Polymeric Nanoparticle Genetic Vaccines

Value Proposition

• Versatile immunotherapy platform with the ability to specifically target antigen presenting cells (APCs) ) following systemic delivery and without requiring targeting ligands to improve treatment efficacy. 
• Flexibility to modulate the immune system through activation or inducing tolerance.
• Useful as a preventative strategy or therapeutic strategy for various diseases with limited treatment options including autoimmune diseases, cancer, and infectious diseases. 

Technology Description

Researchers at Johns Hopkins have developed a flexible nucleic acid delivery system that can specifically target antigen presenting cells to modify immune activity. This highly flexible platform technology can either activate the immune system to fight cancers and infectious diseases or tolerize it to treat autoimmune diseases. This delivery system’s ability to successfully modulate the immune system and significantly increase efficacy is due to its unique ability to target APCs following systemic delivery and without requiring targeting ligands. These outcomes are a result of the next-generation polymer compositions of matter formulations used in the nanoparticle design.

Unmet Need

Cancer and infectious diseases were responsible for approximately 18 million global deaths in 2019. Furthermore, based on a UK cohort study, autoimmune diseases are estimated to affect 1 in 10 individuals. Immune modulation is a promising therapeutic strategy for these conditions due to its ability to stimulate a patient’s immune system to appropriately attack diseased cells or dampen an inappropriate immune response. While immunotherapy remains an active developmental area of cancer therapeutics, there are currently no curative treatments for many of the patients with these chronic and/or fatal diseases. Therefore, there is a strong need to develop immune-modulating therapies as curative solutions.

Stage of Development: Preclinical proof-of-concept studies have been completed in mouse models.

Data Availability: Data available upon request.

Publications:

• WO 2023/056293
• Ben-Akiva, Elana, et al. "Biodegradable lipophilic polymeric mRNA nanoparticles for ligand-free targeting of splenic dendritic cells for cancer vaccination." Proceedings of the National Academy of Sciences 120.26 (2023): e2301606120.
• Hu, Yizong, et al. "Supramolecular assembly of polycation/mRNA nanoparticles and in vivo monocyte programming." Proceedings of the National Academy of Sciences 121.35 (2024): e2400194121.