At pacDNA, we aim to transform the field of oligonucleotide therapeutics with safer and more potent drugs that distribute to a wide range of non-liver organs and tissues such as the heart, blood, lung, skeletal muscle, skin, and bone. Our mission is to greatly expand the disease areas where an oligonucleotide-based therapy can be developed, to include major disease categories such as cancer, immunology, metabolic disease, and infectious diseases.

Our proprietary delivery technology, termed the Brushield™, boosts the biodistribution in otherwise hard-to-deliver sites, which makes it possible to develop multiple therapies for diseases that are currently out of reach. The technology also promotes tissue retention to up to several months, producing lasting potency. With our technology, there is potential to yield much better treatments, with very low toxicity or side effects from the carrier, increased potency, and smaller, less frequent dosages.

Conventional gene regulation agents face a difficult dilemma. The combined challenges of poor tissue accumulation and rapid clearance of agents by the liver and kidney require larger doses of agents. Increased doses lead to toxicity and inflammatory or immune responses. Collectively, these challenges limit the therapeutic window between a therapy that is efficacious and one that is toxic, increase the costs to patients, and very often limit the places in the body where effective therapies can be developed.

What is Brushield™? It is a bottlebrush polymer-based vector technology that can provide entropic shielding to the conjugated oligonucleotide. The technology mitigates toxicities and almost all other side effects stemming from unwanted nucleic acid-protein interactions, while making the active component more efficacious. The bottlebrush architecture is fundamentally different from the conventional linear polymer and exhibits far superior biopharmaceutical properties including reduced side effects, non-immunogencity, enhanced plasma pharmacokinetics, and non-liver/kidney biodistribution. Our research has generated substantial data pointing to Brushield™ conjugates being a highly translatable approach for realizing non-hepatic gene regulation, which remains a key challenge in the development of viable therapeutics.