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New Phase 1 Clinical Trial for PCD

Apr 13, 2024

Recode Therapeutics has announced its First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Trial for the treatment of Primary Ciliary Dyskinesia. 

A Please 1 trial to evaluate safety and tolerability of a single ascending dose of RCT1100, an inhaled genetic medicine, has been planned for an initial roll out in 2023. The study is being conducted in New Zealand by Recode Therapeutics, a genetic medicines company that is powering the next wave of messenger RNA (mRNA) and gene correction therapeutics. The trial is expected to enrol approximately 32 healthy adults who will receive a single dose of either placebo or RCT1100. 

This trial will be a step towards a precision genetic medicine for the treatment of people living with PCD, of which there are currently no approved treatments. Patients with PCD suffer from chronic respiratory infections and bronchiectasis, often developing respiratory failure. 

RCT1100 is designed to be a disease-modifying mRNA-based therapeutic for PCD and will be delivered as an aerosol directly into the airway using an optomised eFlow® Nebulizer System (PARI). The Phase 1 double-blind, placebo-controlled, first-in-human study of RCT1100 is designed to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered via nebulizer. The hope is that RCT1100 will restore ciliary function in the lungs of people with PCD who have mutations in the DNAI1 gene. 

The programme is the result of many years of work and offers the opportunity to alter the course of disease for many people living with rare genetic disorders. 

More information about the trial is available here: ReCode Therapeutics Press Release