“These are encouraging data highlighting how ReNAgade’s industry-leading delivery platform can help us target a broader range of therapeutic areas beyond the liver and position us to overcome the limitations of RNA medicines,” said Pete Smith, Ph.D., Chief Scientific Officer of ReNAgade. “Delivery to extra-hepatic tissue is foundational to our approach, and these data help validate how our diverse LNP library can complement our multi-modal RNA platform to create a robust therapeutic pipeline. We look forward to building impactful RNA medicines using novel technologies to target indications with unmet medical needs that were previously not tractable due to poor delivery.”
Key Highlights from the Presentation:
- Delivery of proprietary LNPs encasing cre-recombinase mRNA led to in vivo editing in both short- and long-term hematopoietic stem cells (HSCs), as well as multipotent progenitor cells (MPP) after a single intravenous administration in a murine reporter system. Reporter-positive (cre+) colonies were observed in bone marrow colony forming assays at 24 hours.
- Delivery of reporter mRNA via a proprietary LNP resulted in mRNA expression in CD34+ hematopoietic stem and progenitor cells (HSPC) in non-human primate (NHP) and humanized murine models 24 hours after a single intravenous administration.
- Proprietary LNP delivery to natural killer (NK) cells was also identified via a high-throughput, functional barcoding screening in NHP models.
- Taken together, potent delivery of ReNAgade’s proprietary LNPs was observed in natural killer cells and several subsets of HSCs over multiple animal models (including NHP and humanized murine models), demonstrating robust LNP delivery of mRNA cargo to extra-hepatic tissue.
Details of the Presentation:
TITLE: “Novel LNP-Based Delivery of mRNA to Extra-Hepatic Tissues in NHPs”
PRESENTER: Muthusamy Jayaraman, Ph.D., Senior VP Delivery Chemistry, ReNAgade Therapeutics
DATE AND TIME: Wednesday, November 1, 2023 at 3:18PM CET (10:18AM ET)
About ReNAgade Therapeutics
ReNAgade exists to unlock the potential for RNA medicines to treat disease anywhere in the body. We combine our novel RNA delivery platforms with a comprehensive RNA platform allowing for an all-RNA system for coding, editing, and gene insertion to develop new medicines.
To accelerate the future of medicine, we bring together a team with deep RNA and delivery expertise to develop paradigm-shifting RNA medicines.
ReNAgade Therapeutics—RNA Without Limits
For more information about the company, its technologies, and its leadership, visit www.renagadetx.com