Acuitas Therapeutics

Vancouver, Canada

Acuitas Therapeutics

Vancouver, Canada
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mRNA Technologies have attracted more than US$ 3.4 billion in equity financing and frontloaded partnership payments. Further billions of US$ have been committed to mRNA R&D funding and potential milestone payments. A select group of major pharmaceutical and rare disease specialist biopharmaceutical companies have partnered with mRNA technology companies. At least 16 mRNA vaccines and therapeutics are in clinical stages and a considerable number is in IND or pre-IND stage. This report "mRNA Vaccines & Therapeutics 2017: an industry analysis of technologies, pipelines, stakeholders and deals" as of June 2017 brings you up-to-date regarding key mRNA players, key mRNA technologies and applications, mRNA vaccines & therapeutics, business projects, business deals and funding opportunities. The report analyzes the mRNA vaccine and therapeutic pipelines and stakeholders in the field, especially technology companies and rare disease biopharma and major pharmaceutical companies. The report highlights the value of mRNA vaccines and therapeutics in terms of partnering economic condition and equity financing rounds. For a long time, messenger RNA (mRNA) was thought to be a poor choice for a therapeutic agent given its relatively short half-life and its immunogenicity. But mRNA is rather versatile and offers a range of advantages. mRNA lacks genomic integration and its use results in transient expression of the encoded protein. This favorable safety profile makes mRNA especially attractive for vaccines and gene editing. mRNA is well defined chemically which ensures reproducible manufacturing at high yield, purity and activity. Improvements of lipid nanoparticle formulations as a vehicle for in vivo systemic delivery of mRNA has greatly favored the development of in vivo transfection strategies. What will you find in the report? 2 Introduction & Overview 3 Profiles of mRNA-based Vaccines & Therapeutics 3.1 Standardized Therapeutic Cancer mRNA Vaccines 3.1.1 BI-1361849; CV9202 & CV9201 3.1.2 CV9104 & CV9103 3.1.3 Tetravalent Lipo-MERIT Vaccine 3.1.4 TriMixDC-Mel 3.2 Individualized Therapeutic Cancer mRNA Vaccines 3.2.1 IVAC Mutanome 3.2.2 IVAC Warehouse 3.2.3 mRNA-4157 3.2.4 Rocapuldencel-T; AGS-003 3.3 Therapeutic Infectious Disease mRNA Vaccines 3.3.1 AGS-004 3.4 Prophylactic Infectious Disease mRNA Vaccines & Adjuvants 3.4.1 CV7201 3.4.2 MRK-1777 3.4.3 mRNA-1325 3.4.4 mRNA-1388 3.4.5 mRNA-1440; VAL-506440 3.4.6 mRNA-1647 3.4.7 mRNA-1653 3.4.8 mRNA-1706 3.4.9 mRNA-1851; VAL-339851 3.4.10 RNAdjuvant; CV8102 3.5 mRNA in Immuno-Oncology 3.5.1 CARMA-hMeso 3.5.2 CTX101 3.5.3 mRNA-2416 3.5.4 mRNA-2905 3.6 mRNA Therapeutic for Monogenetic Diseases 3.6.1 CRISPR/Cas9 TTR mRNA-LNP 3.6.2 PRX-ASL 3.6.3 PRX-ASS1 3.6.4 PRX-OTC 3.7 mRNA Therapeutic for Other Diseases 3.7.1 AZD8601 4 Pipeline Analysis 4.1 Standardized mRNA Cancer Vaccines 4.2 Individualized mRNA Cancer Vaccines 4.3 Therapeutic mRNA Infectious Disease Vaccines 4.4 Prophylactic mRNA Infectious Disease Vaccines 4.5 Replicon RNA Infectious Disease Vaccines 4.6 mRNA Protein Therapeutics for Cancer and Cardiovascular Diseases 4.7 mRNA Protein Therapeutics for Ornithine TransCarbomylase (OTC) Deficiency 4.8 mRNA Protein Therapeutics for Cystic Fibrosis 4.9 mRNA Protein Therapeutics for Other Genetic Diseases 4.10 mRNA Antibody Therapeutics 4.11 Therapeutic mRNA-based Gene Editing 4.12 Ex vivo mRNA-based T-Cell Engineering 5 Profiles of Selected mRNA & Delivery Technologies 5.1 Ex vivo mRNA Loading 5.1.1 Arcelis Technology 5.1.2 Flow Electroporation Technology 5.2 In vivo Carrier & mRNA 5.2.1 Hybrid mRNA Technology 5.2.2 Lipid Nanoparticle (LNP) mRNA Technology 5.2.3 Nanotaxi Technology 5.2.4 RNArt & RNAntibody Technology 5.2.5 UNA & LUNAR Technologies 5.3 mRNA Vaccines & Stimulants / Adjuvants 5.3.1 RNActive Technology 5.3.2 Self-Amplifying mRNA (SAM) Technology 5.3.3 TriMix Technology 6 Technology Analysis 6.1 mRNA Modifications 6.2 mRNA Efficiencies 6.3 Adjuvant/Stimulant & Carrier Technologies for mRNA-based Vaccines 6.4 Carriers for mRNA-based Protein & Antibody Therapeutics 7 Company Profiles 7.1 Companies focused on mRNA Therapeutics and Vaccines 7.1.1 Arcturus Therapeutics 7.1.2 Argos Therapeutics 7.1.3 BioNTech 7.1.4 CureVac 7.1.5 eTheRNA immunotherapies 7.1.6 Ethris 7.1.7 In-Cell-Art 7.1.8 Kernal Biologics 7.1.9 Moderna Therapeutics 7.1.10 PhaseRx 7.1.11 RaNa Therapeutics 7.2 Companies with a focus on self-amplifying mRNA (Replicon RNA) 7.2.1 GlaxoSmithKline Vaccines 7.2.2 Synthetic Genomics 7.2.3 Tiba Biotechnology 7.3 Companies with a focus on mRNA Delivery 7.3.1 Acuitas Therapeutics 7.3.2 Arbutus Biopharma 7.3.3 Silence Therapeutics 7.4 Companies with a focus on Gene Editing and CAR T-Cells 7.4.1 CRISPR Therapeutics & Casebia Therapeutics 7.4.2 Intellia Therapeutics 7.4.3 MaxCyte 7.4.4 Sangamo Therapeutics 7.4.5 ZIOPHARM Oncology 7.5 Companies focused on Contract Manufacturing of mRNA 7.5.1 TriLink BioTechnologies 7.5.2 Precision NanoSystems 7.6 Biopharmaceutical Companies with mRNA Programs 7.6.1 Alexion Pharmaceuticals 7.6.2 Regeneron Pharmaceuticals 7.6.3 Ultragenyx Pharmaceutical 7.6.4 Vertex Pharmaceuticals 7.7 Major Pharmaceutical Companies with mRNA Programs 7.7.1 AstraZeneca 7.7.2 Bayer 7.7.3 Boehringer Ingelheim 7.7.4 Janssen 7.7.5 Merck 7.7.6 Roche 7.7.7 Sanofi 7.7.8 Takeda Pharmaceutical Co. 8 Stakeholder Analysis 8.1 mRNA Technology Companies 8.1.1 Companies Focused on mRNA Vaccines & Therapeutics 8.1.2 Companies Focused on Self-Amplifying mRNA 8.1.3 Companies Focused on mRNA Delivery 8.1.4 Companies Focused on Gene Editing & CAR T-Cells by Use of mRNA 8.2 Pharmaceutical Companies with mRNA Programs 8.2.1 Rare Disease Biopharmaceutical Companies with mRNA Programs 8.2.2 Major Pharmaceutial Companies with mRNA Programs 9 mRNA Manufacturing 9.1 In-house mRNA Manufacturing 9.2 Out-sourced mRNA Manufacturing 10 Financial Perspective on mRNA 10.1 mRNA Partnering Deals 10.2 Funded mRNA Programs 11 Outlook 12 References For more information about this report visit https://www.researchandmarkets.com/research/t6pfx2/mrna_vaccines_and Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716


The paper, titled "mRNA mediates passive vaccination against infectious agents, toxins and tumors," by Thran et al., reported results of a multifaceted research program that was designed to explore whether CureVac's RNAntibody® technology, based on chemically unmodified mRNA, is suitable for passive immunization. The results further build on the data included in CureVac's granted RNAntibody® patent (see press release of July 20, 2017). Investigators from CureVac and Tufts Cummings School of Veterinary Medicine tested various antibodies using different designs to determine expression and characterization in vitro and in vivo in the fields of viral infections, toxin exposure and cancer immunotherapies. Results indicated that single injections of mRNA formulated in lipid nanoparticles (LNPs) provided by Acuitas Therapeutics were sufficient to establish rapid, strong and long-lasting serum antibody titers in vivo, thereby enabling both prophylactic and therapeutic protection against lethal rabies infection or botulinum intoxication. Additionally, therapeutic mRNA-mediated antibody expression allowed mice to survive an otherwise lethal tumor challenge. Based on this evidence, the researchers concluded that the utility of formulated mRNA could present a novel armamentarium for the development of competitive passive immunization therapies. Mariola Fotin-Mleczek, Ph.D., Chief Scientific Officer of CureVac and a co-author of the paper, commented, "Multiple experiments have demonstrated the wide-ranging therapeutic applicability of our technology platform using chemically unmodified mRNA. The research published in EMBO Molecular Medicine now adds passive immunization as another potential therapeutic application for mRNA. Using diverse disease models, mRNA-mediated antibody expression, for up to 28 days, proved capable of providing therapeutic benefit, conferring full protection against intoxication and virus challenge while eradicating neoplastic cells in a mouse tumor model. This suggests that mRNA may be the ideal platform for applications requiring antibody-mediated protection. We are very excited to see that our RNAntibody® technology can provide attractive solutions for different kind of antibodies, including functional IgG, single chain camelid and bi-specific antibodies, demonstrating that our non-immunogenic mRNA can be used to deliver any protein molecule. Our sequence-engineered mRNA has the potential to revolutionize human protein therapies with a lower cost of goods and streamlined manufacturing in our state-of-the art production facilities that are currently being expanded to meet clinical and commercial demands." Charles B. Shoemaker, Ph.D., Professor in the Department of Infectious Disease and Global Health (IDGH) at Tufts Cummings School of Veterinary Medicine and a co-author of the paper, stated, "The study in EMBO Molecular Medicine is very promising and suggests that mRNA may offer an attractive alternative to passive immunization given that mRNA technology appears to enable the in vivo synthesis of antibodies displaying favorable pharmacokinetics in which substantial antibody titers are induced in blood as early as two hours after treatment of mice. Today, passive immunization by antibody injection currently fills only a small niche in preventing or treating infectious diseases and has significant drawbacks. Nevertheless, there is renewed interest in passive immunization due to the emergence of microbial resistance to antibiotics, and this has created a demand for alternative therapies. mRNA seems likely to provide a viable new option for meeting this growing need." The full study in EMBO Molecular Medicine can be found here. About RNAntibody® CureVac's RNAntibody® technology can be applied in many disease indications including cancer, cardiovascular diseases, infectious diseases and autoimmune diseases. RNAntibody® is a component of CureVac's RNArt® portfolio of mRNA-based molecular therapeutics that give the body the information required to produce its own functional proteins. Patent No. EP2101823 from the European Patent Office provides broad patent protection for the Company's RNAntibody® technology. About EMBO Molecular Medicine EMBO Molecular Medicine is a peer-reviewed, online open access journal dedicated to a new research discipline at the interface between clinical research and basic biology. It offers clinicians and researchers in this area the opportunity to publish their best work in a broadly distributed and highly visible forum, thereby lending a strong impetus to this important and rapidly developing field and helping to forge new links between clinicians and molecular biologists. Studies based on model organisms also fall within the scope of the journal, provided that the results presented are evidently and directly relevant to human disease. EMBO Molecular Medicine is an open access online journal published by EMBO Press. About CureVac AG CureVac is a leading company in the field of messenger RNA (mRNA) technology with more than 17 years' expertise in handling and optimizing this versatile molecule for medical purposes. The principle of CureVac's proprietary technology is the use of mRNA as a data carrier to instruct the human body to produce its own proteins capable of fighting a wide range of diseases. The company applies its technologies for the development of cancer therapies, prophylactic vaccines and molecular therapies. To date, CureVac has received approximately $370 million (€355 million) in equity investments including significant investments from SAP founder Dietmar Hopp's dievini and an investment of $52 million from the Bill & Melinda Gates Foundation. CureVac has also entered into collaborations with multinational corporations and organizations, including Boehringer Ingelheim, Sanofi Pasteur, the Bill & Melinda Gates Foundation and IAVI. For more information, please visit www.curevac.com


Dublin, Aug. 10, 2017 (GLOBE NEWSWIRE) -- The "mRNA Vaccines & Therapeutics 2017: an Industry Analysis of Technologies, Pipelines, Stakeholders and Deals" report has been added to Research and Markets' offering. mRNA Technologies have attracted more than US$ 3.4 billion in equity financing and frontloaded partnership payments. Further billions of US$ have been committed to mRNA R&D funding and potential milestone payments. A select group of major pharmaceutical and rare disease specialist biopharmaceutical companies have partnered with mRNA technology companies. At least 16 mRNA vaccines and therapeutics are in clinical stages and a considerable number is in IND or pre-IND stage. This report "mRNA Vaccines & Therapeutics 2017: an industry analysis of technologies, pipelines, stakeholders and deals" as of June 2017 brings you up-to-date regarding key mRNA players, key mRNA technologies and applications, mRNA vaccines & therapeutics, business projects, business deals and funding opportunities. The report analyzes the mRNA vaccine and therapeutic pipelines and stakeholders in the field, especially technology companies and rare disease biopharma and major pharmaceutical companies. The report highlights the value of mRNA vaccines and therapeutics in terms of partnering economic condition and equity financing rounds. For a long time, messenger RNA (mRNA) was thought to be a poor choice for a therapeutic agent given its relatively short half-life and its immunogenicity. But mRNA is rather versatile and offers a range of advantages. mRNA lacks genomic integration and its use results in transient expression of the encoded protein. This favorable safety profile makes mRNA especially attractive for vaccines and gene editing. mRNA is well defined chemically which ensures reproducible manufacturing at high yield, purity and activity. Improvements of lipid nanoparticle formulations as a vehicle for in vivo systemic delivery of mRNA has greatly favored the development of in vivo transfection strategies. Range of clinical mRNA applications Cancer Vaccines What will you find in the report? 2 Introduction & Overview 3 Profiles of mRNA-based Vaccines & Therapeutics 3.1 Standardized Therapeutic Cancer mRNA Vaccines 3.1.1 BI-1361849; CV9202 & CV9201 3.1.2 CV9104 & CV9103 3.1.3 Tetravalent Lipo-MERIT Vaccine 3.1.4 TriMixDC-Mel 3.2 Individualized Therapeutic Cancer mRNA Vaccines 3.2.1 IVAC Mutanome 3.2.2 IVAC Warehouse 3.2.3 mRNA-4157 3.2.4 Rocapuldencel-T; AGS-003 3.3 Therapeutic Infectious Disease mRNA Vaccines 3.3.1 AGS-004 3.4 Prophylactic Infectious Disease mRNA Vaccines & Adjuvants 3.4.1 CV7201 3.4.2 MRK-1777 3.4.3 mRNA-1325 3.4.4 mRNA-1388 3.4.5 mRNA-1440; VAL-506440 3.4.6 mRNA-1647 3.4.7 mRNA-1653 3.4.8 mRNA-1706 3.4.9 mRNA-1851; VAL-339851 3.4.10 RNAdjuvant; CV8102 3.5 mRNA in Immuno-Oncology 3.5.1 CARMA-hMeso 3.5.2 CTX101 3.5.3 mRNA-2416 3.5.4 mRNA-2905 3.6 mRNA Therapeutic for Monogenetic Diseases 3.6.1 CRISPR/Cas9 TTR mRNA-LNP 3.6.2 PRX-ASL 3.6.3 PRX-ASS1 3.6.4 PRX-OTC 3.7 mRNA Therapeutic for Other Diseases 3.7.1 AZD8601 4 Pipeline Analysis 4.1 Standardized mRNA Cancer Vaccines 4.2 Individualized mRNA Cancer Vaccines 4.3 Therapeutic mRNA Infectious Disease Vaccines 4.4 Prophylactic mRNA Infectious Disease Vaccines 4.5 Replicon RNA Infectious Disease Vaccines 4.6 mRNA Protein Therapeutics for Cancer and Cardiovascular Diseases 4.7 mRNA Protein Therapeutics for Ornithine TransCarbomylase (OTC) Deficiency 4.8 mRNA Protein Therapeutics for Cystic Fibrosis 4.9 mRNA Protein Therapeutics for Other Genetic Diseases 4.10 mRNA Antibody Therapeutics 4.11 Therapeutic mRNA-based Gene Editing 4.12 Ex vivo mRNA-based T-Cell Engineering 5 Profiles of Selected mRNA & Delivery Technologies 5.1 Ex vivo mRNA Loading 5.1.1 Arcelis Technology 5.1.2 Flow Electroporation Technology 5.2 In vivo Carrier & mRNA 5.2.1 Hybrid mRNA Technology 5.2.2 Lipid Nanoparticle (LNP) mRNA Technology 5.2.3 Nanotaxi Technology 5.2.4 RNArt & RNAntibody Technology 5.2.5 UNA & LUNAR Technologies 5.3 mRNA Vaccines & Stimulants / Adjuvants 5.3.1 RNActive Technology 5.3.2 Self-Amplifying mRNA (SAM) Technology 5.3.3 TriMix Technology 6 Technology Analysis 6.1 mRNA Modifications 6.2 mRNA Efficiencies 6.3 Adjuvant/Stimulant & Carrier Technologies for mRNA-based Vaccines 6.4 Carriers for mRNA-based Protein & Antibody Therapeutics 7 Company Profiles 7.1 Companies focused on mRNA Therapeutics and Vaccines 7.1.1 Arcturus Therapeutics 7.1.2 Argos Therapeutics 7.1.3 BioNTech 7.1.4 CureVac 7.1.5 eTheRNA immunotherapies 7.1.6 Ethris 7.1.7 In-Cell-Art 7.1.8 Kernal Biologics 7.1.9 Moderna Therapeutics 7.1.10 PhaseRx 7.1.11 RaNa Therapeutics 7.2 Companies with a focus on self-amplifying mRNA (Replicon RNA) 7.2.1 GlaxoSmithKline Vaccines 7.2.2 Synthetic Genomics 7.2.3 Tiba Biotechnology 7.3 Companies with a focus on mRNA Delivery 7.3.1 Acuitas Therapeutics 7.3.2 Arbutus Biopharma 7.3.3 Silence Therapeutics 7.4 Companies with a focus on Gene Editing and CAR T-Cells 7.4.1 CRISPR Therapeutics & Casebia Therapeutics 7.4.2 Intellia Therapeutics 7.4.3 MaxCyte 7.4.4 Sangamo Therapeutics 7.4.5 ZIOPHARM Oncology 7.5 Companies focused on Contract Manufacturing of mRNA 7.5.1 TriLink BioTechnologies 7.5.2 Precision NanoSystems 7.6 Biopharmaceutical Companies with mRNA Programs 7.6.1 Alexion Pharmaceuticals 7.6.2 Regeneron Pharmaceuticals 7.6.3 Ultragenyx Pharmaceutical 7.6.4 Vertex Pharmaceuticals 7.7 Major Pharmaceutical Companies with mRNA Programs 7.7.1 AstraZeneca 7.7.2 Bayer 7.7.3 Boehringer Ingelheim 7.7.4 Janssen 7.7.5 Merck 7.7.6 Roche 7.7.7 Sanofi 7.7.8 Takeda Pharmaceutical Co. 8 Stakeholder Analysis 8.1 mRNA Technology Companies 8.1.1 Companies Focused on mRNA Vaccines & Therapeutics 8.1.2 Companies Focused on Self-Amplifying mRNA 8.1.3 Companies Focused on mRNA Delivery 8.1.4 Companies Focused on Gene Editing & CAR T-Cells by Use of mRNA 8.2 Pharmaceutical Companies with mRNA Programs 8.2.1 Rare Disease Biopharmaceutical Companies with mRNA Programs 8.2.2 Major Pharmaceutial Companies with mRNA Programs 9 mRNA Manufacturing 9.1 In-house mRNA Manufacturing 9.2 Out-sourced mRNA Manufacturing 10 Financial Perspective on mRNA 10.1 mRNA Partnering Deals 10.2 Funded mRNA Programs 11 Outlook 12 References For more information about this report visit https://www.researchandmarkets.com/research/dppgr4/mrna_vaccines_and


Tam Y.K.,Acuitas Therapeutics | Madden T.D.,Acuitas Therapeutics | Hope M.J.,Acuitas Therapeutics
Journal of Drug Targeting | Year: 2016

For the best part of 40 years, lipids and membrane fusion have been at the center of Pieter’s research. Projects range from the purely academic quest of understanding the roles of lipids in biological membranes, to the translation of this knowledge into the most advanced lipid nanoparticle (LNP) drug delivery systems in clinical trials to-date. Pieter’s pioneering work in lipid polymorphism and characterizing the unique properties of unsaturated phospatidyethanolamines (PE), together with the introduction of ionizable, dialkylamino lipids to trigger membrane fusion at acidic pH, provided the foundation on which a new generation of highly potent, well-tolerated LNPs for intravenous delivery of nucleic acid therapeutics has been built. In this contribution to the special edition honoring Pieter’s achievements we highlight key research conducted in Pieter’s laboratory and at several biotechnology companies, some spun out of his research group, which resulted in the development of a fusogenic delivery system for siRNA therapeutics. Patisiran®, an LNP encapsulating siRNA for hepatic gene silencing, is currently in Phase III clinical trials for treatment of Transthyretin amyloidosis as are several other siRNA products employing this delivery technology. Finally, we describe more recent work in which the platform shows real promise in the rapidly growing new field of mRNA therapeutics. © 2016 Informa UK Limited, trading as Taylor & Francis Group.


Pardi N.,University of Pennsylvania | Tuyishime S.,University of Pennsylvania | Muramatsu H.,University of Pennsylvania | Kariko K.,University of Pennsylvania | And 5 more authors.
Journal of Controlled Release | Year: 2015

In recent years, in vitro transcribed messenger RNA (mRNA) has emerged as a potential therapeutic platform. To fulfill its promise, effective delivery of mRNA to specific cell types and tissues needs to be achieved. Lipid nanoparticles (LNPs) are efficient carriers for short-interfering RNAs and have entered clinical trials. However, little is known about the potential of LNPs to deliver mRNA. Here, we generated mRNA-LNPs by incorporating HPLC purified, 1-methylpseudouridine-containing mRNA comprising codon-optimized firefly luciferase into stable LNPs. Mice were injected with 0.005-0.250 mg/kg doses of mRNA-LNPs by 6 different routes and high levels of protein translation could be measured using in vivo imaging. Subcutaneous, intramuscular and intradermal injection of the LNP-encapsulated mRNA translated locally at the site of injection for up to 10 days. For several days, high levels of protein production could be achieved in the lung from the intratracheal administration of mRNA. Intravenous and intraperitoneal and to a lesser extent intramuscular and intratracheal deliveries led to trafficking of mRNA-LNPs systemically resulting in active translation of the mRNA in the liver for 1-4 days. Our results demonstrate that LNPs are appropriate carriers for mRNA in vivo and have the potential to become valuable tools for delivering mRNA encoding therapeutic proteins. © 2015 Elsevier B.V. All rights reserved.


Cullis P.R.,University of British Columbia | Hope M.J.,Acuitas Therapeutics
Molecular Therapy | Year: 2017

Genetic drugs such as small interfering RNA (siRNA), mRNA, or plasmid DNA provide potential gene therapies to treat most diseases by silencing pathological genes, expressing therapeutic proteins, or through gene-editing applications. In order for genetic drugs to be used clinically, however, sophisticated delivery systems are required. Lipid nanoparticle (LNP) systems are currently the lead non-viral delivery systems for enabling the clinical potential of genetic drugs. Application will be made to the Food and Drug Administration (FDA) in 2017 for approval of an LNP siRNA drug to treat transthyretin-induced amyloidosis, presently an untreatable disease. Here, we first review research leading to the development of LNP siRNA systems capable of silencing target genes in hepatocytes following systemic administration. Subsequently, progress made to extend LNP technology to mRNA and plasmids for protein replacement, vaccine, and gene-editing applications is summarized. Finally, we address current limitations of LNP technology as applied to genetic drugs and ways in which such limitations may be overcome. It is concluded that LNP technology, by virtue of robust and efficient formulation processes, as well as advantages in potency, payload, and design flexibility, will be a dominant non-viral technology to enable the enormous potential of gene therapy. Genetic drugs based on RNA and DNA can potentially treat most diseases by silencing pathological genes, expressing therapeutic proteins, or by editing the human genome. This review summarizes progress made using lipid nanoparticle (LNP) formulations of genetic drugs to enable gene therapy to be practiced. © 2017 The American Society of Gene and Cell Therapy.


Acuitas Therapeutics Inc., ein privates Biotechnologie-Unternehmen, das eine hochmoderne Lipidnanopartikel(LNP)-Abgabetechnik für Messenger-RNA (mRNA) entwickelt, hat heute die Veröffentlichung von Daten bekanntgegeben, die zeigen, dass mRNA, die breit neutralisierende Antikörper codiert und in einem Acuitas-LNP-Träger abgegeben wird, humanisierte Mäuse vor einer HIV-1-Infektion schützt. In einem heute in Nature Communications veröffentlichten Artikel zeigten Wissenschaftler und akademische Forscher von Acuitas unter der Leitung von Dr. Drew Weissman, MD, Professor für Infektionskrankheiten an der Perelman School of Medicine der Universität Pennsylvania, dass durch eine einzige Injektion mit mRNA-LNP, die den HIV-1-Antikörper VRC01 (1,4 mg/kg) codieren, nach 24 Stunden eine Plasma-Antikörper-Konzentration von etwa 170 µg/ml erreicht wurde. Des Weiteren wurde durch eine wöchentliche Verabreichung von 1 mg/kg mRNA-LNP eine Antikörperkonzentration mit einem Talspiegel von etwa 40 µg/ml beibehalten. Wichtig hierbei ist, dass eine einzige Injektion mit VRC01 mRNA-LNP humanisierte Mäuse auf dosisabhängige Weise vor einer intravenösen HIV-1-Übertragung schützte. "Diese neueste Veröffentlichung durch Dr. Weissman und seine Kollegen verdeutlicht das Potenzial der mRNA-LNP-Therapie als passive Immuntherapie gegen HIV-1 sowie möglicherweise gegen weitere Infektionskrankheiten. Zudem wird hierdurch das Konzept, die Leber als "Proteinfabrik" zu nutzen, sowie die Durchführbarkeit von wiederholten Dosierungen veranschaulicht", sagte Dr. Thomas Madden, Geschäftsführer und Vorstandsvorsitzender von Acuitas Therapeutics. Informationen zu Messenger-RNA (mRNA) Messenger-RNA ist ein natürliches Molekül, das Zellen die Herstellung von Proteinen ermöglicht. Informationen, die für ein spezifisches Protein codieren, sind in Genen im Zellkern enthalten. Diese Informationen werden in eine Nachricht (mRNA) transkribiert, die dann aus dem Kern wandert und in das spezifische Protein translatiert wird. Synthetische mRNA kann mithilfe von Acuitas-LNP-Trägern in eine Zelle eingebracht werden, um die Zelle zur Herstellung eines therapeutischen Proteins anzuweisen. Solche therapeutischen Proteine könnten ein fehlendes oder defektes Protein ersetzen, als Antikörper zum Schutz vor Infektion agieren oder eine schützende Immunantwort (d. h. eine Impfung) bereitstellen. Informationen zu Acuitas Therapeutics Acuitas Therapeutics ist ein privates Biotechnologie-Unternehmen mit Sitz in Vancouver, British Columbia, Kanada, das neuartige LNP-Träger für mRNA und andere Nukleinsäure-Therapeutika entwickelt, darunter eine Technologie, die unter beschränkter Lizenz von der Arbutus Biopharma Corporation entwickelt wird.


Acuitas Therapeutics Inc., a private biotechnology company developing state-of-the-art lipid nanoparticle (LNP) delivery technology for messenger RNA (mRNA), announced today publication of data demonstrating that mRNA encoding a broadly neutralizing antibody delivered in an Acuitas LNP carrier protects humanized mice from HIV-1 challenge. In a paper published today in Nature Communications, Acuitas' scientists and academic researchers lead by Drew Weissman, MD, PhD, a professor of Infectious Diseases in the Perelman School of Medicine at the University of Pennsylvania showed that a single injection of mRNA-LNP encoding the anti-HIV-1 antibody VRC01 (1.4 mg/kg) resulted in plasma antibody concentrations of ~170 µg/mL at 24 hours. Further, weekly administrations of 1 mg/kg mRNA-LNP maintained trough antibody concentrations of ~40 µg/mL. Importantly a single injection of VRC01 mRNA-LNP protected humanized mice from an intravenous HIV-1 challenge in a dose-dependent manner. "This latest publication with Dr. Weissman and his colleagues illustrates the potential for mRNA-LNP therapeutics to provide passive immunotherapy against HIV-1 and potentially against other infectious diseases. In addition it exemplifies the concept of using the liver as a "protein factory" and the viability of repeat dosing" said Thomas Madden, Ph.D., President and Chief Executive Officer of Acuitas Therapeutics. About messenger RNA (mRNA) Messenger RNA is a natural molecule that allows cells to produce proteins. Information that codes for a specific protein is contained on genes in the cell nucleus. This information is transcribed into a message (mRNA), which then migrates out of the nucleus and is translated into the specific protein. Synthetic mRNA can be introduced into a cell using Acuitas LNP carriers to direct the cell to produce a therapeutic protein. Such therapeutic proteins could replace a missing or defective protein, be an antibody to protect against infection, or provide a protective immune response (i.e. a vaccine). About Acuitas Therapeutics Acuitas Therapeutics is a private biotechnology company located in Vancouver, British Columbia, Canada developing novel LNP carriers for mRNA and other nucleic acid therapeutics, including technology developed under limited license from Arbutus Biopharma Corporation.


Thess A.,CureVac GmbH | Grund S.,CureVac GmbH | Mui B.L.,Acuitas Therapeutics | Hope M.J.,Acuitas Therapeutics | And 3 more authors.
Molecular Therapy | Year: 2015

Being a transient carrier of genetic information, mRNA could be a versatile, flexible, and safe means for protein therapies. While recent findings highlight the enormous therapeutic potential of mRNA, evidence that mRNA-based protein therapies are feasible beyond small animals such as mice is still lacking. Previous studies imply that mRNA therapeutics require chemical nucleoside modifications to obtain sufficient protein expression and avoid activation of the innate immune system. Here we show that chemically unmodified mRNA can achieve those goals as well by applying sequence-engineered molecules. Using erythropoietin (EPO) driven production of red blood cells as the biological model, engineered Epo mRNA elicited meaningful physiological responses from mice to nonhuman primates. Even in pigs of about 20 kg in weight, a single adequate dose of engineered mRNA encapsulated in lipid nanoparticles (LNPs) induced high systemic Epo levels and strong physiological effects. Our results demonstrate that sequence-engineered mRNA has the potential to revolutionize human protein therapies. © The American Society of Gene & Cell Therapy.


VANCOUVER, March 2, 2017 /PRNewswire/ - Acuitas Therapeutics Inc., a private biotechnology company developing state-of-the-art lipid nanoparticle (LNP) delivery technology for messenger RNA (mRNA), announced today publication of data demonstrating that mRNA encoding a broadly neutralizing...

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