ReCode Therapeutics Begins Phase 1b Study of RCT2100 for Cystic Fibrosis

ReCode Therapeutics has begun dosing the first patient in a Phase 1b clinical trial for RCT2100, an experimental inhaled mRNA therapy aimed at treating cystic fibrosis (CF). CF, a genetic disorder caused by mutations in the CFTR gene, affects about 130,000 individuals globally. While CFTR modulator treatments have significantly improved outcomes for many CF patients, those with specific mutations that inhibit the production of functional CFTR protein remain underserved.

Dr. Shehnaaz Suliman, CEO of ReCode Therapeutics, emphasized the importance of this milestone, noting the potential to provide a new treatment option for those who do not benefit from existing therapies. The company is committed to rapid enrollment to expedite bringing this therapy to those in need.

RCT2100 utilizes ReCode's proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform, which is designed to deliver CFTR mRNA directly to lung cells. This approach aims to enable these cells to produce functional CFTR protein, addressing the root cause of CF rather than merely managing symptoms. By restoring functional CFTR protein, the therapy has the potential to significantly improve the quality of life for CF patients.

The Phase 1b study of RCT2100 has received regulatory approval in the United States, United Kingdom, and Europe. Initial enrollment is underway in the Netherlands, with plans for expansion to the U.S., UK, and France.

Cystic fibrosis is a progressive genetic disorder characterized by persistent lung infections and respiratory failure. The disease is caused by mutations in the CFTR gene, which lead to the production of faulty or absent CFTR protein. This protein deficiency results in poor airway hydration, causing thick mucus buildup in the lungs and leading to recurrent infections, inflammation, and respiratory failure. Despite advancements in CFTR modulator therapies, about 10% of CF patients possess genetic mutations that render these treatments ineffective.

ReCode Therapeutics specializes in precision delivery of genetic medicines, leveraging its SORT LNP platform to target genetic treatments to specific organs, tissues, and cells. The company's leading programs include RCT2100 for CF patients with non-responsive CFTR mutations and RCT1100, aimed at treating primary ciliary dyskinesia caused by mutations in the DNAI1 gene. Both therapies are inhaled, disease-modifying mRNA treatments developed using the SORT LNP platform. ReCode is also working to broaden its pipeline to address other rare and common genetic disorders, including those affecting the musculoskeletal system, central nervous system, liver, and infectious diseases.

ReCode has been recognized as a top workplace by the San Francisco Business Times and Silicon Valley Business Journal. The company's efforts in developing advanced genetic therapies aim to bridge gaps in treatment for patients with genetic conditions that currently lack effective solutions.