RCT-1100

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 the appointment of Dean J. Mitchell as chairman of its Board of Directors. Mr. Mitchell is a highly experienced and well-regarded biotechnology and pharmaceutical industry veteran who brings over three decades of experience as a board director and corporate executive to the ReCode board. “We are delighted to have Dean join the board as we continue to advance our respiratory clinical development programs and leverage our novel SORT LNP delivery platform to expand our portfolio,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “His proven track record at growing and scaling biopharma companies is a welcome addition to our board that will support the company’s near- and long-term growth.” “I am pleased to join the talented ReCode board at a pivotal time for the company as it continues to achieve significant milestones for its lead clinical programs and expand its research and discovery efforts into gene correction,” said Mr. Mitchell. “I look forward to working with the board and the executive team to advance these novel therapies with the goal of one day providing much-needed treatments for patients living with rare diseases.” Mr. Mitchell currently serves as chairman of the Board of Directors of Praxis Precision Medicines, Inc., an appointment he has held since 2020, and as a board director for Theravance Biopharma, Inc. (since 2014) and Precigen (since 2009). Mr. Mitchell previously served as board chairman of Kinnate Biopharma, Inc. until its sale in 2024 and as board director for ImmunoGen, Inc. until its sale in 2024. He served as executive board chairman for Covis Pharma Holdings until its sale in 2020 and board chairman of PaxVax Corporation until its sale in 2018. Mr. Mitchell previously held the position of president and chief executive officer at Lux Biosciences, Inc., a biotechnology company focusing on the treatment of ophthalmic diseases, at Alpharma, Inc., a publicly traded specialty pharmaceutical company until its acquisition by King Pharmaceuticals, Inc., and at Guilford Pharmaceuticals, Inc., a publicly traded pharmaceutical company focused on oncology and acute care, until its acquisition by MGI Pharma Inc. He previously held executive roles at Bristol Myers Squibb and GlaxoSmithKline. Mr. Mitchell earned a B.A. from City University London and a B.Sc. in biology from Coventry University. 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 and Instagram.

– 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