Genetic medicines have enormous potential to treat hereditary diseases. More than 1800 mutations have been identified to cause hereditary diseases, and more are likely to be discovered in the future. Once the mutations are identified, researchers can work to find genetic treatments for those diseases.
However, the big challenge with genetic medicines is delivery. It is difficult to precisely deliver molecules to the target site and specific cells in the body safely and efficiently. There are also many different types of intracellular and extracellular barriers that treatments must be able to surpass.
Excitingly, the recent success of mRNA-based COVID vaccines has energized the field of genetic medicines because they can surpass some of these challenges. Now, the experimental concept of RNA medicines has the potential to be translated into clinically viable drugs and vaccines.
Professor Mansoor Amiji and his team at The Laboratory for Biomaterials and Advanced Nano-Delivery Systems (BANDS) are part of this exciting movement around nucleic acid therapies. BANDS is focusing on the development of Northeastern University’s patented multi-compartmental (MCP) formulations for oral RNA delivery for therapeutics and vaccines.
Specifically, their research currently focuses on oral vaccination and therapeutics for diseases of the gastro-intestinal tract, such as inflammatory bowel disease and celiac disease. This research has earned them selection as one of the Spring 2022 Spark Fund awardees.
MCP Oral Nucleic Acid Delivery Technology
Oral administration is the most convenient and patient-friendly route of drug and vaccine administration in the body. Therefore, the MCP oral nucleic acid delivery systems are an oral administration platform that uses polymers and lipids for nanoparticle and microsphere formulations, affording tremendous versatility in MCP design. MCP formulations can be used to deliver mRNA, siRNA, microRNA, and other nucleic acid molecules.
The lab’s research focuses on developing imaging and therapeutic technologies for addressing challenging problems in cancer, inflammation, cardiovascular diseases, and infectious diseases. Their primary focus is on the gastrointestinal tract, but they also target areas of the brain, tumor cells, and immune cells.
The unifying theme of their work is the passion for creating innovative and clinically translatable solutions that can impact the future of medicine and improve patient health.
Commercialization with the Spark Fund
Professor Amiji’s goal is to ensure that the delivery technologies are clinically and commercially translatable. That’s where the Spark Fund comes in.
“Many exciting and promising academic ideas, unfortunately, do not move further because of business challenges,” said Professor Amiji. “Through the Spark fund and CRI, I am able to develop business strategies such that these promising ideas can lead to future products and help patients.”
Working through the Spark Fund will enable MCP formulations to be developed for specific target therapeutic or vaccination areas and to commercialize the technology through effective partnerships.
“The CRI has been instrumental for effective technology transfer and commercialization,” said Professor Amiji. “I am working with the CRI team on invention disclosure and patenting, partnerships with different non-profit and for-profit sectors towards commercialization, and creating a structure at Northeastern that can help us continue to advance our research and technology development toward commercialization by building the necessary infrastructure.”
Learn more about Professor Amiji’s research and the other Spark Fund Award grantees here.