ReCode Therapeutics

ReCode Therapeutics is a genetic medicines company developing treatments for genetically defined diseases, particularly rare conditions with no existing treatment options, including cystic fibrosis. Its platform combines optimized messenger RNA (mRNA), gene correction and selective organ targeting to deliver genetic medicines to specific organs, tissues and cells beyond the liver. Designed to support a range of therapeutic cargoes and routes of administration, the technology includes a modular lipid nanoparticle (LNP) platform and an inhaled LNP approach targeting cells in the lungs.

ReCode’s pipeline focuses on rare disease therapeutics with the potential for repeat dosing. The company reports clinical proof of activity for its inhaled platform in primary ciliary dyskinesia: in the RCT1100 Phase 1b study, bronchoscopy confirmed restoration of protein and ciliary activity in the airway, and 57% of patients achieved meaningful improvement in mucociliary clearance at 12 weeks. Preclinical research on RCT2100 has also shown improved mucociliary clearance in cystic fibrosis ferrets after one administration.

ReCode offers partnering opportunities and shares research, pipeline and clinical development updates