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PHL-001
- Development stage
- Preclinical
- Lead developer
- Phlox Therapeutics
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
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
02
Targets
LMNA (Lamin A)
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