ReCode Therapeutics RCT1100, an inhaled disease-modifying mRNA-based investigational therapy
ReCode Therapeutics is a clinical-stage genetic medicines company that utilizes precision delivery to usher in the next era of mRNA and gene correction therapeutics.
Their Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform facilitates highly precise and targeted delivery of genetic medicines directly to the organs and cells involved in disease, resulting in enhanced efficacy and potency.
Primary ciliary dyskinesia (PCD) is a rare genetic disorder that affects the cilia, tiny hair-like structures that line the respiratory tract and other organs. Cilia help to clear mucus and debris from the lungs and other organs, and in PCD, the cilia are unable to function properly, leading to a build-up of mucus and recurrent infections.
Primary ciliary dyskinesia (PCD) is a rare genetic disorder that affects the cilia, tiny hair-like structures that line the respiratory tract and other organs.
PCD caused by inherited mutations in more than 40 different genes. Currently, there are no disease-modifying therapies available, and treatments are limited to palliative care for the management of symptoms. ReCode is developing RCT1100, an inhaled disease-modifying mRNA-based investigational therapy for the treatment of PCD caused by mutations in DNAI1, an essential gene for ciliary movement.On March 1 2023, announced the first healthy volunteer participants had been dosed in a Phase 1 clinical trial of RCT1100. In the press release, the company stated that 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 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 RCT1100.
RCT1100 has the potential to be a transformative treatment for patients with PCD and could provide a potentially curative treatment for this serious disease.
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