Pharma Focus Europe

ReCode Therapeutics Receives FDA Orphan Drug Designation for RCT1100 Targeting Primary Ciliary Dyskinesia

Friday, June 28, 2024

ReCode Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to RCT1100 for the treatment of primary ciliary dyskinesia (PCD). This genetic disease impairs respiratory function due to defective cilia, leading to chronic infections and respiratory decline, sometimes necessitating lung transplants. Mutations in the DNAI1 gene are responsible for approximately seven percent of PCD cases.

RCT1100 is an experimental therapy developed by ReCode, utilizing their Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform to deliver DNAI1 mRNA to airway cells. This mRNA is intended to produce the DNAI1 protein inside cells, aiming to restore proper ciliary function and address the underlying cause of the disease. Preclinical studies have demonstrated promising results in restoring ciliary function in relevant models of PCD.

Currently, ReCode is enrolling PCD patients in Part B of a Phase 1 study to assess the safety and tolerability of RCT1100 administered via nebulizer. The study is underway in the U.S. and UK, with updates expected upon completion of patient enrollment.

PCD affects an estimated 1 in 7,500 people globally, with many cases going undiagnosed due to limited screening. ReCode has launched, offering free genetic testing for eligible PCD patients, accessible through direct enrollment or healthcare professional referrals.



Thermo Fisher Scientific - mRNA ServicesWorld Orphan Drug Congress 2024World Vaccine Congress Europe 2024Advanced Therapies USA 2024