PHL003

PHL003 is a preclinical-stage gene therapy being developed by Phlox Therapeutics for the treatment of laminopathies, a group of rare genetic disorders caused by mutations in the *LMNA* gene. The program specifically targets laminopathies associated with a dominant-negative (DN) mechanism, where the mutant Lamin A/C protein interferes with the function of the wild-type protein. PHL003 is part of a pipeline of RNA-based and gene therapy assets designed to address the underlying genetic causes of cardiomyopathy and other lamin-related diseases. As a follow-on program to the lead candidate PHL002, PHL003 aims to restore proper lamin A/C function or mitigate the toxic effects of the mutant protein. The specific molecular mechanism and delivery modality remain largely undisclosed, though it is positioned within the company's gene therapy portfolio.