RCT2100 / ReCode Therap - LARVOL DELTA

FDA clears next phase of RCT2100 clinical trial for cystic fibrosis (Cystic Fibrosis News Today) - "This portion of the Phase 2 trial (NCT06237335) will test RCT2100 together with Kalydeco (ivacaftor) in people with CF who are either not eligible for CFTR modulators or are not taking them. Enrollment is underway at sites in the U.S., with study locations in the European Union and U.K. expected to open in early 2026."

IND • Trial status • Cystic Fibrosis

A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF (clinicaltrials.gov) - P2 | N=192 | Recruiting | Sponsor: ReCode Therapeutics | Phase classification: P1 ➔ P2 | Trial completion date: Mar 2026 ➔ Dec 2026 | Trial primary completion date: Dec 2025 ➔ Aug 2026

Phase classification • Trial completion date • Trial primary completion date • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT2100 for the Treatment of Cystic Fibrosis (Businesswire) - "ReCode Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for RCT2100, a novel, investigational mRNA therapy being developed to treat cystic fibrosis (CF)."

Orphan drug • Cystic Fibrosis

A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants and in Participants With CF (clinicaltrials.gov) - P1 | N=158 | Recruiting | Sponsor: ReCode Therapeutics | N=100 ➔ 158 | Trial completion date: Dec 2025 ➔ Mar 2026 | Trial primary completion date: Apr 2025 ➔ Dec 2025

Enrollment change • Trial completion date • Trial primary completion date • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

ReCode Therapeutics Announces Additional Funding From the Cystic Fibrosis Foundation to Accelerate Development of Novel Gene Correction Therapeutics to Treat Cystic Fibrosis (Businesswire) - "The Cystic Fibrosis Foundation has agreed to invest up to $15 million in new funding to support the company’s gene correction development program with Intellia Therapeutics for the treatment of cystic fibrosis....ReCode 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 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."

Financing • Cystic Fibrosis

RCT2100 rescue of CFTR function in CF patient human bronchial epithelial cells and mucociliary clearance in CF ferrets (NACFC 2024) - " When dosed apically in HBECs, we show clear dose response for rescue of CFTR-mediated chloride transport function in F508del/F508del HBECs treated with RCT2100 and efficacy approaching 70% of elexacaftor/ tezacaftor/ivacaftor (VX-661/VX-450/VX-770) levels after a single dose. Overall, these preclinical data support a potential therapeutic approach to address a significant fraction of the patient population that does not benefit from current CFTR modulator therapy."

Clinical • Pulmonary Disease • Respiratory Diseases

ReCode Therapeutics Presents Preclinical Data from its Cystic Fibrosis Program at the 2024 North American Cystic Fibrosis Conference (NACFC) in Boston (Businesswire) - "ReCode Therapeutics...presented preclinical data from its mRNA-based cystic fibrosis (CF) program at the North American Cystic Fibrosis Conference (NACFC)....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."

Preclinical • Cystic Fibrosis

ReCode Therapeutics Doses First Patient in Phase 1b Clinical Study of RCT2100 for the Treatment of Cystic Fibrosis (Businesswire) - "ReCode 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)....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."

Trial status • Cystic Fibrosis

A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants and in Participants With CF (clinicaltrials.gov) - P1 | N=100 | Recruiting | Sponsor: ReCode Therapeutics | Phase classification: P1/2 ➔ P1 | N=32 ➔ 100 | Trial primary completion date: Sep 2024 ➔ Apr 2025

Enrollment change • Phase classification • Trial primary completion date • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants (clinicaltrials.gov) - P1/2 | N=32 | Recruiting | Sponsor: ReCode Therapeutics | Phase classification: P1 ➔ P1/2 | Trial completion date: Aug 2024 ➔ Dec 2025 | Trial primary completion date: May 2024 ➔ Sep 2024

Phase classification • Trial completion date • Trial primary completion date • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

RCT2100 rescues CFTR function in human bronchial epithelial cells and improves mucociliary clearance in CF ferrets (ECFS 2024) - "Administration of RCT2100 to CF ferret (Sun et al ., 2019) trachea confirmed that it could penetrate mucus, transfect the epithelium, and significantly improve mucociliary clearance (MCC) after a single administration. This preclinical data supports a potential therapeutic approach to address a significant fraction of the patient population that does not benefit from current CFTR modulator therapy."

CFTR

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 (Businesswire) - "ReCode Therapeutics....today announced that the first cohort of healthy volunteer participants have been dosed in a Phase 1 clinical study of RCT2100....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....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."

IND • Trial status • Cystic Fibrosis

A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants (clinicaltrials.gov) - P1 | N=32 | Recruiting | Sponsor: ReCode Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

A Phase 1, Single-Center Study Evaluating the Safety and Tolerability of RCT2100 in Healthy Participants (clinicaltrials.gov) - P1 | N=32 | Not yet recruiting | Sponsor: ReCode Therapeutics

New P1 trial • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases