Aphios Granted US Patent for Improved Delivery of Targeted siRNA Using Nanotechnology

Aphios Corporation recently announced it has been granted United States Patent No. 9,981,238 titled Apparatus and Methods for Making Nanosomes Loaded with Nucleic Acids. 

The present invention pertains to the nanoencapsulation of small interfering ribonucleic acids (siRNAs) and other biologics in phospholipid nanosomes for improved delivery to targeted diseased human or animal organs and cells. siRNAs are small double stranded RNA, usually 20 to 25 nucleotides in length which bind to other nucleic acids and interfere with or silence disease expression events, and have the potential to prevent, treat and cure chronic diseases such as cancers and HIV. Nanosomes, small uniform liposomes having diameters measured in nanometers, present a novel frontier for vehicle-free delivery (VFD) of siRNA and alternative biologics.

There is wide consensus that non-viral delivery of RNA has intersecting positive attributes. Progress has been made in chemical-based delivery strategies using liposomes, molecular-sized chemical conjugates, and supramolecular nanocarriers. However, nucleic acids are relatively large, negatively charged polymers, and present significant clinical challenges from the standpoint of delivery to cells. Thus, even though RNA-based therapeutics hold great promise for disease prevention, treatment and cure, significant delivery and stability-related obstacles still need to be overcome to hasten clinical development.

Dr. Trevor P. Castor, the company’s President and CEO and inventor of the technology, explains, “Encapsulation in nanosomes provides a delivery matrix to bypass impediments like product degradation due to instability, safety concerns due to replicative risks of viral delivery vectors, toxicity associated with larger dosing for desired effect, and limited potency due to restricted tissue distribution. Moreover, nanosomal siRNA has the greater potential to hasten scale-up of on-demand manufacturing can be delivered directly into the cytoplasm where gene expression occurs without the need for nuclear localization and can interact directly with host innate defense system to stimulate or regulate specific outcomes.”

Dr. Castor continues, “Encapsulation of siRNA molecules in smaller widely distributed nanosomes represent a valuable pharmacological innovation opportunity to facilitate product discovery and efficacy evaluation that will revolutionize disease therapeutics, prevention, and cure. Nanosomal siRNA (nanosiRNA) has the increased potential to penetrate tissue barriers to confer potent silencing of specifically targeted genes that promote infection and diseases. These RNA nanosomes can be effective at nano concentrations that mitigate systemic toxicities. Pegylation is introduced during encapsulation and manufacturing to increase nanosiRNA biological residence time, therapeutic efficacy and overall therapeutic index.”

Aphios Corporation (www.aphios.com) is a clinical-stage biotechnology company developing green enabling technology platforms for improving drug discovery, manufacturing, delivery, and safety, and enhanced natural therapeutics for health maintenance and disease prevention, and the treatment of cancers and supportive care, infectious diseases such as HIV, and CNS disorders such as Alzheimer’s disease and Multiple Sclerosis.