ReCode Therapeutics Announces First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Study of Inhaled mRNA-Based Genetic Medicine, RCT2100, for the Treatment of Cystic Fibrosis

February 21, 2024 |

4 min read

ReCode 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.

– 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, 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 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.