ReCode Therapeutics Gets FDA Orphan Drug Status for RCT1100 to Treat Primary Ciliary Dyskinesia

ReCode Therapeutics, based in MENLO PARK, Calif., has announced that its investigational therapy RCT1100 has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of primary ciliary dyskinesia (PCD). RCT1100 is specifically developed to target PCD cases caused by mutations in the DNAI1 gene.

PCD is a rare genetic disorder that affects the function of cilia, the tiny hairlike structures in the respiratory tract responsible for moving mucus out of the airways. When cilia do not function properly, mucus accumulates, trapping bacteria and viruses, which leads to chronic respiratory infections and reduced lung function. In severe cases, some patients may even require lung transplants. About seven percent of PCD cases are linked to mutations in the DNAI1 gene.

According to Dr. Shehnaaz Suliman, CEO of ReCode Therapeutics, there are currently no FDA-approved treatments for PCD, making this designation a significant milestone for those affected by the disease. ReCode is actively enrolling patients across the U.S. and UK in a study focused on the safety and tolerability of RCT1100, with results to be shared upon completion of enrollment.

The development of RCT1100 is powered by ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform. This platform allows the inhaled mRNA therapy to be delivered directly to airway cells. Once inside the cells, the DNAI1 mRNA is translated into the DNAI1 protein, aiming to restore proper ciliary function by addressing the root cause of the disease. Preclinical studies have shown that the DNAI1 mRNA can efficiently produce the DNAI1 protein and restore ciliary function in relevant PCD models.

Currently, ReCode is conducting Part B of a Phase 1, open-label study to evaluate the safety and tolerability of a single ascending dose of RCT1100 delivered via a nebulizer to PCD patients with DNAI1 gene mutations.

PCD is characterized by deficient mucociliary clearance (MCC), leading to chronic respiratory infections, bronchiectasis, and a decline in respiratory function over time. More than 50 different gene mutations can cause dysfunctional cilia and loss of MCC. PCD is a progressive disease with no available cure or disease-modifying treatments. It affects approximately 1 in 7,500 people across various ethnicities, with around 45,000 patients in the U.S. Due to inadequate screening, it is estimated that 80% of PCD cases remain undiagnosed.

To aid in diagnosis, ReCode has launched ThinkPCD.com, a program offering genetic testing at no cost to eligible PCD patients. Patients or their healthcare providers can visit the website to determine eligibility and enroll in the testing program.

ReCode Therapeutics is at the forefront of developing genetic medicines using precision delivery techniques. Their SORT LNP platform allows for highly targeted delivery of genetic treatments to specific organs, tissues, and cells. In addition to RCT1100 for PCD, ReCode is developing RCT2100 to treat cystic fibrosis patients with specific CFTR gene mutations that do not respond to existing CFTR modulators. Both therapies are inhaled and utilize the SORT LNP delivery system. ReCode aims to expand its pipeline to include treatments for other rare and common genetic diseases, such as those affecting the musculoskeletal and central nervous systems, liver, and infectious diseases.

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