Presentation of industry-leading T-cell delivery in NHPs
Oral presentation highlighting an all-RNA system enabling exon-sized genome insertions
CAMBRIDGE, Mass.–(BUSINESS WIRE)–ReNAgade Therapeutics, a company unlocking the limitless potential for RNA medicines, today announced an oral and poster presentation highlighting preclinical data supporting its comprehensive RNA technology platform at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting being held May 7-11, 2024, in Baltimore, Maryland.
“We are excited to highlight foundational research and development advancements at ASGCT supported by our poster presentation illuminating ReNAgade’s continued work in validating our immune tropic LNP-based delivery systems in NHPs with broad potential applications in oncology and autoimmune diseases,†said Pete Smith, Ph.D., Chief Scientific Officer of ReNAgade. “Additionally, our oral presentation demonstrates a non-viral, all-RNA gene editing system enabling in vivo, exon-sized insertion and probable re-dosing with the goal of higher specificity and a better safety profile when used in the clinical setting. Together, these promising data are another important step in our efforts to innovate beyond the current limitations of RNA medicine.â€
Key Highlights from the Abstracts:
- Employed a systematic barcoding screening approach to evaluate and identify engineered lipid nanoparticles (LNP) from a library of 200+ LNPs across multiple structural and chemically distinct classes of ionizable, helper, structural, and hydrophilic lipids. Lead LNP candidate demonstrated ~60% delivery efficiency in total splenic T cells and ~80% delivery efficiency in peripheral blood T cells in non-human primate models.
- Improved retron-mediated target insertion through a combination of retron reverse transcriptase (RT) engineering, non-coding RNA (ncRNA) minimization, chemical modification, and homology-dependent repair manipulation. Combination of retron system optimization and ncRNA engineering led to 2-fold higher 305 base pair insertion and ~40% editing efficiency in hematopoietic stem cells.
ASGCT Annual Meeting Presentation Details:
- TITLE: “Retron Mediated Exon-Sized Genome Insertion Using an All-RNA Systemâ€
SESSION TITLE: New Technologies for Gene Targeting and Gene Correction
PRESENTATION TYPE: Oral
ABSTRACT NUMBER: 14
PRESENTER: Inna Shcherbakova, Ph.D., Senior Director, Platform, ReNAgade Therapeutics
DATE AND TIME: Tuesday, May 7, 2024, from 2:15 p.m. – 3:30 p.m. ET
- TITLE: “Novel Immune Tropic LNP-Based mRNA Delivery in Non-Human Primatesâ€
SESSION TITLE: Wednesday Posters: Other Nonviral Delivery
PRESENTATION TYPE: Poster
ABSTRACT NUMBER: 749
PRESENTER: Juliet Crabtree, Ph.D., Associate Principal Scientist, ReNAgade Therapeutics
DATE AND TIME: Wednesday, May 8, 2024, at 12:00 p.m. 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.
Contacts
Investor Relations Contact:
Emily Brabbit, Argot Partners
(212) 600-1902
[email protected]
Media Relations Contact:
Sarah Sutton, Argot Partners
(212) 600-1902
[email protected]

