Value Proposition:
· Platform technology for prophylactic and therapeutic vaccination with large RNA payloads.
· Can deliver several types of nucleic acid payloads including self-amplifying mRNA, plasmid, linear DNA, siRNA, miRNA, mRNA.
· Adaptable as a therapeutic delivery strategy for genetic, musculoskeletal, and neurological disorders.
Unmet Need:
Vaccinology is moving towards synthetic RNA platforms that allow for rapid, scalable, and cell-free manufacturing of prophylactic and therapeutic vaccines, a necessity made apparent by the SARS-CoV-2 pandemic. Self-amplifying mRNA (SAM) has been explored alongside conventional mRNA vaccine strategies. SAM exhibits promise as a vaccine strategy because it mitigates the necessity of large doses or repeat administrations. However, issues include effective delivery of payloads and stability/integrity of payloads once delivered and gene delivery strategies are required for effective delivery. Microparticulate delivery systems have been a promising advancement in vaccines yet conventional constructs have shown little promise in intramuscular delivery. Thus, there is a strong need to develop an effective multiparticulate delivery system for the intramuscular delivery of nucleic acid payloads, including SAM.
Technology Overview
The disclosed technology is a rapidly biodegradable, polymeric nanoparticle platform as a delivery vector for intramuscular large RNA construct delivery. The technology can deliver an assortment of nucleic acid payloads including self-amplifying mRNA, plasmid, linear DNA, siRNA, miRNA, mRNA, and others. Potential mRNA therapy applications outside of vaccines for genetic diseases, neurological diseases, and heart attack victims.
Stage of Development
· In vitro preclinical studies are ongoing.
Publications:
1. WO2023077150
