ReCode Therapeutics

The page lists a clinical study for people with CF who have specific CFTR mutations and Phase 1 and Phase 2 studies evaluating RCT2100’s safety and tolerability.

Cystic fibrosis (CF) is a progressive, fatal genetic disease caused by mutations in the CFTR gene and affects approximately 105,000 people worldwide.

ReCode is developing an inhaled mRNA therapy for people with CF who have CFTR mutations and do not respond to currently approved CFTR modulators; this group represents 10% of people with CF.

The company’s indications include primary ciliary dyskinesia (PCD), cystic fibrosis (CF), and liver, spleen, and central nervous system (CNS) diseases.

The Cystic Fibrosis Foundation has agreed to invest up to $15 million in new funding to support the company’s gene correction development program with Intellia Therapeutics for the treatment of cystic fibrosis

Cystic Fibrosis Foundation provides $3 million in new funding to accelerate development of inhaled mRNA therapy for cystic fibrosis. Research collaboration with Praxis Precision Medicines to develop LNP formulations for ASO delivery.

The company says its leadership team has expertise in genetic medicines, orphan drug development, and technology development from small and large biopharma companies.

The company says its mRNA and gene correction therapeutics have the potential to help people with genetic diseases benefit from genetic medicines.

ReCode’s platform enables targeted delivery to organs, tissues, and cells beyond the liver. Its rare disease pipeline uses optimized mRNA and gene correction therapies with redosing capability.

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