Presentation of industry-leading T-cell delivery in NHPs

Oral presentation highlighting an all-RNA system enabling exon-sized genome insertions

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) 27^th 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.”

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 System”

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 Primates”

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.

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