ReCode begins subject dosing in primary ciliary dyskinesia therapy trial

ReCode Therapeutics has dosed the first healthy volunteer participants in a Phase I clinical trial of the disease-modifying mRNA-based genetic medicine, RCT1100, to treat primary ciliary dyskinesia (PCD), which is caused by pathogenic mutations in the DNAI1 gene.

The first-in-human, placebo-controlled, double-blind Phase I trial has been designed for evaluating the tolerability and safety of a single ascending dose of inhaled RCT1100 given through a nebuliser.

The company expects to enrol nearly 32 healthy adult participants, who will receive a single dose of either placebo or RCT1100 in the trial, which is being conducted in New Zealand.

It intends to submit an Investigational New Drug application (IND) for the Phase I trial to the US Food and Drug Administration (FDA) in the second half of this year.

ReCode Therapeutics is also planning to submit another IND for its CFTR mRNA therapeutic to treat cystic fibrosis, which is an inherited, life-threatening genetic disorder.

ReCode Therapeutics chief scientific officer and president David Lockhart said: “PCD is a genetic disease, with a serious impact on respiratory health, that significantly affects quality of life and currently has no approved treatments.

“We are excited to take this next step toward a first-in-class, precision genetic medicine for the treatment of people living with PCD.

“This programme was the result of many years of work, beginning with the design and optimisation of the DNAI1 mRNA, and continuing all the way through pharmacology and toxicology studies to support dosing in humans.”

RCT1100 has been formulated using the SORT LNP delivery platform of the company.

It is nebulised and given as an aerosol into the airway directly using an optimised eFlow Nebulizer System (PARI).

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

Free Whitepaper

Optimise your cell therapy process: a guide to cell thawing

Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies.

Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell...

Read more

Thank you.

You will receive an email shortly. Please check your inbox to download the Whitepaper.

By Cytiva Thematic

You have a right to withdraw your consent at any time, by clicking here. We may still continue to send you service-related and other non-promotional communications. For more information relating to our privacy practices, we invite you to review our privacy policy.

Sign up for our daily news round-up!

Give your business an edge with our leading industry insights.

Sign up