SEATTLE, Nov. 11, 2015 /PRNewswire/ -- PhaseRx, Inc., a leading biotechnology company focused on developing mRNA therapeutics to treat orphan liver diseases, today announced it will be presenting data on its mRNA Technology Platform at the 3rd International mRNA Health Conference Berlin on Wednesday, November 11, 2015. The presentation entitled "Targeted mRNA Delivery to the Liver and Therapeutic Efficacy in an Orphan Liver Disease Model" highlights PhaseRx's recent advances in the delivery of mRNA therapeutics. In particular, the company will present therapeutic mRNA efficacy of its lead formulation establishing normalization of a key clinical and approvable biomarker in a well-accepted orphan liver disease model.
Through the application of its mRNA Technology Platform, PhaseRx has demonstrated highly effective and selective expression to liver with multiple mRNAs, with no expression observed in other tissues. Moreover, the formulations are well-tolerated enabling dosing up to 10 mg/kg mRNA with efficacy demonstrated at levels 100-fold lower. In addition to a wide therapeutic index, the formulations can be repeat-dosed without loss of activity, a major hurdle in the field. The ability to maintain activity under repeat dosing regimens enables access to a number of chronic, liver-specific conditions. In fact, PhaseRx's first therapeutic indication is a single-gene disorder in the liver for which there are no curative therapeutics on the market.
"The PhaseRx mRNA Technology Platform is a targeted delivery system designed to specifically deliver mRNA molecules into cells. It is a natural extension of our highly efficacious and well tolerated SMARTT Polymer Technology® with applications beyond mRNA-based replacement therapies, including the delivery of gene editing-based therapeutics as well as other nucleic acid-based therapeutic modalities. We are pleased to have successfully demonstrated the ability of this innovative system to specifically and actively target delivery of mRNA to hepatocytes and mediate efficacy in preclinical disease models," said Robert Overell, Ph.D., President and Chief Executive Officer at PhaseRx. "Furthermore, we are excited to advance this technology into clinical development for orphan liver diseases and potentially address a large unmet medical need for patients."
PhaseRx mRNA Technology Platform
PhaseRx's mRNA Technology Platform is designed to address the challenges of RNA delivery through protection of the mRNA in the systemic circulation, specifically targeting hepatocytes and by mediating endosome escape and mRNA delivery into cytoplasm. The platform has been developed into an industrialized system that provides robust and scalable manufacture that is well-tolerated with minimal induction of innate immunity. The system has demonstrated delivery of a wide range of therapeutic mRNAs to the liver including mRNAs to both cytosolic and secreted proteins.
Unmet Medical Need in Orphan Liver Diseases
There are a large number of inherited metabolic diseases of the liver that are caused by single-gene defects. These genetic deficiencies, or inborn errors in metabolism, are orphan diseases that have severe consequences for patients. The few available drug therapies are often palliative and have significant shortcomings. In many cases, the only cure for these diseases is allogeneic liver transplantation.
PhaseRx is a privately held RNA therapeutics company developing treatments for orphan liver disease. The Company is utilizing its proprietary mRNA Technology Platform, which offers the ability to deliver messenger RNA therapeutics predictably to selected tissues in vivo, thereby unlocking the value of mRNA as a new therapeutic modality. PhaseRx is headquartered in Seattle, Wash. For more information, visit www.phaserx.com.
SOURCE PhaseRx, Inc.