Engineered ARMMs efficiently deliver CRISPR/Cas9 complexes, base editing complexes and RNA therapeutics
Additional presentations demonstrate biodistribution to a range of tissue types and scalable manufacturing processes
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, showcased eight data presentations at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting held May 16-20, 2023 in Los Angeles. New data demonstrate the potential for the Company’s ARMM (ARrestin-domain 1 Mediated Microvesicles) technology to functionally deliver therapeutics to a broad range of disease-relevant cells and tissues.
“We were pleased to share a robust data package highlighting the transformative capacity of our non-viral ARMMs technology to overcome fundamental delivery challenges that limit the potential of promising new treatment modalities,” said Paulash Mohsen, Chief Executive Officer at Vesigen. “Based on the substantial evidence generated to date, we believe our approach enables highly efficient, tunable, and re-dosable delivery of multiple emerging therapeutic modalities. As we advance toward the clinic, we continue to evaluate partnerships where we may combine our ARMM delivery technology with novel therapeutic modalities in order to develop more effective, next-generation medicines.”
In Vivo and in Vitro Functional Delivery of Gene Editing Complexes and Therapeutic RNA Molecules
Data presentations demonstrated engineering of ARMMs to package and functionally deliver CRISPR/Cas9 complexes, base editing complexes, messenger RNA (mRNA), and short hairpin RNA (shRNA) to a diverse range of cell types in vitro and in vivo.
In vivo, a single intranasal administration of ARMMs loaded with gene or base editors targeting the immune modulatory genes NLRP3 or IRF5 resulted in 60+% on-target editing in macrophages and blunted the release of associated cytokines in murine lung tissue.
In vitro evaluation of Vesigen-engineered ARMMs loaded with CRISPR/Cas9 or base editors, in collaboration with David Liu, Ph.D., Professor at the Broad Institute and Harvard University, resulted in efficient editing of multiple genomic loci across a range of human and murine cell types, including primary and post-mitotic cells. ARMMs loaded with mRNA or shRNA were shown to result in robust translation of delivered transcript or knockdown of target gene, respectively.
In Vivo Biodistribution of Engineered ARMMs
Two data presentations highlighted in vivo biodistribution of engineered ARMMs in non-human primates and rodents. In mouse models, functional delivery of engineered ARMMs was observed in the retina, lung, spleen, liver, and other tissues. In non-human primates, data showed delivery of engineered ARMMs intrathecally to cells in the peripheral nervous system.
Advances in Process Development
Three data presentations, including collaborations with Lonza Cell and Gene Technologies and NanoFCM, highlighted development of scalable approaches to produce and characterize ARMMs. The presentations provided evidence supporting production, purification, and characterization processes that enable GMP manufacturing of engineered ARMMs at scale. The use of standard unit operations and readily available starting materials support cost-efficient future manufacturability for clinical trials and commercialization.
Presentation materials are available upon request.
Vesigen Poster Presentations at ASGCT 2023:
Wednesday, May 17
- Targeted Delivery of Genome Editors Complexed with Guide RNA Using ARMMs for Non-Viral In Vivo Delivery (Abstract 498)
- 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 (Abstract 530; collaboration with the laboratory of David Liu, Ph.D., Broad Institute, Harvard University)
- ARMMs as a Versatile Non-Viral Delivery Platform for Therapeutic RNA Molecules (Abstract 560)
- Scalable Suspension Cell-Based Production and Purification of Engineered ARMMs as a Platform for Non-Viral Therapeutics (Abstract 561)
- Novel Approach for Fluorescent Labeling of Intraluminal Protein Payloads in ARMMs as a Model Extracellular Vesicle (Abstract 562; collaboration with NanoFCM)
Thursday, May 18
- Biodistribution of Engineered ARRDC1-Mediated Microvesicles for Delivery of Intracellular Payloads In Vivo (Abstract 909)
Friday, May 19
- Delivery of PMP22-targeting shRNA by ARMMs as A Disease-Modifying Therapeutic Approach for Charcot Marie Tooth 1A (Abstract 1307)
- Production, Purification and Characterization of Engineered ARRDC1-Mediated Microvesicles Using a Scalable Manufacturing Platform (Abstract 1321; 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.
Investor and Media:
Adam Bero, Ph.D.
Kendall Investor Relations