Background

RNA therapeutics have shown great potential in treating many types of disease. Small interfering RNAs (siRNAs) are viewed as the most promising type of RNA-based therapeutic oligonucleotide drug, since their mechanism of action is catalytic and each siRNA molecule can inactivate several target RNA molecules in a sequence-specific manner.

However, the promise of oligonucleotide therapeutics has not yet been fully realized. There remain a number of biological barriers, which include: siRNA capture by immune cells, cleavage by serum ribonucleases, siRNA filtration by the kidneys, and the endothelial barrier. Additionally, chemical modification and use of carriers to overcome these barriers often lead to adverse effects.

Technology Overview

Northeastern spinout, pacDNA, has developed a new platform technology that uniquely provides oligonucleotides with improved biopharmaceutical properties, enhanced transfection efficiency, and an unconventional biodistribution profile. pacDNA also reduces many forms of adverse effects while improving drug potency.

While current oligonucleotide drugs focus on disease in the liver or those that can be addressed by local injection, such as the spinal cord or eye, pacDNA can deliver oligonucleotides throughout the body, including major organs, muscle, and skin.

pacDNA’s drug delivery platform is based on the production of a polymer-RNA conjugate consisting of a brush form of the noncationic, biocompatible polymer, polyethylene glycol. The densely packed PEG brush provides the embedded RNA strands with enhanced nuclease stability and improved cellular uptake.

Benefits

Prolonged tissue retention
Long plasma pharmacokinetics
Reduced side effects/immune system activation
Can use natural DNA or RNA
Safe chemical composition
Increased bioactivity
Broadens use for many disease areas

Applications

Therapeutics for a range of diseases and disorders
Protein replacement therapy

Opportunity

Connecting with prospective investors
Identifying space for company to work off-campus

Oligonucleotide Delivery Platform for Improved Nucleic Acid-based Treatments

IP Status

Seeking