CAMBRIDGE, Mass., May 2, 2023 /Business Wire/ — Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, today announced eight data presentations at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting being held May 16-20, 2023 in Los Angeles. New preclinical data demonstrate efficient delivery of large genome editor and guide RNA complexes, providing further support of the Company’s novel delivery platform.
Vesigen’s technology comprises engineered forms of a class of naturally existing vesicles to enable efficient functional delivery of complex intracellular payloads, while overcoming challenges observed with other modalities such as tissue specificity and re-dosability. Accepted abstracts are available on the ASGCT website.
“Vesigen’s non-viral vehicles, called ARMMs, enable single particle packaging and in vivo delivery of a variety of complex therapeutic payloads,” said Joseph Nabhan, Ph.D., Chief Scientific Officer, Vesigen Therapeutics. “We are excited to share eight poster presentations, at the upcoming ASGCT conference. Our data demonstrate robust functional delivery of large genome editor and guide RNA complexes, including Cas9 and base editors, both in vivo and in vitro. Vesigen is leveraging the differentiated biodistribution of ARMMs, which we think have tremendous potential to lead to meaningful progress in the development of disease-modifying strategies for intractable genetic diseases of the nervous system, and for complex immune disorders through the targeting of well validated nodes in immune cells. We continue to make excellent progress advancing our programs toward the clinic.”
The Company will also provide updates on Vesigen’s scalable ARMMs production platform, a novel approach for single particle characterization, as well as RNA packaging and delivery.
Details of the poster presentations are listed below. All posters will be presented in the poster hall of the Los Angeles Convention Center at 12 PM PDT.
Wednesday, May 17
1. Title: Targeted Delivery of Genome Editors Complexed with Guide RNA Using ARMMs for Non-Viral In Vivo Delivery
Presenting Author: Qiyu Wang, Ph.D.
Abstract Number: 498
2. Title: ARRDC1-Mediated Microvesicles (ARMMs) as a Novel Non-Viral Modality for Efficient Functional Delivery of Adenine and Cytidine Base Editor Proteins Complexed with Guide RNA
Presenting Author: Wendy Zhao, Ph.D.
Abstract Number: 530
3. Title: ARMMs as a Versatile Non-Viral Delivery Platform for Therapeutic RNA Molecules
Presenting Author: Nelly Valkov, Ph.D.
Abstract Number: 560
4. Title: Scalable Suspension Cell-Based Production and Purification of Engineered ARMMs as a Platform for Non-Viral Therapeutics
Presenting Author: Steven Greenway
Abstract Number: 561
5. Title: Novel Approach for Fluorescent Labeling of Intraluminal Protein Payloads in ARMMs as a Model Extracellular Vesicle
Presenting Authors: Wendy Zhao, Ph.D.
Abstract Number: 562
In collaboration with NanoFCM
Thursday, May 18
6. Title: Biodistribution of Engineered ARRDC1-Mediated Microvesicles for Delivery of Intracellular Payloads In Vivo
Presenting Author: Shu-Lin Liu, Ph.D.
Abstract Number: 909
Friday, May 19
7. Title: Delivery of PMP22-targeting shRNA by ARMMs as A Disease-Modifying Therapeutic Approach for Charcot Marie Tooth 1A
Presenting Author: Nelly Valkov, Ph.D.
Abstract Number: 1307
8. Title: Production, Purification and Characterization of Engineered ARRDC1-Mediated Microvesicles Using a Scalable Manufacturing Platform
Presenting Author: Kristin Luther, Ph.D.
Abstract Number: 1321
In collaboration with Lonza
About Vesigen Therapeutics
Vesigen Therapeutics is a biotechnology company developing a novel, non-viral delivery technology for gene editing, RNA, and protein-based therapeutics. Vesigen’s patented technology, called ARMMs (ARRDC1 Mediated Microvesicles), can be used to precisely deliver a wide range of payloads to a unique set of tissue and cell types. Vesigen has demonstrated highly efficient in vitro and in vivo functional delivery of a range of payloads across multiple cell types and is committed to developing transformative medicines that address current unmet medical needs. ARMMs were discovered and engineered into a drug delivery system at the Harvard T.H. Chan School of Public Health.
For additional information visit www.vesigen.com.
Adam Bero, Ph.D.
Kendall Investor Relations