Platform

Using intracellular enzyme replacement therapy (i-ERT).  This i-ERT approach is enabled by our proprietary Hybrid mRNA TechnologyTM platform, which allows synthesis of the missing enzyme inside the cell

PhaseRx’s groundbreaking Hybrid mRNA TechnologyTM platform Our i-ERT approach is accomplished by delivering normal copies of the mRNA that make the missing enzyme inside the liver cell, thereby reinstating the normal physiology and correcting the disease. A key challenge with mRNA therapeutics historically has been their satisfactory delivery into the patients’ cells.

Our Hybrid mRNA Technology provides a differentiated polymer-LNP-based formulation approach for the delivery of mRNA into the hepatocytes in the liver. The Hybrid mRNA Technology utilizes our SMARTT Polymer Technology in combination with an inert LNP which functions as a carrier for the mRNA. The LNP encapsulates and protects the mRNA while it transits the blood and is taken up into the hepatocytes while the polymer delivers mRNAs into the cytoplasm by mediating their escape from endosomes. The synthetic polymers exploit a proprietary mechanism to effect passage of mRNA molecules across the endosomal membrane.

We are focused on inherited, single-gene disorders of metabolism in the liver that result in deficiency of an intracellular enzyme and thus have been unable to be treated with conventional ERT. Our approach is to deliver mRNA encoding the missing enzyme into the cell using our Hybrid mRNA Technology, such that the mRNA makes the missing enzyme inside the cell, restores the intracellular enzyme function and corrects the disease.

pipeline-tech-1

Our Hybrid mRNA Technology has been shown in our internal preclinical studies to result in synthesis of intended proteins in hepatocytes with a fast onset of action, suggesting highly effective delivery of mRNA molecules, and the synthesis of a number of protein classes including cytosolic proteins, mitochondrial proteins and secreted proteins. By developing a rapid in vivo-based screening program, we have gained valuable information about the structure-activity relationships of formulation components.

Benefits of PhaseRx Hybrid mRNA Technology*:

  • Results in high-level synthesis of the desired protein in the hepatocyte
  • Improved tolerability
  • Ability to repeat-dose without loss of effectiveness, thus enabling treatment of chronic conditions.

 

*based in internal preclinical studies