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) 26^th 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.

Contact:

Adam Bero, Ph.D.
Kendall Investor Relations
abero@kendallir.com

CAMBRIDGE, Mass., February 7, 2023 /Business Wire/ — Vesigen Therapeutics, Inc., a biotechnology company developing transformative therapeutics by engineering naturally existing vesicles, today announced the expansion of the Scientific Advisory Board with the appointments of Connie Cepko, PhD and Kate Fitzgerald, PhD.

“We are thrilled to welcome Dr. Cepko and Dr. Fitzgerald to our Scientific Advisory Board” said Dr. Joseph Nabhan, Chief Scientific Officer of Vesigen Therapeutics, “At Vesigen, we’re developing therapeutics using a differentiated delivery technology to treat a range of diseases with unmet need. Dr. Cepko brings a wealth of knowledge in retinal diseases and Dr. Fitzgerald adds extensive expertise in innate immune system sensing and signaling. We look forward to their valuable contributions to our drug discovery programs.”

CONNIE CEPKO, PhD

Dr. Cepko is the Bullard Professor of Genetics and Neuroscience at Harvard Medical School in the Department of Genetics and in the Department of Ophthalmology. She co-directs the Leder Human Biology and Translational Medicine Program for PhD students at Harvard University. Her ground-breaking research has advanced understanding of the development of the central nervous system (CNS) and mechanisms of retinal degeneration.

A HHMI Investigator and author of over 230 peer-reviewed publications, Dr. Cepko has earned distinguished honors for her work, ranging from induction to the American Academy of Arts and Sciences in 1999 to receiving a Leading Women Award in 2003, presented by the Patriots’ Trail Girl Scout Council in Boston. Dr. Cepko was also elected to the National Academy of Sciences in 2002.

She trained in virology at Massachusetts Institute of Technology (MIT) with Dr. Phillip Sharp where she earned her PhD and later was a postdoctoral fellow at the MIT Whitehead Institute with Dr. Richard Mulligan, where she created some of the first retroviral vectors. Her laboratory is developing gene therapy to treat retinal degenerative diseases, such as retinitis pigmentosa and age-related macular degeneration.

KATE FITZGERALD, PhD

Dr. Fitzgerald is Professor and Vice Chair of Medicine, Chief of the Division of Innate Immunity, and the Worcester Foundation in Biomedical Sciences Research Chair at the University of Massachusetts Chan Medical School. Her work is focused on the innate immune system aimed at understanding the molecular basis of the inflammatory response during Infection and in autoinflammatory diseases. The long-term goal of her work is to determine how innate immune sensing and signaling contribute to infectious, inflammatory, and autoimmune diseases in humans.

Dr. Fitzgerald completed her education in Ireland. She received her B.Sc. in Biochemistry in 1995 from University College Cork, Ireland and her PhD in Biochemistry in 1999 from Trinity College Dublin, Ireland. After pursing a post-doctoral fellowship at Trinity College Dublin, she joined UMass Chan as Instructor where she has been since 2001. She is currently a tenured Professor of Medicine.

Dr. Fitzgerald is an elected fellow of the American Society of Microbiology and an elected member of the Royal Irish Academy, the National Academy of Sciences (USA) and the National Academy of Medicine (USA). She is the recipient of several awards including the Thermo-Fischer Meritorious Career Award (from the American Association of Immunology), the Saint Patrick’s Day Medal (from the Irish Government and Science Foundation Ireland) and the Milstein Award for Excellence in Interferon and Cytokine research (from the International Cytokine and Interferon Society), amongst others.

She has extensive service both locally at UMass Chan and nationally including service on local and national advisory boards (e.g., Massachusetts Center For Pathogen Readiness, NIAID Board of Scientific Councillors, Burroughs Wellcome Fund Pathogenesis of Infectious Diseases and the Cancer Research Institute). She was also the recent past President of the International Cytokine and Interferon Society.

About Vesigen Therapeutics

Vesigen Therapeutics is a biotechnology company developing novel therapeutics enabled by the engineering of naturally existing vesicles for delivery. Vesigen’s patented technology, called ARMMs (ARRDC1 Mediated Microvesicles), can be used to precisely deliver a wide range of payloads, including gene editing complexes, proteins, and RNAs 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.

Contact Vesigen:

Vanessa Le
Manager, Business Operations
Email: info@vesigentx.com

CAMBRIDGE, Mass., Apr. 11, 2022 /Business Wire/ — Vesigen Therapeutics, Inc., a biotechnology company pioneering a novel extracellular vesicle delivery technology, ARMMs, today announced the appointment of Paulash Mohsen as Chief Executive Officer.

“Paulash joins Vesigen with a broad range of skills, experience and accomplishments that are an excellent fit to lead an emerging innovation-focused biotechnology company such as Vesigen,” said Gerald Chan, Sc.D., Chairman of Vesigen’s Board of Directors. “His experience at both large pharma and small biotech companies will be invaluable as we continue advancing our novel programs. On behalf of our Board and management team, we welcome Paulash and look forward to his executive leadership.” Mr. Mohsen is a seasoned biopharmaceutical executive who has served in a variety of roles across multiple disciplines. Prior to his appointment at Vesigen, he served as Chief Business Officer at YumanityTherapeutics, and helped the company grow from a seed stage, research-based startup to a publiccompany, with a lead clinical program in Parkinson’s disease. He was responsible for securing a strategic research collaboration and license agreement with Merck & Co. for two pipeline programs in amyotrophic lateral sclerosis and frontotemporal lobar dementia. Before Yumanity, Mr. Mohsen served as Country Manager in Canada for Cubist Pharmaceuticals (acquired by Merck), where he led the company’s first international operation from conceptualization through commercialization. In this role, he oversaw the approval and launch of two novel anti-infective therapeutics and led the company’s commercial, scientific and administrative functions. Mr. Mohsen joined Cubist via acquisition of Optimer Pharmaceuticals, where he was Vice President, Strategy and Business Operations and helped establish the U.S. commercial infrastructure and led launch preparedness for DIFICID, an anti-infective. Preceding Optimer, Mr. Mohsen held strategic and operational roles at Pfizer, including Vice President of Strategy and Vice President, Multi-Channel Management. Mr. Mohsen holds a B.S. in Chemical Engineering from Brown University, an M.S. in Chemical Engineering from the Massachusetts Institute of Technology and an MBA from Harvard Business School.

Vesigen is a biotechnology company developing drugs based on its patented ARRDC1-mediated microvesicles (ARMMs) technology, a class of fusogenic extracellular vesicles produced by cells to package and deliver communication signals between cells and tissues. Currently, a wide range of therapeutic agents, including RNAs, proteins, and gene-editing complexes have been packaged into ARMMs and functionally delivered intracellularly in vitro and in vivo.

“What attracted me to Vesigen was both its innovative platform and the experience and ingenuity of its management team and research staff,” said Mr. Mohsen. “I am excited to begin working with this worldclass team to break through delivery barriers and enable the next generation of therapeutics for patients worldwide.”

About Vesigen Therapeutics

Vesigen Therapeutics is a biotechnology company advancing groundbreaking therapies directed to intracellular targets using a fusogenic extracellular vesicle delivery technology. Our patented technology, called ARMMs (ARRDC1 Mediated Microvesicles), is expanding the universe of druggable targets by enabling the delivery of a wide range of payloads, including RNAs (mRNA, shRNA, ribozymes), proteins (signaling proteins, enzymes, antibody fragments), and gene-editing complexes (Cas9/gRNA) directly into the cytoplasm of target cells. The team is committed to leveraging ARMMs technology to develop transformative medicines and address currently unmet medical needs. For additional information visit www.vesigentx.com.

About ARMMs

ARMMs vesicles (ARRDC1-mediated microvesicles) are a class of fusogenic extracellular vesicles produced by cells to package and deliver communication signals between cells and tissues. The ARMMs cellular machinery is partially hijacked by many enveloped viruses, such as HIV and Ebola, to produce viruses which bud out of host cells and have specific targeting properties. ARMMs possess unique properties not found in other classes of extracellular vesicles (e.g. exosomes), making them better suited for delivering therapeutic agents. It has been demonstrated that a wide range of labile and difficult to deliver therapeutic agents, including RNAs (mRNA, shRNA, ribozymes), proteins (signaling proteins, enzymes, antibody fragments), and gene-editing complexes (Cas9/gRNA) can be packaged into ARMMs and functionally delivered intracellularly in vitro and in vivo. ARMMS vesicles were discovered and engineered into a drug delivery system in the lab of Quan Lu, PhD, Professor of Environmental Genetics and Physiology at the Harvard T.H. Chan School of Public Health with support in particular from Harvard’s Blavatnik Biomedical Accelerator. Vesigen will advance the ARMMs platform to develop innovative medicines for patients through an exclusive license agreement with Harvard University.

Contact Vesigen:

Vanessa Le
Manager, Business Operations
Email: info@vesigentx.com

CAMBRIDGE, Mass., Mar. 16, 2021 /PRNewswire/ — Vesigen Therapeutics, Inc., a biotechnology company pioneering a novel extracellular vesicle delivery technology to develop transformative therapeutics, today announced that the United States Patent and Trademark Office has issued U.S. Patent No. 10,945,954 entitled “ARRDC1-Mediated Microvesicles (ARMMs) and Uses Thereof”. This patent, exclusively licensed to Vesigen from Harvard University, broadly covers many of the drug delivery features of any ARRDC1-mediated microvesicles (ARMMs).

This patent covers ARMMs compositions containing an enzymatic payload. Important enzymes include those needed in enzyme replacement therapy as well as those intended to create a therapeutic function.

“The issuance of this patent has strengthened Vesigen’s intellectual property estate and provided us with a vast therapeutic blueprint to enable a robust pipeline of agents for use in CNS, oncology, ocular applications and beyond,” said Robert Millman, Co-Founder and CEO of Vesigen Therapeutics.

ARMMs particles are a unique class of extracellular vesicles that are naturally produced by cells and serve as a vehicle to package and deliver communication signals, including transcription complexes, between cells and tissues. ARMMs show enhanced functionality in packaging and transferring payloads, by fusing directly with the cell membrane of a target cell. The cellular ARRDC1 system is a pathway co-opted by enveloped viruses, such as HIV, using a functional homolog, Gag. Like ARMMs particles, enveloped virus particles avoid immune clearance and deliver their viral genome payload specifically to target cells in a fusogenic delivery. Vesigen is engineering ARMMs particles to improve this natural mechanism for more effective targeted delivery of therapeutic agents.

About Vesigen Therapeutics

Vesigen Therapeutics is a biotechnology company advancing groundbreaking therapies directed to intracellular targets using a fusogenic extracellular vesicle delivery technology. Our patented technology, called ARMMs (ARRDC1 Mediated Microvesicles), is expanding the universe of druggable targets by enabling the delivery of a wide range of payloads, including RNAs (mRNA, shRNA, ribozymes), proteins (signaling proteins, enzymes, antibody fragments), and gene-editing complexes (Cas9/gRNA) directly into the cytoplasm of target cells. The team is committed to leveraging ARMMs technology to develop transformative medicines and address currently unmet medical needs. For additional information visit www.vesigentx.com.

About ARMMs

ARMMs vesicles (ARRDC1-mediated microvesicles) are a class of fusogenic extracellular vesicles produced by cells to package and deliver communication signals between cells and tissues. The ARMMs cellular machinery is partially hijacked by many enveloped viruses, such as HIV and Ebola, to produce viruses which bud out of host cells and have specific targeting properties. ARMMs possess unique properties not found in other classes of extracellular vesicles (e.g. exosomes), making them better suited for delivering therapeutic agents. It has been demonstrated that a wide range of labile and difficult to deliver therapeutic agents, including RNAs (mRNA, shRNA, ribozymes), proteins (signaling proteins, enzymes, antibody fragments), and gene-editing complexes (Cas9/gRNA) can be packaged into ARMMs and functionally delivered intracellularly in vitro and in vivo. ARMMS vesicles were discovered and engineered into a drug delivery system in the lab of Quan Lu, PhD, Professor of Environmental Genetics and Physiology at the Harvard T.H. Chan School of Public Health with support in particular from Harvard’s Blavatnik Biomedical Accelerator. Vesigen will advance the ARMMs platform to develop innovative medicines for patients through an exclusive license agreement with Harvard University.

Contact Vesigen:
Bridgette Chandhoke
Head of Corporate Communications
Email: info@vesigentx.com