Multifunctional Antisense Therapeutics Nanocarrier and Inhalation Device

To address the CBD need for improved formulations to enhance the ease of use and bioavailability of antisense therapeutics, Physical Optics Corporation (POC) proposes to develop a Multifunctional Antisense Therapeutics Nanocarrier and Inhalation Device (MATEN). This proposed system is based on a new dry powder formulation and a novel design of an inhaler that uses in-house developed mature components. POC’s innovations in the nanocarrier (NC) construct and in the design of the inhaler, which is capable of delivering a single dose from the MATEN during one deep and slow inspiration, will enhance the bioavailability for antisense therapeutics that is necessary for the prophylaxes and early treatment of warfighters against emerging bio-warfare threats. As a result, this technology offers a formulation that is stable at ambient temperature, minimizes the potential for introduction of immunogenic materials and the bulk and complexity of dispensing, and which directly addresses the requirements of the Transformational Medical Technologies Initiative (TMTI) program. In Phase I, POC will demonstrate the feasibility of the MATEN by demonstrating product stability and establishing enhanced bioavailability in an animal model. In Phase II, POC will optimize candidate formulation for maximum bioavailability and conduct initial pharmacokinetic and pharmacodynamic studies in an animal model.