Revolutionary Treatment: First Patients Receive RCT1100 in Clinical Trial to Combat Primary Ciliary Dyskinesia

ReCode Therapeutics, a private, clinical-stage genetic medicines company, is leading the way with the next wave of mRNA and gene correction therapeutics. Today, the first healthy volunteer participants were dosed in a Phase 1 clinical trial of RCT1100, a pioneering mRNA-based genetic medicine that has the potential to provide a transformative treatment for people with primary ciliary dyskinesia (PCD) caused by pathogenic mutations in the DNAI1 gene.

ReCode Therapeutics has taken a major step forward with the initiation of a clinical study of RCT1100, a drug developed using the Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform. This innovative platform enables precise delivery of genetic medicines to cells impacted by disease, bringing us closer to initiating a clinical trial in PCD patients. This is a significant milestone towards realizing the potential of extra-hepatic delivery of genetic medicines.

RCT1100 is an exciting, mRNA-based therapeutic that has the potential to revolutionize the treatment of PCD – a rare genetic disease that results in the loss of ciliary activity and causes chronic respiratory infections, bronchiectasis, and respiratory failure. Delivered directly into the airway using an optimized eFlow® Nebulizer System (PARI), this innovative drug is formulated with ReCode’s proprietary SORT LNP delivery platform and is designed to lead to DNAI1 protein production, thereby restoring ciliary function. Recent preclinical studies presented at the American Thoracic Society (ATS) 2022 International Conference have demonstrated the promising mechanism of action of RCT1100 in vitro and in vivo, offering hope for a disease-modifying treatment for PCD in the near future.

We are thrilled to take the next step in providing a first-in-class, precision genetic medicine for the treatment of PCD, a genetic disease with a serious impact on respiratory health. After years of design and optimization of the DNAI1 mRNA, as well as conducting pharmacology and toxicology studies to support dosing in humans, our SORT LNP delivery platform has enabled us to translate the potential of genetic medicines into clinical outcomes, with the goal of altering the course of disease for those living with rare genetic disorders.

ReCode is conducting a groundbreaking Phase 1 clinical study in New Zealand to assess the safety and tolerability of RCT1100, an inhaled treatment administered via nebulizer. This double-blind, placebo-controlled, first-in-human study will include 32 healthy adults who will receive a single dose of either placebo or RCT1100. Be a part of this exciting research and join the cutting edge of medical advancement!

ReCode is aiming to make a difference in the lives of those suffering from cystic fibrosis and primary ciliary dyskinesia. In the second half of 2023, the company plans to submit Investigational New Drug applications (INDs) to the U.S. Food and Drug Administration for clinical trials of RCT1100 for PCD patients and a CFTR mRNA therapeutic for cystic fibrosis sufferers. This inherited life-threatening genetic disorder affects around 100,000 people worldwide and, if successful, could be a game-changer for those living with the condition.

About Primary Ciliary Dyskinesia

PCD is a rare and progressive genetic disorder, affecting an estimated 87,000 people across North America and Europe, and even more worldwide. It is characterized by dysfunctional cilia, leading to a loss of mucociliary clearance (MCC) and a myriad of chronic respiratory problems, such as respiratory tract infections, bronchiectasis, and declining respiratory function. Unfortunately, there is no cure for PCD, and its effects will only worsen over time.

About ReCode Therapeutics

ReCode Therapeutics is revolutionizing the field of genetic medicines with its innovative platform – Selective Organ Targeting (SORT) Lipid Nanoparticle (LNP). This next-generation technology enables precise delivery of messenger RNA (mRNA) and gene correction therapeutics to target organs and cells beyond the liver. Through its SORT LNP platform and nucleic acid technologies, ReCode is developing a pipeline of disease-modifying mRNA and gene correction therapeutics with the aim of revolutionizing the treatment of primary ciliary dyskinesia and cystic fibrosis. In 2022, ReCode was named among Fierce Biotech’s “Fierce 15” and was highlighted by Nature as one of the “Seven Technologies to Watch in 2022” for its groundbreaking work.

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