New article Published in Cell Describes the Safe and Effective Delivery of DNA and RNA Using Proteolipid Vehicles
- This is the first study to demonstrate re-dosable systemic delivery of DNA and RNA to extrahepatic tissues and organs using a fusion-powered nanoparticle.
- FAST-PLVs showed broad biodistribution to tissues beyond the liver and lungs with effective mRNA and DNA delivery in mouse and non-human primate models.
- Administration of follistatin DNA gene therapy with FAST-PLVs raised circulating follistatin levels and significantly increased muscle mass and grip strength.
EDMONTON, Alberta – September 10, 2024 – Entos® Pharmaceuticals (Entos), a clinical-stage genetic medicines company committed to using its Fusogenix™ PLV™ technology to develop cures and improve the lives of patients and their families, today announced the publication of a research article in Cell. The article, titled “Safe and Effective Delivery of DNA and RNA Using Proteolipid Vehicles,” details the development of a novel delivery platform for nucleic acid medicines that overcomes the limitations of current delivery systems. Incorporating fusion associated small transmembrane (FAST) proteins into a proteo-lipid vehicle (PLV), or FAST-PLV, this new platform can effectively deliver mRNA or plasmid DNA into a wide variety of cells and tissues with low immunogenicity and broad distribution throughout the body. Entos led research that involved collaborators from the University of Alberta, Dalhousie University, Oisín Biotechnologies and AegisLife, Inc., to develop the platform and conduct studies demonstrating its advantages over lipid nanoparticle (LNP) delivery systems. The research findings were published by Cell online this morning and will appear in the journal’s September 19th print issue.
“Currently AAV and LNPs are the main delivery platforms for genetic medicine. Both platforms have significantly advanced the field with approved therapies and vaccines for a variety of serious and often life-threatening diseases,” said John Lewis, Ph.D., senior author of the study and Bird Dogs Chair of Translational Oncology at the University of Alberta and CEO of Entos Pharmaceuticals. “Despite these advancements, gaps remain in the ability of current technologies to fully realize the potential of genetic medicines. We designed the FAST-PLV platform to bridge these gaps and deliver on the promise of genetic medicines that patients urgently need.”
The FAST-PLV platform is a fusion-powered delivery system for genetic medicines that combines aspects of viral and non-viral approaches to leverage a completely new mechanism for intracellular delivery of RNA and DNA. While AAV and LNP rely on endocytosis, the FAST protein enables direct fusion of the PLV to the plasma membrane of target cells. This substantially improves the potency and tolerability of genetic medicines, while enabling the development of formulations that are inherently non-liver tropic, enabling the delivery of therapeutic doses of nucleic acid therapies to cells and tissues throughout the body. In this work, the platform was evaluated for safety, efficacy, and biodistribution in various animal models, as well as demonstrating that the FAST-PLV platform is non-immunogenic, which allows for repeat dosing of genetic medicines.
“I am incredibly proud of the strides we’ve made in genetic medicine with our FAST-PLV platform. This breakthrough not only underscores the potential of our technology to overcome current limitations in gene delivery but also reinforces our commitment to developing safer, more effective, and re-dosable therapies for patients. The publication of our work in Cell is a significant milestone, and we believe it marks the beginning of a new era in the field of genetic medicine,” said Arun Raturi PhD., senior author and CSO of Entos Pharmaceuticals.
FAST-PLVs form the basis of Entos’ Fusogenix™ PLV™ delivery platform, which is being utilized by Entos Pharmaceuticals to develop novel therapies for congenital generalized lipodystrophy, obesity, Stargardt disease and cystic fibrosis in addition to its clinical VAX-002 vaccine program. Entos and its valued partners are developing applications in the central and peripheral nervous systems, oncology, longevity and more.
About Entos Pharmaceuticals Inc.
A new reality in genetic medicine lies ahead, one that will be ushered in with the advent of safe, effective, and re-dosable nucleic acid delivery technologies. Since its inception in 2016, Entos® has been dedicated to advancing next-generation genetic medicines using our proprietary FusogenixTM PLVTM drug delivery system. The Fusogenix PLV platform is formulated with FAST proteins to enable the delivery of nucleic acid to target cells through direct fusion. Entos is pioneering the development of life-changing medicines for patients and has partnered with global companies, such as Eli Lilly, to accelerate and expand the impact of our platform. Entos Pharmaceuticals Inc. is headquartered in Edmonton, Canada, with its wholly owned U.S. and U.K. subsidiaries based in San Diego, California and London, United Kingdom, respectively. For more information, visit www.entospharma.com, or follow Entos on LinkedIn.
Forward Looking Statements
This press release contains forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements contained in this press release include statements regarding Entos and its belief as to the mode of action and potential and benefits of the Fusogenix proteolipid vehicle platform and the mode of action and potential and benefits of Covigenix VAX-002 for the treatment of COVID-19; and other statements related to anticipated developments in the Company’s business and technologies. In any forward-looking statement in which Entos expresses an expectation or belief as to future results, such expectations or beliefs are expressed in good faith and are believed to have a reasonable basis, but there can be no assurance that the statement or expectation, or belief will be achieved. Such forward-looking statements involve known and unknown risks and uncertainties, which could cause the Company’s actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue research and development projects, the efficacy of Fusogenix as a nucleic acid delivery vehicle, the efficacy of Covigenix VAX-002, uncertainties related to the research and development of pharmaceuticals, uncertainties related to the regulatory process and general changes to the economic environment. We may incur expenses or delays relating to such events outside of our control, which could have a material adverse impact on our business, operating results, and financial condition. Investors should consult with the U.S. Securities Commission for additional information on risks and uncertainties relating to the forward-looking statements. Investors are cautioned against placing undue reliance on forward-looking statements. The Company does not undertake any obligation to update these forward-looking statements except as required by applicable laws.
Entos® word mark and design logo, FusogenixTM and PLVTM are registered trademarks of Entos Pharmaceuticals Inc. All other trademarks and registered trademarks are the property of their respective owners.
Contacts
Entos Pharmaceuticals Corporate Contact:
John Lewis, Ph.D., Founder and CEO
info@entospharma.com
Entos Pharmaceuticals Media Contact:
Perrin Beatty, Ph.D.
media@entospharma.com
1-800-727-0884