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.

“We are delighted to partner with FARA to harness the therapeutic potential of ARMMs as vehicles for targeted genome editing in proprioceptive neurons, which reside in the dorsal root ganglia. We believe that our technology is uniquely suited to enable cell type-specific correction of the underlying cause of a genetic disorder, such as FA,” said Joseph Nabhan, PhD, Chief Scientific Officer, Vesigen Therapeutics.

The FARA Grant Program funds competitive grants across the spectrum from basic research through drug development and clinical research programs. “The work being done by Vesigen Therapeutics has the potential to be transformative for FA patients,” says Jennifer Farmer, CEO of FARA. “We are grateful for Dr. Nabhan and his team for their work on this targeted genome editing strategy.”

This is the first grant in neurological disorders received by Vesigen Therapeutics.

About FARA

The Friedreich’s Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), non-profit, tax-exempt organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich’s ataxia. FARA’s Mission is to marshal and focus the resources and relationships needed to cure FA. www.cureFA.org

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, 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

Vesigen Therapeutics Appoints Paulash Mohsen as Chief Executive Officer

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

Vesigen Therapeutics Presents at 3rd Exosome-Based Therapeutic Development Summit: Expanding the Therapeutic Boundaries of Exosomes

CAMBRIDGE, Mass., Nov. 16, 2021 /Business Wire/ — Vesigen Therapeutics, Inc., a biotechnology company pioneering a novel extracellular vesicle non-viral delivery technology to develop transformative therapeutics, will be featured at the 3rd Exosome-Based Therapeutic Development Summit occurring November 16-18, 2021, in a presentation led by Vesigen’s Chief Scientific Officer, Joseph Nabhan, PhD.

Showcasing the reunion of leading pharma, biotech, technology providers and academic teams to advance therapeutically relevant exosome-based therapeutics in regenerative medicine, autoimmunity, COVID-19 and beyond, the 3rd Exosome-Based Therapeutic Development Summit is an exciting opportunity that marks Vesigen’s first presentation event at an industry-dedicated meeting.

The presentation highlights new data announced earlier this year on ARMMs payloading, purification and characterization, including demonstration of cellular uptake and intracellular cargo activity. These results illustrate the ability of Vesigen’s proprietary platform to produce ARMMs in a scalable process and effectively deliver functional payloads.

“Using ARMMs as drug delivery vehicles overcomes hurdles faced by other viral and non-viral delivery platforms,” said Joseph Nabhan, PhD, Chief Scientific Officer at Vesigen Therapeutics. “Vesigen has achieved unparalleled levels of intraluminal payloading into ARMMs which, along with functional intracellular delivery of cargo and extensive biodistribution studies, is permitting identification of appropriate therapeutic applications for our platform.”

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.

Vesigen Contact:

Vanessa Le

Manager, Business Operations

Email: info@vesigentx.com

Vesigen Therapeutics Presents Data on Recent Advances to its Engineered ARMMs Therapeutic Delivery Platform at ISEV 2021

CAMBRIDGE, Mass., May 18, 2021 /PRNewswire/ — Vesigen Therapeutics, Inc., a biotechnology company pioneering development of a novel extracellular vesicle-mediated delivery platform, today announced new data supporting the therapeutic potential of ARRDC1 Mediated Microvesicles (ARMMs) as vehicles for intracellular delivery of macromolecules. Data will be shared in two virtual poster presentations at the 2021 International Society for Extracellular Vesicles (ISEV) Annual Meeting occurring May 18-21. The presentations will highlight ARMMs payloading, purification and characterization, including demonstration of cellular uptake and intracellular cargo activity. These results illustrate the ability of Vesigen’s proprietary platform to produce ARMMs in a scalable process and effectively deliver functional payloads.

 

ISEV 2021 Annual Meeting Poster Presentations

Title: Scalable Production of ARRDC1 Mediated Microvesicles (ARMMs) as Non-Viral Vehicles for the Delivery of Therapeutic Payloads
Presenting Author: Kristin Luther, PhD, Senior Scientist
Session: Separation and Concentration II
Abstract Number: PS24.08

Title: High Payloading Efficiency and Functional Intracellular Delivery of Diverse Macromolecules Using ARRDC1 Mediated Microvesicles (ARMMs)
Presenting Author: Joseph Nabhan, PhD, VP, Drug Discovery
Session: EV-based Therapeutics & Delivery Vehicles
Abstract Number: PS22.06

The poster presentations will highlight Vesigen’s ability to 1) produce and purify engineered ARMMs in a scalable process, 2) augment payloading of ARMMs with cargo using our proprietary approach to ~300 molecules/vesicle, and 3) enrich ARMMs to 90% of the purified extracellular vesicle preparation. Data will also be presented on stability testing of ARMMs and on cellular uptake of a variety of payloads including functional delivery of a nuclear enzyme.

“We look forward to sharing our team’s data that support ARMMs as versatile intracellular delivery vehicles for a variety of cargo, such as effector proteins, transcription factors or enzymes,” said Joseph Nabhan, PhD, VP of Drug Discovery at Vesigen Therapeutics. “Our team has demonstrated how to engineer cells to produce high levels of payloaded ARMMs, purify them at scale, and deliver biologically active payloads to the cytoplasm and nucleus of target cells, which represents a significant step forward in establishing this platform for non-viral delivery of therapeutic molecules.”

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.

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

Vesigen Therapeutics Appoints Industry Veteran Dieter Weinand to Board of Directors

CAMBRIDGE, Mass., Apr. 6, 2021 /PRNewswire/ — Vesigen Therapeutics, Inc., a biotechnology company pioneering a novel extracellular vesicle delivery technology, today announced the addition of seasoned life sciences executive, Dieter Weinand, to its Board of Directors. Weinand joins Gerald Chan, Chairman, Stephen Bruso, Juergen Eckhardt, Lucio Iannone, and Robert Millman, Co-Founder and Chief Executive Officer, on Vesigen’s Board.

“We have now confirmed the ARMMs platform as a means to translate validated biology into drug leads,” said Millman. “I look forward to working with such an experienced industry veteran to drive Vesigen into being the next big drug development company.”

Weinand has an extensive background in the international pharmaceutical and biotech community. In addition to Vesigen’s Board, he presently serves as the Chairman of the Board of Directors of ZielBio and Replimune Group Inc. He was formally the Executive Vice President of Primary Care at Sanofi, while also serving as a member of their Executive Committee. Before moving to Sanofi, he was CEO and Chairman of the Board of Management of Bayer Pharma AG and member of the Management Board at Bayer AG.  Weinand has also held several roles providing commercial, operational, and corporate strategy at Pfizer, Bristol Myers Squibb, and Otsuka.

Vesigen is a start-up 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. ARMMs possess distinct properties, not found in other classes of extracellular vesicles, such as exosomes, making them better suited for delivering therapeutic payloads. 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.

“This is an exciting time for me to join the Vesigen team as they industrialize their platform and move delivery to the forefront of drug development,” said Weinand. “The ARMMs technology presents a unique opportunity to transition innovative technologies into viable drug leads needed within the pharmaceutical industry and ultimately transform patient care.”

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.

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

Vesigen Therapeutics Announces Issuance of U.S. Patent for Broad Applications of ARMMs Technology

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

Vesigen Therapeutics Names Lucio Iannone to Board of Directors

CAMBRIDGE, Mass., Dec. 1, 2020 /PRNewswire/ — Vesigen Therapeutics, Inc., a biotechnology company pioneering a novel extracellular vesicle delivery technology to develop transformative therapeutics, today announced the addition of Dr. Lucio Iannone, Senior Director of Venture Investments at Leaps by Bayer, to its Board of Directors. Iannone joins Gerald Chan, Chairman, and Stephen Bruso of Morningside Ventures and Juergen Eckhardt of Leaps by Bayer on Vesigen’s Board.

“We are pleased to welcome Lucio to our Board of Directors to join us on our mission to progress the ARMMs technology forward across a wide range of therapeutic targets and areas,” said Vesigen Co-Founder and Chief Executive Officer, Robert Millman. “Since our launch, we have significantly scaled-up our research and operational growth and look forward to leveraging the valuable insight from our Board of Directors to help guide us with target expansion and preclinical development.”

Iannone brings years of global life sciences perspective to Vesigen. At Leaps by Bayer, he is responsible for developing investment cases and finalizing deal execution. He is also involved in sourcing, screening, and mentoring companies evolving groundbreaking science. Iannone was as an integral figure in the financing, and currently serves as a board member, for Khloris Biosciences, Pyxis Oncology, Immunitas Therapeutics, eGenesis, Azitra, and Axxam. Prior to Leaps by Bayer, Iannone held several senior roles at various biotechnology companies and venture capital firms. Iannone obtained his Ph.D. in Medicine at the Imperial College of London.

Vesigen is a start-up 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. ARMMs possess unique properties, not found in other classes of extracellular vesicles, such as exosomes, making them better suited for delivering therapeutic agents. 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) have been packaged into ARMMs and functionally delivered intracellularly in vitro and in vivo.

“I’m thrilled to be joining the Vesigen team at this early stage, as they endeavor to disrupt current barriers in drug delivery and design new pathways for treatment options enabling curative treatments in a large spectrum of disease areas,” said Lucio Iannone, Ph.D. “Leaps by Bayer is committed to investing in transformative biotechnologies with the ability to move the paradigm from treatment to cure.”

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

Vesigen Therapeutics launches with USD 28.5 million Series A investment led by Leaps by Bayer and Morningside Ventures

  • Investment addresses today’s challenge to efficiently transport next-generation therapeutics such as cell and gene therapy to the target and unlock their full potential as innovative therapies for patients
  • Vesigen’s ARMMs technology enables translation of new modalities such as RNAs and gene-editing complexes into novel treatments for multiple diseases

Cambridge, Massachusetts, USA / Berlin, Germany, July 22, 2020 – Vesigen Therapeutics launched today with the objective to overcome the hurdle of targeted intracellular drug delivery of next-generation therapeutics, such as CRISPR-cas9, RNA molecules and other therapeutic proteins, by using a proprietary delivery technology. Leaps by Bayer and Morningside Ventures led the company’s Series A financing with USD 28.5 million and were joined by Linden Lake Ventures and Alexandria Venture Investments.

New modalities, such as gene editing, mRNA replacement, and RNA interference, have huge potential for future therapies and even curative treatments. However, delivery of these modalities to target disease in specific cells and tissues has proven difficult. As more than 80 percent of identified and biologically validated drug targets are located within a human cell, there is a high need for innovative intracellular delivery technology to transport these molecules to the target and unlock the full potential of new therapeutic modalities. Vesigen is developing a novel delivery technology that addresses this hurdle and opens new pathways to transformative treatment options for patients.

“Our mission is to realize the therapeutic potential of many of the new modalities, such as RNA interference, mRNA replacement, and gene editing, that have been used to identify and validate targets, but have proven difficult to translate into functional therapeutics due to delivery barriers,” said Robert Millman, Co-Founder and CEO of Vesigen Therapeutics.

Vesigen is developing drugs based on the proprietary ARRDC1-mediated microvesicles (ARMMs) technology, a class of fusogenic extracellular vesicles that nature evolved to package and deliver communication signals between cells and tissues. ARMMs possess unique properties, making them better suited for producing and delivering therapeutic agents. Vesigen and its scientific founders have demonstrated a wide range of therapeutic payloads can be packaged in ARMMs, including RNA, protein, and gene-editing systems and functionally delivered intracellularly in vitro and in vivo.

“Leaps by Bayer is investing in transformative biotechnologies with the ability to move the paradigm from treatment to cure,” said Juergen Eckhardt, MD, Head of Leaps by Bayer.
“We believe that Vesigen’s ARMMs technology has the potential to do exactly that, to help enable new curative treatments in a large spectrum of disease areas.”

“Vesigen’s engineered extracellular vesicles show versatility and we will be supporting their development in a wide range of indications, including oncology, neurology, ophthalmology, and other localized applications – We look forward to seeing their work translated into a clinical context,” said Gerald Chan, Sc.D, Morningside Ventures.

Vesigen will use the capital raised to build out the ARMMs platform as well as to advance numerous therapeutic agents into preclinical and clinical development. With the financing, Vesigen has named Gerald Chan of Morningside Ventures as Chairman, while Stephen Bruso of Morningside Ventures and Juergen Eckhardt and Jak Knowles of Leaps by Bayer have been named to its Board of Directors.

The company’s Scientific Advisory Board will include:

  • Quan Lu, PhD, Scientific Co-Founder, Vesigen Therapeutics
  • Stephen Haggarty, PhD, Massachusetts General Hospital
  • Thomas Reh, PhD, University of Washington

“We engineered the ARMMs system as a platform capable of delivering several agents into specific tissues or cells that others have been exploring for years,” said Quan Lu, PhD, Professor of Environmental Genetics and Physiology at the Harvard T.H. Chan School of Public Health. “I’m hopeful that our work may enable new therapies that save lives.”

About Bayer and Leaps by Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2019, the Group employed around 104,000 people and had sales of 43.5 billion euros. Capital expenditures amounted to 2.9 billion euros, R&D expenses to 5.3 billion euros. For more information, go to www.bayer.com.

Leaps by Bayer, a unit of Bayer AG, leads impact investments into solutions to some of today’s biggest challenges in health and agriculture. The investment portfolio includes more than 30 companies. They are all working on potentially breakthrough technologies to overcome some specific challenges such as, e.g. regenerating lost tissue function, reducing the environmental impact of farming, preventing or curing cancer, and others. For more information, go to www.leaps.bayer.com

About Morningside Ventures
Founded in 1986 by the Chan family, Morningside is a technology and life science venture investment firm. With offices in Boston, Shanghai and London, the firm invests globally. Its portfolio emphasizes spin-outs from universities. In 2014, the founders of Morningside made a gift to Harvard University which renamed its public health school the Harvard T.H. Chan School of Public Health. To learn more, visit www.morningside.com.

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), enables 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, expanding the universe of druggable targets. 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 hijacked by many enveloped viruses, such as HIV and Ebola, to produce envelope packages of 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 producing therapeutic agents. It could be demonstrated that a wide range of labile and difficult to deliver therapeutics 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 have been 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 Bayer:

US Media Contact
Daniel Childs, phone +1 (973) 437-0809
Email: daniel.childs@bayer.com

Global Media Contact
Sarah-Christine Wanner, phone +49 175 309 21 93
Email: sarah-christine.wanner@bayer.com

Find more information at www.leaps.bayer.com
Our online press service is just a click away: media.bayer.com
Follow us on Twitter: @LeapsByBayer

Contact Vesigen:
Bridgette Chandhoke
Head of Corporate Communications
Email: info@vesigentx.com
Follow us on Twitter: @vesigen_tx

scw (2020-0172)

Forward-Looking Statements
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

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