Vesigen Receives National Institutes of Health Award in Targeted Genome Editor Delivery Challenge

CAMBRIDGE, Mass., December 19, 2023 /Business Wire/ — Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, was selected as a Phase 1 winner of the National Institutes of Health TARGETED (Targeted Genome Editor Delivery) Challenge. The Company’s proposal, “Engineered ARMMs: Promising Human-Derived Vectors for Cell Type-Specific Delivery of Genome Editors”, builds on recent data demonstrating directed tropism of the Company’s non-viral ARMM (ARrestin-domain 1 Mediated Microvesicles) delivery platform and was among five proposals selected as “Winning Solutions” for programmable delivery systems for gene editing.

Vesigen Highlights Data Demonstrating Directed Tropism of Non-Viral Delivery Platform at the 30th Annual Congress of the European Society of Gene & Cell Therapy

CAMBRIDGE, Mass., October 25, 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 presented data highlighting the potential of the Company’s non-viral ARMM (ARrestin-domain 1 Mediated Microvesicles) technology to overcome fundamental delivery challenges that limit the clinical application of emerging treatment modalities. Data highlighted the first demonstration of cell type-specific targeting of ARMMs and functional delivery of base editing complexes across diverse human cell types. The data were presented at the 30th Annual Congress of the European Society of Cell and Gene Therapy (ESGCT), taking place October 24-27 in Brussels, Belgium.

Vesigen Highlights Non-Human Primate Biodistribution of Non-Viral Technology and Functional Delivery of Genome Editors In Vivo at the Cracking the Code: The Dawn of Nucleic Acid Medicines Meeting

CAMBRIDGE, Mass., October 19, 2023 /Business Wire/ — Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, presented data further highlighting the potential of the Company’s non-viral ARMM (ARrestin-domain 1 Mediated Microvesicles) technology to deliver novel therapeutic payloads to cell and tissue types relevant to multiple therapeutic areas with high unmet need. Specifically, the Company presented new data demonstrating retinal biodistribution of engineered ARMMs across multiple species – including non-human primates – and data demonstrating successful in vivo functional delivery of a Cas9/guide RNA gene editing complex. The data were presented at the Cracking the Code: The Dawn of Nucleic Acid Medicines meeting, taking place October 17-19, 2023, in Worcester, MA.

 

Vesigen to Present at the 2023 Cell and Gene Meeting on the Mesa

CAMBRIDGE, Mass., October 5, 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 that Paulash Mohsen, Chief Executive Officer, will present at the 2023 Cell and Gene Meeting on the Mesa on October 12 at 9:15 AM PT in Carlsbad, CA.

 

Vesigen Highlights New Data on Non-Viral Delivery Platform at the Exosome Based Therapeutic Development Summit

CAMBRIDGE, Mass., September 7, 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 showcased new data at the Exosome Based Therapeutic Development Summit 5th Annual Meeting held September 5-7, 2023 in Boston. The presentation highlighted new data on the company’s proprietary ARMM (ARrestin-domain 1 Mediated Microvesicles) delivery technology, including therapeutic in vivo gene editing in a preclinical model of acute liver injury, and summarized biodistribution translation evidence generated across species, including non-human primates.

 

Vesigen Highlights Therapeutic Platform for Non-Viral Delivery of Gene Editors and RNA at ASGCT 2023

CAMBRIDGE, Mass., May 22, 2023 /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.”

 

Vesigen Therapeutics Announces Presentations at the American Society of Gene and Cell Therapy 26th Annual Meeting

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 Therapeutics Awarded Grant from Friedreich’s Ataxia Research Alliance (FARA) to Develop a Targeted Genome Editing Therapeutic Strategy

CAMBRIDGE, Mass., March 14, 2023 /Business Wire/ — Vesigen Therapeutics, Inc., a biotechnology company developing targeted therapies by engineering a distinct class of human extracellular vesicles called ARMMs (ARrestin-domain 1 Mediated Microvesicles), is pleased to announce receipt of a FARA General Research Grant. Vesigen will evaluate its proprietary technology to deliver CRISPR-Cas genome editing complexes as a non-viral disease-modifying strategy for patients diagnosed with the neurodegenerative disease Friedreich’s Ataxia (FA). Under the terms of the grant, Vesigen Therapeutics will evaluate the use of ARMMs as a non-viral delivery vehicle for genome editing tools to excise the pathogenic repeat expansion in the Frataxin gene. A major focus of this work will require engineering of ARMMs to engage specific tissues and cell types most affected in FA.

Vesigen Therapeutics, Inc. Announces Expansion of Scientific Advisory Board

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

Vesigen Therapeutics Names Rakhshita Dhar to Board of Directors

CAMBRIDGE, Mass., January 30, 2023 /Business Wire/ — Vesigen Therapeutics, Inc., a biotechnology company developing transformative therapeutics by engineering naturally existing vesicles, today announced the addition of Rakhshita Dhar, Senior Director of Venture Investments Health at Leaps by Bayer, to its Board of Directors.

“We are pleased to welcome Rakhshita to our Board of Directors, and for her to join us on our mission to develop transformative therapeutics enabled by our novel and differentiated delivery technology,” said Vesigen Chief Executive Officer, Paulash Mohsen. “Since our launch, we have significantly advanced our drug discovery programs and look forward to working with Rakhshita and other Board of Directors to help guide our programs towards the clinic and patients with unmet medical needs.”

Dhar brings years of global life sciences perspective to Vesigen. At Leaps by Bayer, she is responsible for search and evaluation and healthcare-focused investment. She currently sits on the boards of Deka Biosciences, Gro Bio, and Edifice Health. Prior to joining Leaps, Rakhshita was Director of Business Development at Roche Pharmaceuticals. She also spent a few years at MassBio developing an accelerator program for life-science start-ups. She earned her Undergraduate degree in Biochemistry from Mumbai University and her Master’s in Molecular Biology from Georgetown University.

“It’s an exciting time at Vesigen and I am glad to join the board as the team continues to gain advancements in their novel drug delivery platform with the power to address current challenges and deliver innovative therapies to patients,” said Rakhshita Dhar. “Leaps by Bayer is committed to investing in paradigm-shifting technologies that can transform the lives of millions of patients.”

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