PHL-001

PHL-001 is a preclinical RNA-based gene therapy developed by Phlox Therapeutics for the treatment of cardiac laminopathy, specifically LMNA-related dilated cardiomyopathy with conduction disease (DCM-CD). The therapy is based on over 17 years of research from the Amsterdam University Medical Center and utilizes a 'smart RNA' design capable of targeting multiple mutations within the LMNA (Lamin A/C) gene simultaneously. This versatile approach is intended to treat a broad range of patients harboring different Lamin mutations without requiring mutation-specific drug candidates. Proof-of-concept has been demonstrated in patient-derived stem cell cardiomyocytes, showing selective therapeutic effects.

Other names

PHL001PHL-001PHL 001