaltrials.gov. About Primary Ciliary Dyskinesia Primary ciliary dyskinesia (PCD) is a rare genetic disease characterized by deficient mucociliary clearance (MCC), chronic respiratory tract infections, bronchiectasis, and declining respiratory function. Mutations in more than 50 genes result in dysfunctional cilia and loss of MCC. PCD is a progressive disorder, meaning it will get worse over time, for which no cure or disease-modifying treatments currently exist. PCD is estimated to affect at least 1 in 7,500 people across diverse ethnicities, with approximately 45,000 patients in the U.S. Due to the lack of widespread screening, approximately 80% of people with PCD are thought to be undiagnosed. ReCode has launched ThinkPCD.com, a program that provides genetic testing to PCD patients who qualify at no cost. Patients can enroll directly or through a healthcare professional who can go to the website to determine if their patients are eligible for genetic testing through the program. 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 or other mutations in the CFTR gene that 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 and Instagram.