Pipeline

We are building a pipeline covering the whole segment of cardiac laminopathies 
PHL002 - Lead
PHL002 is a gene replacement therapy designed to restore functional Lamin expression in patients with LMNA-related dilated cardiomyopathy. By targeting the underlying genetic defect, PHL002 aims to slow disease progression and improve cardiac function. Preclinical studies have shown improved survival and disease outcomes, supporting its potential as a disease-modifying therapy for a large subset of LMNA DCM patients who currently have no targeted treatment options.
PHL001 - Follow on
PHL001 is an RNA-based therapeutic that allows for parallel targeting of multiple Lamin mutations. The versatile nature of these RNA-designs prevents the need for generating mutation specific compounds to treat each unique mutation, making these compounds efficacious for the majority of patients harboring a Lamin mutation. Proof-of-concept experiments show that these compounds can induce highly selective and favorable therapeutic effects in human stem cell-derived cardiomyocytes from patients suffering from laminopathy.
PHL003 - Follow on
Undisclosed