EA201991747A1 - НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR - Google Patents

НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR

Info

Publication number
EA201991747A1
EA201991747A1 EA201991747A EA201991747A EA201991747A1 EA 201991747 A1 EA201991747 A1 EA 201991747A1 EA 201991747 A EA201991747 A EA 201991747A EA 201991747 A EA201991747 A EA 201991747A EA 201991747 A1 EA201991747 A1 EA 201991747A1
Authority
EA
Eurasian Patent Office
Prior art keywords
cftr mrna
new codon
optimized cftr
codon
optimized
Prior art date
Application number
EA201991747A
Other languages
English (en)
Inventor
Ануша Диаз
Джонатан Абисал
Брайан Беттанкур
Франк Дероза
Майкл Хартлейн
Original Assignee
Транслейт Био, Инк.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Транслейт Био, Инк. filed Critical Транслейт Био, Инк.
Publication of EA201991747A1 publication Critical patent/EA201991747A1/ru

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0075—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a dry powder inhaler [DPI], e.g. comprising micronized drug mixed with lactose carrier particles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07006—DNA-directed RNA polymerase (2.7.7.6)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions

Abstract

В настоящем изобретении предложены, помимо прочего, улучшенные способы и фармацевтические композиции для лечения муковисцидоза на основе кодон-оптимизированной мРНК, кодирующей муковисцидозный регулятор трансмембранной проводимости (CFTR).
EA201991747A 2017-02-27 2018-02-27 НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR EA201991747A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762464215P 2017-02-27 2017-02-27
PCT/US2018/020011 WO2018157154A2 (en) 2017-02-27 2018-02-27 Novel codon-optimized cftr mrna

Publications (1)

Publication Number Publication Date
EA201991747A1 true EA201991747A1 (ru) 2020-06-04

Family

ID=61622756

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201991747A EA201991747A1 (ru) 2017-02-27 2018-02-27 НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR

Country Status (9)

Country Link
US (2) US11253605B2 (ru)
EP (1) EP3585417B1 (ru)
AU (1) AU2018224326B2 (ru)
CA (1) CA3054062A1 (ru)
EA (1) EA201991747A1 (ru)
IL (1) IL268854A (ru)
MA (1) MA47603A (ru)
MX (1) MX2019010155A (ru)
WO (1) WO2018157154A2 (ru)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3998064A1 (en) 2011-06-08 2022-05-18 Translate Bio, Inc. Cleavable lipids
MA45053A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique
IL296445A (en) * 2020-04-27 2022-11-01 4D Molecular Therapeutics Inc Adeno-linked variants, formulations and methods of pulmonary administration
CN116096430A (zh) * 2020-04-27 2023-05-09 衣阿华大学研究基金会 用于治疗囊性纤维化的组合物和方法
US20220023442A1 (en) * 2020-05-01 2022-01-27 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
US20230245721A1 (en) * 2020-05-07 2023-08-03 Translate Bio, Inc. Generation of optimized nucleotide sequences
US20230181619A1 (en) * 2020-05-07 2023-06-15 Translate Bio, Inc. Improved compositions for cftr mrna therapy
TW202231651A (zh) * 2020-10-15 2022-08-16 美商崔斯雷生物公司 大規模合成訊息ｒｎａ
US20220160633A1 (en) * 2020-11-09 2022-05-26 Translate Bio, Inc. Compositions for delivery of codon-optimized mrna
WO2023023487A1 (en) * 2021-08-16 2023-02-23 Translate Bio, Inc. Screening codon-optimized nucleotide sequences

Family Cites Families (435)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2647121A (en) 1951-02-02 1953-07-28 Ruth P Jacoby Diamine-bis-acetamides
US2819718A (en) 1953-07-16 1958-01-14 Isidore H Goldman Drainage tube
US2717909A (en) 1953-09-24 1955-09-13 Monsanto Chemicals Hydroxyethyl-keryl-alkylene-ammonium compounds
US2844629A (en) 1956-04-25 1958-07-22 American Home Prod Fatty acid amides and derivatives thereof
US3096560A (en) 1958-11-21 1963-07-09 William J Liebig Process for synthetic vascular implants
FR1378382A (fr) 1962-12-01 1964-11-13 Sandoz Sa Amides de l'acide amino-propionique, utilisables en particulier pour le traitement des fibres textiles
GB1072118A (en) 1962-12-01 1967-06-14 Sandoz Ag Amides of aminopropionic acid
JPS5141663B1 (ru) 1966-03-12 1976-11-11
NL143127B (nl) 1969-02-04 1974-09-16 Rhone Poulenc Sa Versterkingsorgaan voor een defecte hartklep.
US3614955A (en) 1970-02-09 1971-10-26 Medtronic Inc Standby defibrillator and method of operation
US3614954A (en) 1970-02-09 1971-10-26 Medtronic Inc Electronic standby defibrillator
JPS5024216Y1 (ru) 1970-12-29 1975-07-21
US3945052A (en) 1972-05-01 1976-03-23 Meadox Medicals, Inc. Synthetic vascular graft and method for manufacturing the same
US3805301A (en) 1972-07-28 1974-04-23 Meadox Medicals Inc Tubular grafts having indicia thereon
JPS49127908A (ru) 1973-04-20 1974-12-07
JPS5624664B2 (ru) 1973-06-28 1981-06-08
US4013507A (en) 1973-09-18 1977-03-22 California Institute Of Technology Ionene polymers for selectively inhibiting the vitro growth of malignant cells
JPS5123537A (ja) 1974-04-26 1976-02-25 Adeka Argus Chemical Co Ltd Kasozaisoseibutsu
GB1527592A (en) 1974-08-05 1978-10-04 Ici Ltd Wound dressing
US3995623A (en) 1974-12-23 1976-12-07 American Hospital Supply Corporation Multipurpose flow-directed catheter
JPS5813576B2 (ja) 1974-12-27 1983-03-14 アデカ ア−ガスカガク カブシキガイシヤ 安定化された合成高分子組成物
DE2520814A1 (de) 1975-05-09 1976-11-18 Bayer Ag Lichtstabilisierung von polyurethanen
US4281669A (en) 1975-05-09 1981-08-04 Macgregor David C Pacemaker electrode with porous system
JPS5210847A (en) 1975-07-16 1977-01-27 Nippon Steel Corp Pinch roll
US4096860A (en) 1975-10-08 1978-06-27 Mclaughlin William F Dual flow encatheter
CA1069652A (en) 1976-01-09 1980-01-15 Alain F. Carpentier Supported bioprosthetic heart valve with compliant orifice ring
US4134402A (en) 1976-02-11 1979-01-16 Mahurkar Sakharam D Double lumen hemodialysis catheter
US4072146A (en) 1976-09-08 1978-02-07 Howes Randolph M Venous catheter device
US4335723A (en) 1976-11-26 1982-06-22 The Kendall Company Catheter having inflatable retention means
US4099528A (en) 1977-02-17 1978-07-11 Sorenson Research Co., Inc. Double lumen cannula
US4140126A (en) 1977-02-18 1979-02-20 Choudhury M Hasan Method for performing aneurysm repair
US4265745A (en) 1977-05-25 1981-05-05 Teijin Limited Permselective membrane
US4182833A (en) 1977-12-07 1980-01-08 Celanese Polymer Specialties Company Cationic epoxide-amine reaction products
US4180068A (en) 1978-04-13 1979-12-25 Motion Control, Incorporated Bi-directional flow catheter with retractable trocar/valve structure
DE2960875D1 (en) 1978-04-19 1981-12-10 Ici Plc A method of preparing a tubular product by electrostatic spinning
US4284459A (en) 1978-07-03 1981-08-18 The Kendall Company Method for making a molded catheter
US4227533A (en) 1978-11-03 1980-10-14 Bristol-Myers Company Flushable urinary catheter
US4375817A (en) 1979-07-19 1983-03-08 Medtronic, Inc. Implantable cardioverter
DE3010841A1 (de) 1980-03-21 1981-10-08 Ulrich Dr.med. 6936 Haag Uthmann Katheder
US4308085A (en) 1980-07-28 1981-12-29 Jenoptik Jena Gmbh Process for the preparation of high molecular thermoplastic epoxide-amine-polyadducts
US4339369A (en) 1981-04-23 1982-07-13 Celanese Corporation Cationic epoxide-amine reaction products
US4406656A (en) 1981-06-01 1983-09-27 Brack Gillium Hattler Venous catheter having collapsible multi-lumens
US4475972A (en) 1981-10-01 1984-10-09 Ontario Research Foundation Implantable material
US4401472A (en) 1982-02-26 1983-08-30 Martin Marietta Corporation Hydraulic cement mixes and processes for improving hydraulic cement mixes
US4568329A (en) 1982-03-08 1986-02-04 Mahurkar Sakharam D Double lumen catheter
US4546499A (en) 1982-12-13 1985-10-15 Possis Medical, Inc. Method of supplying blood to blood receiving vessels
US4530113A (en) 1983-05-20 1985-07-23 Intervascular, Inc. Vascular grafts with cross-weave patterns
US4550447A (en) 1983-08-03 1985-11-05 Shiley Incorporated Vascular graft prosthesis
US4647416A (en) 1983-08-03 1987-03-03 Shiley Incorporated Method of preparing a vascular graft prosthesis
US5104399A (en) 1986-12-10 1992-04-14 Endovascular Technologies, Inc. Artificial graft and implantation method
US4571241A (en) 1983-12-16 1986-02-18 Christopher T Graham Urinary catheter with collapsible urethral tube
US4710169A (en) 1983-12-16 1987-12-01 Christopher T Graham Urinary catheter with collapsible urethral tube
US4737518A (en) 1984-04-03 1988-04-12 Takeda Chemical Industries, Ltd. Lipid derivatives, their production and use
US4562596A (en) 1984-04-25 1986-01-07 Elliot Kornberg Aortic graft, device and method for performing an intraluminal abdominal aortic aneurysm repair
US4782836A (en) 1984-05-24 1988-11-08 Intermedics, Inc. Rate adaptive cardiac pacemaker responsive to patient activity and temperature
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4662382A (en) 1985-01-16 1987-05-05 Intermedics, Inc. Pacemaker lead with enhanced sensitivity
US4762915A (en) 1985-01-18 1988-08-09 Liposome Technology, Inc. Protein-liposome conjugates
US4860751A (en) 1985-02-04 1989-08-29 Cordis Corporation Activity sensor for pacemaker control
US5223263A (en) 1988-07-07 1993-06-29 Vical, Inc. Liponucleotide-containing liposomes
CA1320724C (en) 1985-07-19 1993-07-27 Koichi Kanehira Terpene amino alcohols and medicinal uses thereof
US4701162A (en) 1985-09-24 1987-10-20 The Kendall Company Foley catheter assembly
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
DE3616824A1 (de) 1986-05-17 1987-11-19 Schering Ag Verwendung von haertbaren kunstharzmischungen fuer oberflaechenbeschichtungen und druckfarben und verfahren zu ihrer herstellung
DE3780374D1 (de) 1986-07-31 1992-08-20 Irnich Werner Frequenzadaptierender herzschrittmacher.
US4960409A (en) 1986-09-11 1990-10-02 Catalano Marc L Method of using bilumen peripheral venous catheter with adapter
JPH0829776B2 (ja) 1986-10-29 1996-03-27 東燃化学株式会社 合成樹脂製容器及びその製造用金型
US4720517A (en) 1986-11-24 1988-01-19 Ciba-Geigy Corporation Compositions stabilized with N-hydroxyiminodiacetic and dipropionic acids and esters thereof
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
DE3728917A1 (de) 1987-08-29 1989-03-09 Roth Hermann J Neue lipide mit unsymmetrisch substituierter disulfidbruecke
US4946683A (en) 1987-11-18 1990-08-07 Vestar, Inc. Multiple step entrapment/loading procedure for preparing lipophilic drug-containing liposomes
US5047540A (en) 1987-12-17 1991-09-10 Shionogi & Co., Ltd. Lipid derivatives
US5138067A (en) 1987-12-17 1992-08-11 Shionogi & Co. Ltd. Lipid derivatives
US4892540A (en) 1988-04-21 1990-01-09 Sorin Biomedica S.P.A. Two-leaflet prosthetic heart valve
US5176661A (en) 1988-09-06 1993-01-05 Advanced Cardiovascular Systems, Inc. Composite vascular catheter
US5024671A (en) 1988-09-19 1991-06-18 Baxter International Inc. Microporous vascular graft
US5200395A (en) 1988-10-18 1993-04-06 Ajinomoto Company, Inc. Pharmaceutical composition of BUF-5 for treating anemia
CA2001401A1 (en) 1988-10-25 1990-04-25 Claude Piantadosi Quaternary amine containing ether or ester lipid derivatives and therapeutic compositions
EP0465529B1 (en) 1989-03-21 1998-04-29 Vical, Inc. Expression of exogenous polynucleotide sequences in a vertebrate
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US6214804B1 (en) 1989-03-21 2001-04-10 Vical Incorporated Induction of a protective immune response in a mammal by injecting a DNA sequence
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5194654A (en) 1989-11-22 1993-03-16 Vical, Inc. Lipid derivatives of phosphonoacids for liposomal incorporation and method of use
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5101824A (en) 1990-04-16 1992-04-07 Siemens-Pacesetter, Inc. Rate-responsive pacemaker with circuitry for processing multiple sensor inputs
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5405379A (en) 1990-07-26 1995-04-11 Lane; Rodney J. Self expanding vascular endoprosthesis for aneurysms
US5693338A (en) 1994-09-29 1997-12-02 Emisphere Technologies, Inc. Diketopiperazine-based delivery systems
JPH0765267B2 (ja) 1990-08-22 1995-07-12 花王株式会社 柔軟仕上剤
DK0480667T3 (da) 1990-10-09 1996-06-10 Cook Inc Perkutan stentkonstruktion
EP0491082B1 (de) 1990-12-19 1995-04-12 Peter Dr. Ing. Osypka Herzschrittmacherleitung mit einem inneren Kanal und mit einem Elektrodenkopf
US5116360A (en) 1990-12-27 1992-05-26 Corvita Corporation Mesh composite graft
US5405363A (en) 1991-03-15 1995-04-11 Angelon Corporation Implantable cardioverter defibrillator having a smaller displacement volume
US5330768A (en) 1991-07-05 1994-07-19 Massachusetts Institute Of Technology Controlled drug delivery using polymer/pluronic blends
US5545449A (en) 1991-10-02 1996-08-13 Weyerhaeuser Company Polyether-reinforced fiber-based materials
US5151105A (en) 1991-10-07 1992-09-29 Kwan Gett Clifford Collapsible vessel sleeve implant
US5284491A (en) 1992-02-27 1994-02-08 Medtronic, Inc. Cardiac pacemaker with hysteresis behavior
US5352461A (en) 1992-03-11 1994-10-04 Pharmaceutical Discovery Corporation Self assembling diketopiperazine drug delivery system
SE9200951D0 (sv) 1992-03-27 1992-03-27 Kabi Pharmacia Ab Pharmaceutical composition containing a defined lipid system
WO1993020079A1 (en) 1992-04-06 1993-10-14 Biosite Diagnostics Incorporated Novel opiate derivatives and protein and polypeptide opiate derivative conjugates and labels
US6670178B1 (en) 1992-07-10 2003-12-30 Transkaryotic Therapies, Inc. In Vivo production and delivery of insulinotropin for gene therapy
CA2141685A1 (en) 1992-08-04 1994-02-17 Koji Naito Antiallergic composition
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
US5461223A (en) 1992-10-09 1995-10-24 Eastman Kodak Company Bar code detecting circuitry
US5300022A (en) 1992-11-12 1994-04-05 Martin Klapper Urinary catheter and bladder irrigation system
US5496362A (en) 1992-11-24 1996-03-05 Cardiac Pacemakers, Inc. Implantable conformal coil patch electrode with multiple conductive elements for cardioversion and defibrillation
US5552155A (en) 1992-12-04 1996-09-03 The Liposome Company, Inc. Fusogenic lipsomes and methods for making and using same
US5716395A (en) 1992-12-11 1998-02-10 W.L. Gore & Associates, Inc. Prosthetic vascular graft
WO1994019314A1 (en) 1993-02-19 1994-09-01 Nippon Shinyaku Co., Ltd. Glycerol derivative, device and pharmaceutical composition
US5395619A (en) 1993-03-03 1995-03-07 Liposome Technology, Inc. Lipid-polymer conjugates and liposomes
US5697953A (en) 1993-03-13 1997-12-16 Angeion Corporation Implantable cardioverter defibrillator having a smaller displacement volume
US5624976A (en) 1994-03-25 1997-04-29 Dentsply Gmbh Dental filling composition and method
US5314430A (en) 1993-06-24 1994-05-24 Medtronic, Inc. Atrial defibrillator employing transvenous and subcutaneous electrodes and method of use
DE4325848A1 (de) 1993-07-31 1995-02-02 Basf Ag Verfahren zur Herstellung von N-(2-Hydroxyethyl)-piperazin
EP0875280B1 (en) 1993-10-06 2001-08-22 The Kansai Electric Power Co., Inc. Method for removing carbon dioxide from combustion exhaust gas
US5609624A (en) 1993-10-08 1997-03-11 Impra, Inc. Reinforced vascular graft and method of making same
SE9303481L (sv) 1993-10-22 1995-04-23 Berol Nobel Ab Hygienkomposition
WO1995013033A1 (en) 1993-11-08 1995-05-18 Lazarus Harrison M Intraluminal vascular graft and method
JPH09505593A (ja) 1993-11-24 1997-06-03 メガバイオス・コーポレイション ピペラジンの両親媒性誘導体
US5595756A (en) 1993-12-22 1997-01-21 Inex Pharmaceuticals Corporation Liposomal compositions for enhanced retention of bioactive agents
US5464924A (en) 1994-01-07 1995-11-07 The Dow Chemical Company Flexible poly(amino ethers) for barrier packaging
US5844107A (en) 1994-03-23 1998-12-01 Case Western Reserve University Compacted nucleic acids and their delivery to cells
KR100315132B1 (ko) 1994-04-12 2002-06-26 질레스피 카롤 융합할수있는리포좀과이를만드는방법그리고이의용도
US6392118B1 (en) 1994-07-20 2002-05-21 Neurotech S.A. Mx-1 conditionally immortalized cells
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5641665A (en) 1994-11-28 1997-06-24 Vical Incorporated Plasmids suitable for IL-2 expression
US6071890A (en) 1994-12-09 2000-06-06 Genzyme Corporation Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy
US5965434A (en) 1994-12-29 1999-10-12 Wolff; Jon A. Amphipathic PH sensitive compounds and delivery systems for delivering biologically active compounds
US6485726B1 (en) 1995-01-17 2002-11-26 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of therapeutics
US5830430A (en) 1995-02-21 1998-11-03 Imarx Pharmaceutical Corp. Cationic lipids and the use thereof
EP0822835A1 (en) 1995-04-17 1998-02-11 Imarx Pharmaceutical Corp. Hybrid magnetic resonance contrast agents
US5772694A (en) 1995-05-16 1998-06-30 Medical Carbon Research Institute L.L.C. Prosthetic heart valve with improved blood flow
US5783383A (en) 1995-05-23 1998-07-21 The Board Of Trustees Of The Leland Stanford Junior University Method of detecting cytomegalovirus (CMV)
US5609629A (en) 1995-06-07 1997-03-11 Med Institute, Inc. Coated implantable medical device
EP0832271B8 (en) 1995-06-07 2005-03-02 INEX Pharmaceuticals Corp. Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5705385A (en) 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5607385A (en) 1995-08-17 1997-03-04 Medtronic, Inc. Device and algorithm for a combined cardiomyostimulator and a cardiac pacer-carioverter-defibrillator
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
FR2740978B1 (fr) 1995-11-10 1998-01-02 Ela Medical Sa Dispositif medical actif du type defibrillateur/cardioverteur implantable
US5874105A (en) 1996-01-31 1999-02-23 Collaborative Laboratories, Inc. Lipid vesicles formed with alkylammonium fatty acid salts
ATE273320T1 (de) 1996-04-11 2004-08-15 Univ British Columbia Fusogene liposomen
US5935936A (en) 1996-06-03 1999-08-10 Genzyme Corporation Compositions comprising cationic amphiphiles and co-lipids for intracellular delivery of therapeutic molecules
US5913848A (en) 1996-06-06 1999-06-22 Luther Medical Products, Inc. Hard tip over-the-needle catheter and method of manufacturing the same
US5677124A (en) 1996-07-03 1997-10-14 Ambion, Inc. Ribonuclease resistant viral RNA standards
US5736573A (en) 1996-07-31 1998-04-07 Galat; Alexander Lipid and water soluble derivatives of drugs
US7288266B2 (en) 1996-08-19 2007-10-30 United States Of America As Represented By The Secretary, Department Of Health And Human Services Liposome complexes for increased systemic delivery
WO1998008489A1 (en) 1996-08-26 1998-03-05 Transgene S.A. Cationic lipid-nucleic acid complexes
CN1138533C (zh) 1996-09-13 2004-02-18 利普森有限公司 脂质体
TW520297B (en) 1996-10-11 2003-02-11 Sequus Pharm Inc Fusogenic liposome composition and method
AU738384B2 (en) 1996-11-04 2001-09-20 Qiagen Gmbh Cationic reagents for transfection
US6887665B2 (en) 1996-11-14 2005-05-03 Affymetrix, Inc. Methods of array synthesis
US5985930A (en) 1996-11-21 1999-11-16 Pasinetti; Giulio M. Treatment of neurodegenerative conditions with nimesulide
US6204297B1 (en) 1996-11-26 2001-03-20 Rhodia Inc. Nonionic gemini surfactants
JPH10197978A (ja) 1997-01-09 1998-07-31 Mitsubishi Paper Mills Ltd ハロゲン化銀写真感光材料
EP0853123A1 (de) 1997-01-10 1998-07-15 Roche Diagnostics GmbH Reinigung von DNA durch Cross-Flow-Filtration
FR2760193B1 (fr) 1997-02-28 1999-05-28 Transgene Sa Lipides et complexes de lipides cationiques et de substances actives, notamment pour la transfection de cellules
US5837283A (en) 1997-03-12 1998-11-17 The Regents Of The University Of California Cationic lipid compositions targeting angiogenic endothelial cells
US5945326A (en) 1997-03-20 1999-08-31 New England Biolabs, Inc. Method for cloning and producing the Spel restriction endonuclease
JP4656675B2 (ja) 1997-05-14 2011-03-23 ユニバーシティー オブ ブリティッシュ コロンビア 脂質小胞への荷電した治療剤の高率封入
US6835395B1 (en) 1997-05-14 2004-12-28 The University Of British Columbia Composition containing small multilamellar oligodeoxynucleotide-containing lipid vesicles
US20030104044A1 (en) 1997-05-14 2003-06-05 Semple Sean C. Compositions for stimulating cytokine secretion and inducing an immune response
JPH115786A (ja) 1997-06-13 1999-01-12 Pola Chem Ind Inc 新規アミノヒドロキシプロピルピペラジン誘導体
US6067471A (en) 1998-08-07 2000-05-23 Cardiac Pacemakers, Inc. Atrial and ventricular implantable cardioverter-defibrillator and lead system
JPH1180142A (ja) 1997-09-05 1999-03-26 Pola Chem Ind Inc ジフェニルアルキル化合物の製造法
EP1021549A2 (en) 1997-09-19 2000-07-26 Sequitur, Inc. SENSE mRNA THERAPY
US6165763A (en) 1997-10-30 2000-12-26 Smithkline Beecham Corporation Ornithine carbamoyltransferase
US6096075A (en) 1998-01-22 2000-08-01 Medical Carbon Research Institute, Llc Prosthetic heart valve
US6617171B2 (en) 1998-02-27 2003-09-09 The General Hospital Corporation Methods for diagnosing and treating autoimmune disease
US6271209B1 (en) 1998-04-03 2001-08-07 Valentis, Inc. Cationic lipid formulation delivering nucleic acid to peritoneal tumors
US6176877B1 (en) 1998-04-20 2001-01-23 St. Jude Medical, Inc. Two piece prosthetic heart valve
DE19822602A1 (de) 1998-05-20 1999-11-25 Goldschmidt Ag Th Verfahren zur Herstellung von Polyaminosäureestern durch Veresterung von sauren Polyaminosäuren oder Umesterung von Polyaminosäureestern
NO313244B1 (no) 1998-07-08 2002-09-02 Crew Dev Corp Fremgangsmåte for isolering og produksjon av magnesitt eller magnesiumklorid
US6055454A (en) 1998-07-27 2000-04-25 Cardiac Pacemakers, Inc. Cardiac pacemaker with automatic response optimization of a physiologic sensor based on a second sensor
JP4898991B2 (ja) 1998-08-20 2012-03-21 クック メディカル テクノロジーズ エルエルシー 被覆付植込式医療装置
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6656498B1 (en) 1998-11-25 2003-12-02 Vanderbilt University Cationic liposomes for gene transfer
US6248725B1 (en) 1999-02-23 2001-06-19 Amgen, Inc. Combinations and methods for promoting in vivo liver cell proliferation and enhancing in vivo liver-directed gene transduction
US6379698B1 (en) 1999-04-06 2002-04-30 Isis Pharmaceuticals, Inc. Fusogenic lipids and vesicles
JP5117648B2 (ja) 1999-04-20 2013-01-16 ザ・ユニバーシティ・オブ・ブリティッシュ・コロンビア カチオン性ｐｅｇ脂質および使用方法。
AU769425B2 (en) 1999-04-23 2004-01-29 Alza Corporation Conjugate having a cleavable linkage for use in a liposome
US6169923B1 (en) 1999-04-23 2001-01-02 Pacesetter, Inc. Implantable cardioverter-defibrillator with automatic arrhythmia detection criteria adjustment
WO2000069913A1 (en) 1999-05-19 2000-11-23 Lexigen Pharmaceuticals Corp. EXPRESSION AND EXPORT OF INTERFERON-ALPHA PROTEINS AS Fc FUSION PROTEINS
US6696424B1 (en) 1999-05-28 2004-02-24 Vical Incorporated Cytofectin dimers and methods of use thereof
US6346382B1 (en) 1999-06-01 2002-02-12 Vanderbilt University Human carbamyl phosphate synthetase I polymorphism and diagnostic methods related thereto
WO2001005375A1 (en) 1999-07-16 2001-01-25 Purdue Research Foundation Vinyl ether lipids with cleavable hydrophilic headgroups
KR100669305B1 (ko) 1999-07-23 2007-01-17 제넨테크, 인크. Ｒｎａ 분해효소 및 유기용매의 작용없이 접선 흐름여과를 이용한 플라스미드 ｄｎａ 정제법
US6358278B1 (en) 1999-09-24 2002-03-19 St. Jude Medical, Inc. Heart valve prosthesis with rotatable cuff
US6371983B1 (en) 1999-10-04 2002-04-16 Ernest Lane Bioprosthetic heart valve
US7060291B1 (en) 1999-11-24 2006-06-13 Transave, Inc. Modular targeted liposomal delivery system
JP2003519199A (ja) 1999-12-30 2003-06-17 ノバルティス アクチエンゲゼルシャフト 遺伝子治療のための新規なコロイド合成ベクター
EP1261379A2 (en) 2000-02-17 2002-12-04 Genzyme Corporation Genetic modification of the lung as a portal for gene delivery
US6370434B1 (en) 2000-02-28 2002-04-09 Cardiac Pacemakers, Inc. Cardiac lead and method for lead implantation
US6565960B2 (en) 2000-06-01 2003-05-20 Shriners Hospital Of Children Polymer composite compositions
EP1297169B1 (en) 2000-06-26 2012-08-08 Ethris Gmbh Method for transfecting cells using a magnetic field
IL138474A0 (en) 2000-09-14 2001-10-31 Epox Ltd Highly branched water-soluble polyamine oligomers, process for their preparation and applications thereof
USRE43612E1 (en) 2000-10-10 2012-08-28 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US6998115B2 (en) 2000-10-10 2006-02-14 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US7427394B2 (en) 2000-10-10 2008-09-23 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
JP2004511572A (ja) 2000-10-25 2004-04-15 ザ　ユニバーシティ　オブ　ブリティッシュ　コロンビア 標的化送達のための脂質製剤
GB0028361D0 (en) 2000-11-21 2001-01-03 Glaxo Group Ltd Method of separating extra chromosomal dna from other cellular components
US20020094528A1 (en) 2000-11-29 2002-07-18 Salafsky Joshua S. Method and apparatus using a surface-selective nonlinear optical technique for detection of probe-target interations
JP2002167368A (ja) 2000-12-01 2002-06-11 Nitto Denko Corp アルキル置換デンドリマーおよびその製造法
US20050004058A1 (en) 2000-12-07 2005-01-06 Patrick Benoit Sequences upstream of the carp gene, vectors containing them and uses thereof
DE10109897A1 (de) 2001-02-21 2002-11-07 Novosom Ag Fakultativ kationische Liposomen und Verwendung dieser
US20020192721A1 (en) 2001-03-28 2002-12-19 Engeneos, Inc. Modular molecular clasps and uses thereof
KR100823815B1 (ko) 2001-04-23 2008-04-21 신에쓰 가가꾸 고교 가부시끼가이샤 에스테르 구조를 갖는 신규 3급 아민 화합물 및 그 제조방법
US6585410B1 (en) 2001-05-03 2003-07-01 The United States Of America As Represented By The Administrator Of The National Aeronautics And Space Administration Radiant temperature nulling radiometer
US7514099B2 (en) 2005-02-14 2009-04-07 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
ES2240745T3 (es) 2001-06-05 2005-10-16 Curevac Gmbh Composicion farmaceutica que contiene un arnm estabilizado y optimizado para la traduccion, adecuada como vacuna y como regenerante de tejidos.
AU2002347035B2 (en) 2001-09-28 2008-04-03 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V Micro-RNA molecules
WO2003040288A2 (de) 2001-11-09 2003-05-15 Bayer Healthcare Ag Isotopencodierte affinitätsmarker 3
DE10162480A1 (de) 2001-12-19 2003-08-07 Ingmar Hoerr Die Applikation von mRNA für den Einsatz als Therapeutikum gegen Tumorerkrankungen
DE10207178A1 (de) 2002-02-19 2003-09-04 Novosom Ag Komponenten für die Herstellung amphoterer Liposomen
DE10214983A1 (de) 2002-04-04 2004-04-08 TransMIT Gesellschaft für Technologietransfer mbH Vernebelbare Liposomen und ihre Verwendung zur pulmonalen Applikation von Wirkstoffen
US20030215395A1 (en) 2002-05-14 2003-11-20 Lei Yu Controllably degradable polymeric biomolecule or drug carrier and method of synthesizing said carrier
US7601367B2 (en) 2002-05-28 2009-10-13 Mirus Bio Llc Compositions and processes using siRNA, amphipathic compounds and polycations
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
DE10229872A1 (de) 2002-07-03 2004-01-29 Curevac Gmbh Immunstimulation durch chemisch modifizierte RNA
US20040028804A1 (en) 2002-08-07 2004-02-12 Anderson Daniel G. Production of polymeric microarrays
US20050244961A1 (en) 2002-08-22 2005-11-03 Robert Short Cell culture surface
EP1565512B1 (de) 2002-11-04 2018-07-04 Momentive Performance Materials GmbH Lineare polyamino- und/oder polyammonium-polysiloxancopolymere i
CA2506843A1 (en) 2002-11-22 2004-06-10 Novo-Nordisk A/S 2,5-diketopiperazines for the treatment of obesity
US7169892B2 (en) 2003-01-10 2007-01-30 Astellas Pharma Inc. Lipid-peptide-polymer conjugates for long blood circulation and tumor specific drug delivery systems
US8054291B2 (en) 2003-01-20 2011-11-08 Asahi Kasei Emd Corporation Pointing device
WO2004078940A2 (en) 2003-03-05 2004-09-16 Senesco Technologies, Inc. USE OF ANTISENSE OLIGONUCLEOTIDES OR siRNA TO SUPPRESS EXPRESSION OF eIF-5A1
US20040224912A1 (en) 2003-05-07 2004-11-11 Isis Pharmaceuticals Inc. Modulation of PAI-1 mRNA-binding protein expression
US7619017B2 (en) 2003-05-19 2009-11-17 Wacker Chemical Corporation Polymer emulsions resistant to biodeterioration
US7507859B2 (en) 2003-06-16 2009-03-24 Fifth Base Llc Functional synthetic molecules and macromolecules for gene delivery
CA2539670A1 (en) 2003-09-15 2005-03-31 Massachusetts Institute Of Technology Nanoliter-scale synthesis of arrayed biomaterials and screening thereof
JP4842821B2 (ja) 2003-09-15 2011-12-21 プロチバ バイオセラピューティクス インコーポレイティッド ポリエチレングリコール修飾脂質化合物およびその使用
US20050069590A1 (en) 2003-09-30 2005-03-31 Buehler Gail K. Stable suspensions for medicinal dosages
JP4814099B2 (ja) 2003-10-10 2011-11-09 パウダージェクト バクシーンズ，インコーポレイテッド 核酸構築物
WO2005037226A2 (en) 2003-10-17 2005-04-28 Georgia Tech Research Corporation Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders
KR20060111471A (ko) 2003-11-10 2006-10-27 니폰 가야꾸 가부시끼가이샤 디이모늄염 화합물 및 그의 용도
US7022214B2 (en) 2004-01-21 2006-04-04 Bio-Rad Laboratories, Inc. Carrier ampholytes of high pH range
US7556684B2 (en) 2004-02-26 2009-07-07 Construction Research & Technology Gmbh Amine containing strength improvement admixture
US20060228404A1 (en) 2004-03-04 2006-10-12 Anderson Daniel G Compositions and methods for treatment of hypertrophic tissues
WO2005121348A1 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
WO2005120152A2 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
US7670595B2 (en) 2004-06-28 2010-03-02 Merck Patent Gmbh Fc-interferon-beta fusion proteins
GB0418172D0 (en) 2004-08-13 2004-09-15 Ic Vec Ltd Vector
DE102004042546A1 (de) 2004-09-02 2006-03-09 Curevac Gmbh Kombinationstherapie zur Immunstimulation
DE102004043342A1 (de) 2004-09-08 2006-03-09 Bayer Materialscience Ag Blockierte Polyurethan-Prepolymere als Klebstoffe
EP1807050A1 (en) 2004-11-05 2007-07-18 Novosom AG Improvements in or relating to pharmaceutical compositions comprising an oligonucleotide as an active agent
GB0502482D0 (en) 2005-02-07 2005-03-16 Glaxo Group Ltd Novel compounds
US7977452B2 (en) 2005-03-28 2011-07-12 Dendritic Nanotechnologies, Inc. Janus dendrimers and dendrons
JP5777846B2 (ja) 2005-06-15 2015-09-09 マサチューセッツ インスティテュート オブ テクノロジー アミン含有脂質およびその使用
EP4174179A3 (en) 2005-08-23 2023-09-27 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
EP1948674A4 (en) 2005-11-02 2009-02-04 Protiva Biotherapeutics Inc MODIFIED SIRNA MOLECULES AND APPLICATIONS THEREOF
US7238791B1 (en) 2005-12-16 2007-07-03 Roche Diagnostics Operations, Inc. 6-monoacetylmorphine derivatives useful in immunoassay
WO2007073489A2 (en) 2005-12-22 2007-06-28 Trustees Of Boston University Molecules for gene delivery and gene therapy, and methods of use thereof
CN100569877C (zh) 2005-12-30 2009-12-16 财团法人工业技术研究院 含多uv交联反应基的分枝状结构化合物及其应用
EP2010659B1 (en) 2006-04-14 2014-06-18 CellScript, Inc. Kits and methods for generating 5' capped RNA
US9085778B2 (en) 2006-05-03 2015-07-21 VL27, Inc. Exosome transfer of nucleic acids to cells
US20070275923A1 (en) 2006-05-25 2007-11-29 Nastech Pharmaceutical Company Inc. CATIONIC PEPTIDES FOR siRNA INTRACELLULAR DELIVERY
US8808681B2 (en) 2006-06-05 2014-08-19 Massachusetts Institute Of Technology Crosslinked, degradable polymers and uses thereof
US20090186805A1 (en) 2006-07-06 2009-07-23 Aaron Thomas Tabor Compositions and Methods for Genetic Modification of Cells Having Cosmetic Function to Enhance Cosmetic Appearance
ES2293834B1 (es) 2006-07-20 2009-02-16 Consejo Superior Investig. Cientificas Compuesto con actividad inhibidora de las interacciones ubc13-uev, composiciones farmaceuticas que lo comprenden y sus aplicaciones terapeuticas.
WO2008011561A2 (en) 2006-07-21 2008-01-24 Massachusetts Institute Of Technology End-modified poly(beta-amino esters) and uses thereof
CA2665225C (en) 2006-10-03 2015-06-30 Alnylam Pharmaceuticals, Inc. Lipid containing formulations
EP2076291A2 (en) * 2006-10-12 2009-07-08 Copernicus Therapeutics, Inc. Codon optimized cftr
DE102006051516A1 (de) 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
WO2008140615A2 (en) 2006-12-21 2008-11-20 Novozymes, Inc. Modified messenger rna stabilizing sequences for expressing genes in bacterial cells
DE102007001370A1 (de) 2007-01-09 2008-07-10 Curevac Gmbh RNA-kodierte Antikörper
US8859229B2 (en) 2007-02-02 2014-10-14 Yale University Transient transfection with RNA
WO2008113364A2 (en) 2007-03-20 2008-09-25 Recepticon Aps Amino derivatives to prevent nephrotoxicity and cancer
JP5186126B2 (ja) 2007-03-29 2013-04-17 公益財団法人地球環境産業技術研究機構 新規トリアジン誘導体ならびにその製法およびそのガス分離膜としての用途
KR101539785B1 (ko) 2007-04-18 2015-07-27 코너스톤 파마슈티칼스, 인크. 리포산 유도체를 함유하는 약제학적 제형
US8678686B2 (en) 2007-05-01 2014-03-25 Pgr-Solutions Multi-chain lipophilic polyamines
US20090163705A1 (en) 2007-05-21 2009-06-25 Alnylam Pharmaceuticals, Inc. Cationic lipids
WO2009030254A1 (en) 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
US8389238B2 (en) 2007-09-12 2013-03-05 Copernicus Therapeutics, Inc. Long-term in vivo transgene expression
CA2701274A1 (en) 2007-10-02 2009-04-09 Mdrna, Inc. Lipopeptides for delivery of nucleic acids
WO2009046739A1 (en) 2007-10-09 2009-04-16 Curevac Gmbh Composition for treating prostate cancer (pca)
EP2221371B1 (en) 2007-11-22 2013-11-13 Japan Science and Technology Agency Translation regulation system in cell or artificial cell model by using low-molecular-weight rna
EP2229186A2 (en) 2007-12-04 2010-09-22 Alnylam Pharmaceuticals Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CA2710983A1 (en) 2007-12-27 2009-10-01 The Ohio State University Research Foundation Lipid nanoparticle compositions and methods of making and using the same
AU2008342535B2 (en) 2007-12-27 2015-02-05 Arbutus Biopharma Corporation Silencing of polo-like kinase expression using interfering RNA
AU2008347250A1 (en) 2008-01-02 2009-07-16 Tekmira Pharmaceuticals Corporation Screening method for selected amino lipid-containing compositions
CA2721183C (en) 2008-04-11 2019-07-16 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
DK2279254T3 (en) 2008-04-15 2017-09-18 Protiva Biotherapeutics Inc PRESENT UNKNOWN LIPID FORMS FOR NUCLEIC ACID ADMINISTRATION
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
US20090263407A1 (en) 2008-04-16 2009-10-22 Abbott Laboratories Cationic Lipids and Uses Thereof
EP2297322A1 (en) 2008-06-04 2011-03-23 The Board of Regents of The University of Texas System Modulation of gene expression through endogenous small rna targeting of gene promoters
JP5024216B2 (ja) 2008-07-23 2012-09-12 トヨタ自動車株式会社 内燃機関の点火時期制御装置及び点火時期制御方法
US20100035249A1 (en) 2008-08-05 2010-02-11 Kabushiki Kaisha Dnaform Rna sequencing and analysis using solid support
US8404198B2 (en) 2008-08-27 2013-03-26 Life Technologies Corporation Apparatus for and method of processing biological samples
WO2010037408A1 (en) 2008-09-30 2010-04-08 Curevac Gmbh Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof
ES2475065T3 (es) 2008-10-09 2014-07-10 Tekmira Pharmaceuticals Corporation Aminol�pidos mejorados y métodos para la administración de ácidos nucleicos
SG196818A1 (en) 2008-10-16 2014-02-13 Marina Biotech Inc Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics
US9080211B2 (en) 2008-10-24 2015-07-14 Epicentre Technologies Corporation Transposon end compositions and methods for modifying nucleic acids
WO2010062322A2 (en) 2008-10-27 2010-06-03 Massachusetts Institute Of Technology Modulation of the immune response
US8969353B2 (en) 2008-11-07 2015-03-03 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
KR102459839B1 (ko) 2008-11-10 2022-10-27 알닐람 파마슈티칼스 인코포레이티드 치료제 운반용 신규 지질 및 조성물
WO2010056403A1 (en) 2008-11-17 2010-05-20 Enzon Pharmaceuticals, Inc. Branched cationic lipids for nucleic acids delivery system
US9023820B2 (en) 2009-01-26 2015-05-05 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein C-III expression
US20100222489A1 (en) 2009-02-27 2010-09-02 Jiang Dayue D Copolymer composition, membrane article, and methods thereof
NZ594995A (en) 2009-03-12 2013-06-28 Alnylam Pharmaceuticals Inc LIPID FORMULATED COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF HUMAN KINESIN FAMILY MEMBER 11 (Eg5) AND VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) GENES
EP2415124B1 (en) 2009-04-02 2017-02-15 The Siemon Company Telecommunications patch panel
DK2419144T3 (da) 2009-04-17 2019-10-21 Univ Oxford Innovation Ltd Sammensætning til levering af genetisk materiale
EP2421506B1 (en) 2009-04-22 2015-08-19 Emory University Nanocarrier therapy for treating invasive tumors
KR20210031549A (ko) 2009-05-05 2021-03-19 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
SI3431076T1 (sl) 2009-06-10 2022-04-29 Arbutus Biopharma Corporation Izboljšana lipidna formulacija
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
CA2764832A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Lipid formulated dsrna targeting the pcsk9 gene
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011000106A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Improved cationic lipids and methods for the delivery of therapeutic agents
CA2767129C (en) 2009-07-01 2015-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
US8716464B2 (en) 2009-07-20 2014-05-06 Thomas W. Geisbert Compositions and methods for silencing Ebola virus gene expression
TR201907804T4 (tr) 2009-07-30 2019-06-21 Spiral Therapeutics Inc Apaf-1 inhibitör bileşikleri.
EP2459231B1 (de) 2009-07-31 2016-06-08 Ethris Gmbh Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
DE102009043342A1 (de) 2009-09-29 2011-03-31 Bayer Technology Services Gmbh Stoffe für selbstorganisierte Träger zur kontrollierten Freisetzung eines Wirkstoffs
US8326547B2 (en) 2009-10-07 2012-12-04 Nanjingjinsirui Science & Technology Biology Corp. Method of sequence optimization for improved recombinant protein expression using a particle swarm optimization algorithm
TR201901311T4 (tr) 2009-12-01 2019-02-21 Translate Bio Inc İnsan genetik hastalıklarında mRNA'nın teslimi için steroid türevi.
LT3287525T (lt) 2009-12-07 2020-02-10 The Trustees Of The University Of Pennsylvania Rnr preparatai, apimantys išgrynintą modifikuotą rnr, skirti ląstelių reprogramavimui
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
NZ600725A (en) 2009-12-18 2015-08-28 Univ British Colombia Methods and compositions for delivery of nucleic acids
EP2338520A1 (de) 2009-12-21 2011-06-29 Ludwig Maximilians Universität Konjugat mit Zielfindungsligand und dessen Verwendung
CA2785492C (en) 2009-12-23 2018-07-24 Novartis Ag Lipids, lipid compositions, and methods of using them
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
KR101967411B1 (ko) 2010-06-03 2019-04-10 알닐람 파마슈티칼스 인코포레이티드 활성제의 전달을 위한 생분해성 지질
CN101863544B (zh) 2010-06-29 2011-09-28 湖南科技大学 一种氰尿酸基重金属螯合絮凝剂及其制备方法
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
EP2600901B1 (en) 2010-08-06 2019-03-27 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
WO2012019630A1 (en) 2010-08-13 2012-02-16 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded protein
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
US20120237975A1 (en) 2010-10-01 2012-09-20 Jason Schrum Engineered nucleic acids and methods of use thereof
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
WO2012113413A1 (en) 2011-02-21 2012-08-30 Curevac Gmbh Vaccine composition comprising complexed immunostimulatory nucleic acids and antigens packaged with disulfide-linked polyethyleneglycol/peptide conjugates
WO2012135025A2 (en) 2011-03-28 2012-10-04 Massachusetts Institute Of Technology Conjugated lipomers and uses thereof
WO2012133737A1 (ja) 2011-03-31 2012-10-04 公益財団法人地球環境産業技術研究機構 架橋性アミン化合物、該化合物を用いた高分子膜及びその製造方法
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
WO2012151503A2 (en) 2011-05-04 2012-11-08 The Broad Institute, Inc. Multiplexed genetic reporter assays and compositions
CA2835428A1 (en) 2011-05-17 2012-11-22 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof for non-human vertebrates
EP2532649B1 (en) 2011-06-07 2015-04-08 Incella GmbH Amino lipids, their synthesis and uses thereof
ES2911653T3 (es) 2011-06-08 2022-05-20 Translate Bio Inc Composiciones de nanopartículas lipídicas y métodos para el suministro de ARNm
EP3998064A1 (en) 2011-06-08 2022-05-18 Translate Bio, Inc. Cleavable lipids
US9862926B2 (en) 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP3384938A1 (en) 2011-09-12 2018-10-10 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
WO2013039857A1 (en) 2011-09-12 2013-03-21 modeRNA Therapeutics Engineered nucleic acids and methods of use thereof
RS62993B1 (sr) 2011-10-03 2022-03-31 Modernatx Inc Modifikovani nukleozidi, nukleotidi, i nukleinske kiseline, i njihove upotrebe
CN104114571A (zh) 2011-10-05 2014-10-22 普洛体维生物治疗公司 用于沉默醛脱氢酶的组合物和方法
KR102272498B1 (ko) 2011-10-27 2021-07-06 메사추세츠 인스티튜트 오브 테크놀로지 약물 캡슐화 마이크로스피어를 형성할 수 있는, ｎ-말단 상에 관능화된 아미노산 유도체
US20140343129A1 (en) 2011-12-14 2014-11-20 Moderna Therapeutics, Inc. Modified nucleic acids, and acute care uses thereof
US20140378538A1 (en) 2011-12-14 2014-12-25 Moderma Therapeutics, Inc. Methods of responding to a biothreat
WO2013090648A1 (en) 2011-12-16 2013-06-20 modeRNA Therapeutics Modified nucleoside, nucleotide, and nucleic acid compositions
JP2015510495A (ja) 2011-12-21 2015-04-09 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 器官または器官移植片の生存可能性または寿命を延長する方法
EP2797634A4 (en) 2011-12-29 2015-08-05 Moderna Therapeutics Inc MODIFIED mRNA ENCODING POLYPEPTIDES PENETRATING IN CELLS
PL3144389T3 (pl) 2011-12-30 2018-10-31 Cellscript, Llc WYTWARZANIE I STOSOWANIE ZSYNTETYZOWANEGO IN VITRO ssRNA DO WPROWADZANIA DO SSACZYCH KOMÓREK W CELU INDUKCJI EFEKTU BIOLOGICZNEGO LUB BIOCHEMICZNEGO
WO2013120497A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
EP3988104A1 (en) 2012-02-24 2022-04-27 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
EP2830596B1 (en) 2012-03-29 2020-12-30 Translate Bio, Inc. Lipid-derived neutral nanoparticles
US20140275229A1 (en) 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
EP3501550A1 (en) 2012-04-02 2019-06-26 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
CN108949772A (zh) 2012-04-02 2018-12-07 现代泰克斯公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US20150050354A1 (en) 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
EP2858677B1 (en) 2012-06-08 2020-08-05 ethris GmbH Pulmonary delivery of messenger rna
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
WO2014028487A1 (en) 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
US20150307542A1 (en) 2012-10-03 2015-10-29 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
SI2922554T1 (sl) 2012-11-26 2022-06-30 Modernatx, Inc. Terminalno modificirana RNA
WO2014089486A1 (en) 2012-12-07 2014-06-12 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
WO2014093574A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
CA2897941A1 (en) 2013-01-17 2014-07-24 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
EP2968397A4 (en) 2013-03-12 2016-12-28 Moderna Therapeutics Inc DIAGNOSIS AND TREATMENT OF FIBROSIS
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
DK2970955T3 (en) 2013-03-14 2019-02-11 Translate Bio Inc METHODS FOR CLEANING MESSENGER RNA
LT2970456T (lt) 2013-03-14 2021-08-10 Translate Bio, Inc. Būdai ir kompozicijos, skirti mrnr koduojamų antikūnų pristatymui
AU2014236305B2 (en) 2013-03-14 2019-01-17 Ethris Gmbh CFTR mRNA compositions and related methods and uses
AU2014239250A1 (en) 2013-03-14 2015-08-27 Shire Human Genetic Therapies, Inc. Quantitative assessment for cap efficiency of messenger RNA
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
EP3750903A1 (en) 2013-03-14 2020-12-16 Translate Bio, Inc. Ribonucleic acids with 4'-thio-modified nucleotides and related methods
EP2970940B1 (en) 2013-03-14 2018-07-25 Translate Bio, Inc. Mrna therapeutic compositions and use to treat diseases and disorders
EA201591286A1 (ru) 2013-03-14 2016-02-29 Шир Хьюман Дженетик Терапис, Инк. Количественная оценка эффективности кэп матричной рнк
EP2983804A4 (en) 2013-03-15 2017-03-01 Moderna Therapeutics, Inc. Ion exchange purification of mrna
US20160017313A1 (en) 2013-03-15 2016-01-21 Moderna Therapeutics, Inc. Analysis of mrna heterogeneity and stability
SI2972360T1 (en) 2013-03-15 2018-08-31 Translate Bio, Inc. Synergistic improvement in the delivery of nucleic acids via mixed formulations
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
EP2971033B8 (en) 2013-03-15 2019-07-10 ModernaTX, Inc. Manufacturing methods for production of rna transcripts
US20160032273A1 (en) 2013-03-15 2016-02-04 Moderna Therapeutics, Inc. Characterization of mrna molecules
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US11377470B2 (en) 2013-03-15 2022-07-05 Modernatx, Inc. Ribonucleic acid purification
US9315472B2 (en) 2013-05-01 2016-04-19 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
US9895443B2 (en) 2013-06-26 2018-02-20 Massachusetts Institute Of Technology Multi-tailed lipids and uses thereof
SI3019619T1 (sl) 2013-07-11 2021-12-31 Modernatx, Inc. Sestave, ki zajemajo sintetične polinukleotide, ki kodirajo proteine, pozvezane s crispr, in sintetične sgrna, ter metode uporabe
CA2919226A1 (en) 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
WO2015034928A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
WO2015051173A2 (en) 2013-10-02 2015-04-09 Moderna Therapeutics, Inc Polynucleotide molecules and uses thereof
WO2015058069A1 (en) 2013-10-18 2015-04-23 Moderna Therapeutics, Inc. Compositions and methods for tolerizing cellular systems
EA201690588A1 (ru) 2013-10-22 2016-09-30 Шир Хьюман Дженетик Терапис, Инк. Доставка мрнк в цнс и ее применение
WO2015061467A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Lipid formulations for delivery of messenger rna
CN105658242A (zh) 2013-10-22 2016-06-08 夏尔人类遗传性治疗公司 用于苯丙酮尿症的mrna疗法
EP4276176A3 (en) 2013-10-22 2024-01-10 Translate Bio, Inc. Mrna therapy for argininosuccinate synthetase deficiency
WO2015085318A2 (en) 2013-12-06 2015-06-11 Moderna Therapeutics, Inc. Targeted adaptive vaccines
US20150167017A1 (en) 2013-12-13 2015-06-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
ES2712092T3 (es) 2013-12-30 2019-05-09 Curevac Ag Moléculas de ácido nucleico artificiales
US20170002060A1 (en) 2014-01-08 2017-01-05 Moderna Therapeutics, Inc. Polynucleotides for the in vivo production of antibodies
EP3590529A1 (en) 2014-03-12 2020-01-08 CureVac AG Combination of vaccination and ox40 agonists
EP3981437A1 (en) 2014-04-23 2022-04-13 ModernaTX, Inc. Nucleic acid vaccines
BR112016024632A2 (pt) 2014-04-25 2018-01-30 Shire Human Genetic Therapies métodos de purificação de rna mensageiro
WO2016054421A1 (en) 2014-10-02 2016-04-07 Protiva Biotherapeutics, Inc Compositions and methods for silencing hepatitis b virus gene expression
WO2016071857A1 (en) 2014-11-07 2016-05-12 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus expression
EP3041948B1 (en) 2014-11-10 2019-01-09 Modernatx, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
EP3218508A4 (en) 2014-11-10 2018-04-18 Modernatx, Inc. Multiparametric nucleic acid optimization
US20180000953A1 (en) 2015-01-21 2018-01-04 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
US20180085474A1 (en) 2015-01-23 2018-03-29 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
WO2016154127A2 (en) 2015-03-20 2016-09-29 Protiva Biotherapeutics, Inc. Compositions and methods for treating hypertriglyceridemia
WO2016164762A1 (en) 2015-04-08 2016-10-13 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor egf-a and intracellular domain mutants and methods of using the same
WO2016183366A2 (en) 2015-05-12 2016-11-17 Protiva Biotherapeutics, Inc. Compositions and methods for silencing expression of hepatitis d virus rna
WO2016197132A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics Inc. Treating hepatitis b virus infection using crispr
WO2016197133A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Delivering crispr therapeutics with lipid nanoparticles
WO2016201377A1 (en) 2015-06-10 2016-12-15 Moderna Therapeutics, Inc. Targeted adaptive vaccines
EP3329003A2 (en) 2015-07-29 2018-06-06 Arbutus Biopharma Corporation Compositions and methods for silencing hepatitis b virus gene expression
WO2017049286A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a morpholino linker
WO2017049275A2 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a stabilizing tail region
US20190054112A1 (en) 2015-09-18 2019-02-21 Moderna Therapeutics, Inc. Polynucleotide formulations for use in the treatment of renal diseases
EP3825400A1 (en) 2016-04-08 2021-05-26 Translate Bio Ma, Inc. Multimeric coding nucleic acid and uses thereof
MA46762A (fr) 2016-11-10 2019-09-18 Translate Bio Inc Procédé amélioré de préparation de nanoparticules lipidiques chargées d'arnm
KR20190120160A (ko) * 2016-11-10 2019-10-23 트랜슬레이트 바이오 인코포레이티드 Mrna의 전달을 위한 개선된 ice계 지질 나노입자 제형
US11173190B2 (en) * 2017-05-16 2021-11-16 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mRNA encoding CFTR

Also Published As

Publication number Publication date
MX2019010155A (es) 2020-12-10
EP3585417A2 (en) 2020-01-01
WO2018157154A3 (en) 2018-10-04
US20220193247A1 (en) 2022-06-23
MA47603A (fr) 2020-01-01
EP3585417B1 (en) 2023-02-22
US20180256741A1 (en) 2018-09-13
CA3054062A1 (en) 2018-08-30
IL268854A (en) 2019-10-31
US11253605B2 (en) 2022-02-22
WO2018157154A2 (en) 2018-08-30
AU2018224326B2 (en) 2024-01-04
AU2018224326A1 (en) 2019-09-19

Similar Documents

Publication Publication Date Title
EA201991747A1 (ru) НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
MX2020011746A (es) Terapias genicas para los trastornos lisosomales.
SG10201913575VA (en) Pharmaceutical compositions for the treatment of cystic fibrosis transmembrane conductance regulator mediated diseases
CY1124761T1 (el) Ρυθμιστες της δραστικοτητας του συμπληρωματος
EA201892581A1 (ru) Варианты комбинированной терапии ингибиторами hdac и ингибиторами pd-l1
SG10201902963PA (en) Modulators of cystic fibrosis transmembrane conductance regulator
EA201791874A1 (ru) Фторированные ингибиторы лизилоксидазоподобного фермента-2 и их применение
MX2020011748A (es) Terapias genicas para los trastornos lisosomales.
MX2020011745A (es) Terapias genicas para los trastornos lisosomales.
CY1121806T1 (el) Ιμιδαζοπυραζινονες ως αναστολεις pde1
MX2022000053A (es) Moduladores de interaccion de sestrina-gator2 y sus usos.
MX2018016038A (es) Compuestos y metodos para modular la funcion del acido ribonucleico (arn).
MX2016014773A (es) Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico.
EA201791500A1 (ru) Циклопропанкарбоксамидные модуляторы муковисцидозного трансмембранного регулятора проводимости
EA201790922A1 (ru) Ингибиторы бромодомена
CY1125150T1 (el) Ενωσεις φαινυλο-2-υδροξυ-ακετυλαμινο-2-μεθυλο-φαινυλιου
EA201892119A1 (ru) Лекарственный препарат, полученный путем комбинирования агониста fxr и arb
PH12019500751A1 (en) Carbamoyl phenylalaninol compounds and uses thereof
MX2021006564A (es) Sistemas activos dinamicos acromosomicos.
EA201791454A1 (ru) Лекарственные формы для трансдермального введения
MX2023005484A (es) Analogos de carbamoil fenilalaninol y usos de los mismos.
EA201892327A1 (ru) Ингаляционные и назальные бензодиазепины
EA201791992A1 (ru) Ингибиторы грелин-o-ацилтрансферазы
MX2018004361A (es) Nuevos compuestos para el tratamiento de la fibrosis quistica.
CY1122473T1 (el) Βελτιστοποιημενο μεσαλαζiνουχο δισκιο υψηλης δοσης