CELL AND GENE THERAPY
Vipergen | February 18, 2021
Vipergen ApS announced today a published study depicting a ground-breaking new medication revelation approach that empowers, interestingly, screening DNA-encoded small-molecule libraries (DELs) inside living cells. The study, published in the peer-reviewed Journal of the American Chemical Society, highlights the potential of Vipergen's Cellular Binder Trap Enrichment® (cBTE) technology to significantly grow the ability to discover novel small molecules, including against difficult-to-drug targets, and reduce attrition rates in drug discovery. Vipergen is offering its cBTE DNA-encoded library drug disclosure platform as a service to biotechnology and pharmaceutical company partners to accelerate and improve their drug discovery processes.
"This research validates the potential for Vipergen's Cellular Binder Trap Enrichment platform to become a revolutionary tool in small-molecule drug discovery based on its ability to successfully screen DELs against drug targets inside living cells, with no requirement for purified active target protein," said Nils Hansen, Ph.D., Chief Executive Officer of Vipergen. "We expect the ability to perform DEL screening under the more physiologically relevant conditions of a living cell to significantly lower attrition rates in drug discovery. Furthermore, by uniquely allowing us to eliminate the requirement for highly purified active target protein, this technology will improve timelines and bring about new options for novel drug development by allowing us to screen many more targets, prioritize those giving the most promising screening results, and screen DELs against challenging targets that are difficult to express and purify in an active form. We look forward to providing this new tool to biotechnology and pharmaceutical partners to substantially improve the efficiency and success of their small-molecule drug discovery efforts."
About Vipergen's Cellular Binder Trap Enrichment®(cBTE) Technology
Vipergen's proprietary Cellular Binder Trap Enrichment®(cBTE) is the first and only technology for screening DNA-encoded small-molecule libraries (DELs) inside a living cell. The platform is designed to accelerate and improve the success rate for discovering novel, high-quality small molecules for drug development—including against challenging drug targets that are difficult to express and purify in an active form.
About Vipergen ApS
Vipergen is a world-leading provider of small-molecule drug discovery services based on DNA-encoded library (DEL) technologies and is the first and only company capable of screening DELs inside a living cell. Vipergen provides its proprietary suite of leading-edge DEL technologies as a service to biotechnology and pharmaceutical company partners, including top pharmaceutical companies in the U.S., EU, and Japan.
Entos Pharmaceuticals, Inc., BioMarin | November 16, 2021
Entos Pharmaceuticals, Inc. (Entos), a clinical-stage biotechnology company developing genetic medicines with its Fusogenix proteolipid vehicle (PLV) nucleic acid delivery platform, and BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that they have entered into an agreement that will see Entos apply its Fusogenix nucleic acid delivery technology to promising gene therapy candidates in the BioMarin pipeline. Under the agreement, Entos will create specially formulated product candidates for BioMarin, a world leader in developing and commercializing innovative biopharmaceuticals for genetic diseases driven by genetic causes.
Entos' Fusogenix platform utilizes a PLV formulation with a novel mechanism of action to deliver molecular payloads, intact and unmodified, directly into target cells. By combining well-tolerated neutral lipids with proprietary fusion-associated small transmembrane (FAST) proteins, the Fusogenix platform efficiently delivers mRNA or DNA to target tissue with excellent tolerability. Fusogenix technology has been applied to a wide range of therapeutic approaches, including vaccines, gene therapy, and gene editing.
"As a company committed to addressing the unmet therapeutic needs of patients living with genetic diseases, BioMarin values novel technologies that enable the development of transformative therapies," said Brinda Balakrishnan, M.D., Ph.D., Group Vice President, Corporate and Business Development at BioMarin. "We believe that Entos' Fusogenix platform offers potentially unique benefits for safe and effective tissue targeting compared with other lipid-based delivery systems. The Fusogenix PLV formulations generated under this agreement are a critical first step in determining how we may incorporate this promising platform into our drug development efforts."
"With a 20-year track record of successfully discovering, developing, and commercializing first- or best-in-class therapies for diverse rare diseases, the BioMarin team has extensive expertise in harnessing the potential of breakthrough technologies. We believe this licensing agreement helps validate the potential of our Fusogenix platform. It also provides additional opportunities for incorporating our technology into cutting-edge treatments that may have significant clinical and commercial value."
John Lewis, Ph.D., Founder and Chief Executive Officer of Entos
Under the terms of the agreement, Entos will use its Fusogenix nucleic acid delivery system to specially formulate BioMarin products directed at multiple undisclosed genetic disease indications. BioMarin will conduct preclinical studies of the Fusogenix-formulated candidates to evaluate their potential as therapies to prevent or treat these conditions.
Entos' most advanced clinical program is Covigenix VAX-001, a COVID-19 DNA vaccine encoding SARS-CoV-2 Spike protein and two genetic adjuvants engineered to stimulate host adaptive and innate immune systems. Formulated using the Fusogenix platform, Covigenix VAX-001 is the subject of an ongoing phase 2 clinical trial in South Africa, with additional trials planned in other regions. Licensees of the Fusogenix platform include Oisín Biotechnologies and OncoSenX, which are using the technology for age-related diseases and oncology applications, respectively.
pH Partners, LLC served as financial advisor to Entos.
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and preclinical product candidates.
About Entos Pharmaceuticals, Inc.
Entos develops next generation nucleic acid-based therapies using its proprietary Fusogenix proteolipid vehicle (PLV) drug delivery system. Fusogenix is formulated with FAST proteins to deliver mRNA or DNA directly into the cytosol of target cells for translation into protective and therapeutic vaccines and medicines.
Vedere Bio | October 29, 2020
Vedere Bio, Inc. (Vedere Bio), a stealth-stage company focused on advancing photoreceptor-protein-based optogenetic therapies that are delivered to the retina intravitreally to restore functional vision, announced today that it has been acquired by Novartis. Shareholders in Vedere Bio received $150 million upfront and will be eligible for up to $130 million in milestone payments, for a total of $280 million. Based on technology from the laboratories of Drs. Ehud Isacoff and John G. Flannery of UC Berkeley, and technology directed at enhanced ocular gene therapy delivery arising jointly between UC Berkeley and the School of Veterinary Medicine at the University of Pennsylvania, Vedere Bio was formed in the Atlas Venture incubator in June 2019. The company was launched with a $21 million Series A financing and began lab operations at LabCentral in Cambridge, MA where it advanced its lead programs from concept to development candidate within one year. Immediately prior to the acquisition, certain earlier-stage vision restoration and vision preservation assets leveraging the company's ocular gene therapy toolbox were spun out into a newly formed entity – Vedere Bio II, Inc.