On May 2, 2022 Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of fully engineered circular RNA therapeutics, reported multiple data presentations at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 25th Annual Meeting taking place in Washington, D.C., or virtually, from May 16 – 19, 2022 (Press release, Orna Therapeutics, MAY 2, 2022, View Source [SID1234613335]). Oral presentations will describe Orna’s pipeline for the first time, revealing key data on its in situ CAR (isCAR) program, amongst others, and detail the development of a powerful, new screening platform (FoRCE). Poster presentations will provide additional information on the development of delivery solutions for the isCAR platform and on the development of our muscle genetic disease program.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
Details for the presentations are shared below.
Oral Presentations:
In-situ CAR Therapy Using oRNA Lipid Nanoparticles Regresses Tumors in Mice
Presenter: Tom Barnes, Ph.D., CEO
Date/Time/Location: Monday, May 16, 2022 from 9:10 – 9:45 a.m. ET in Room 207
Session: Scientific Symposium: Function and Therapeutics Applications of Circular RNAs (circRNAs)
Summary: Orna will introduce its pipeline along with key data from its isCAR program. Data from iterative animal studies will demonstrate that oRNA lipid nanoparticles (oRNA-LNPs) can be designed to overcome current challenges of engineered cell therapies. Orna will also present progress on oRNA-LNP (non-viral) delivery of long forms of dystrophin and advances with vaccine therapies.
Discovery of Translation Initiation Elements Enabled by a Parallel Arrayed Screen of Full-length Viral UTRs in Synthetic Circular RNA
Presenter: Alexander Wesselhoeft, Ph.D., Director, Molecular Biology
Date/Time/Location: Monday, May 16, 2022 from 11:30 – 11:45 a.m. ET in Salon H
Session: Oral Abstract Session: Oligonucleotide Therapeutics
Summary: Newly discovered internal ribosome entry sites (IRES) show greater activity (vs EMCV, a common IRES) and some produce different expression levels based on cell type, granting more options for improved expression and control of oRNA.
Poster Presentations:
Improved Immune Cell Expression with Circular RNA (oRNA) in vivo
Presenter: Kevin Kauffman, Ph.D., Principal Scientist
Date/Time/Location: Monday, May 16, 2022 at 5:30 p.m. ET in Hall D
Session: Poster Session: Oligonucleotide Therapeutics I
Summary: oRNA lipid nanoparticles (oRNA-LNPs) show higher splenic T cell expression and biodistribution to the spleen in vivo with improved formulation characteristics compared to their linear mRNA-LNP counterparts.
Systemic Delivery of Circular RNA Encoding Partial Dystrophins and Expression in Skeletal Muscle
Presenter: Tatiana Fontelonga, Ph.D., Scientist
Date/Time/Location: Tuesday, May 17, 2022 at 5:30 p.m. ET in Hall D
Session: Poster Session: Oligonucleotide Therapeutics II
Summary: Micro and mini versions of the dystrophin gene can be encoded in a high capacity oRNA, delivered via LNP, and properly expressed in primary human cells and the mdx mouse model of Duchenne muscular dystrophy.