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Therapy-resistant heart failure
Today, therapies prescribed for heart failure have successfully improved symptoms and survival for most patients.
However, in a minority of patients, these evidence-based treatments do not halt the progression of the disease, which leaves them with therapy-resistant heart failure. Ironically, this most often concerns young patients with heritable forms of heart failure, including cardiac laminopathies.
Cardiac laminopathies are a distinct and often aggressive form of heart failure, caused by mutations in the Lamin gene. Mutations in the Lamin gene (LMNA) cause a diverse range of diseases, including dilated cardiomyopathy. LMNA-dilated cardiomyopathy is associated with conduction disease (DCM-CD) and is a severe and aggressive form of DCM associated with a poor prognosis and high rate of sudden death and early ventricular arrhythmia. no specific treatment is currently available for the patients.
We are striving to cure these cardiomyopathies by developing gene therapies that alleviate harmful protein products intracellularly. Gene therapy is a promising therapeutic platform because it targets disease-causing genes in a sequence-specific manner, which enables more precise and personalised treatment of diverse life-threatening diseases. By introducing a therapeutic specifically to the heart, gene expression can be downregulated, augmented, or corrected.
PHL-001 is our first innovative RNA-based therapeutic product and lead candidate in the pre-clinical phase.
The technology and design of this therapeutic gene products are based on over 17 years of research and developed in the lab of our CMO, Prof. Yigal M. Pinto, MD at the Amsterdam University Medical Center. Phlox’s technology uses smart RNA-designs that allow for parallel targeting of multiple Lamin mutations. The versatile nature of these RNA-designs, developed by the Amsterdam UMC team, 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.
We are advancing a novel class of cardiac therapeutics with a unique mode of action for unique patients. Next to mutations in Lamin A/C, Phlox Therapeutics aims to apply its smart designs to adjacent treatment-resistant cardiomyopathies. The restorative nature of our approach at the cellular level enables us to find a cure for patient currently on palliative care. The medical need for them is as clear as day. Therefore, we will continue to develop and expand our therapeutic pipeline independently and together with partners.