WO2013151666A3 - Modified polynucleotides for the production of biologics and proteins associated with human disease - Google Patents

Modified polynucleotides for the production of biologics and proteins associated with human disease Download PDF

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Publication number
WO2013151666A3
WO2013151666A3 PCT/US2013/030062 US2013030062W WO2013151666A3 WO 2013151666 A3 WO2013151666 A3 WO 2013151666A3 US 2013030062 W US2013030062 W US 2013030062W WO 2013151666 A3 WO2013151666 A3 WO 2013151666A3
Authority
WO
WIPO (PCT)
Prior art keywords
biologics
production
human disease
proteins associated
modified polynucleotides
Prior art date
Application number
PCT/US2013/030062
Other languages
French (fr)
Other versions
WO2013151666A2 (en
Inventor
Stephane Bancel
Tirtha Chakraborty
Antonin De Fougerolles
Sayda M. ELBASHIR
Matthias John
Atanu Roy
Susan Whoriskey
Kristy M. WOOD
Paul Hatala
Jason P. SCHRUM
Kenechi Ejebe
Jeff Lynn Ellsworth
Justin Guild
Original Assignee
modeRNA Therapeutics
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 modeRNA Therapeutics filed Critical modeRNA Therapeutics
Priority to AU2013243949A priority Critical patent/AU2013243949A1/en
Priority to JP2015504565A priority patent/JP2015518705A/en
Priority to CA2868391A priority patent/CA2868391A1/en
Priority to CN201380028773.4A priority patent/CN104411338A/en
Priority to EP13712973.0A priority patent/EP2833920A2/en
Priority to EP18208097.8A priority patent/EP3520821A1/en
Priority to JP2015504587A priority patent/JP6348482B2/en
Priority to AU2013243834A priority patent/AU2013243834A1/en
Priority to CN202010645626.3A priority patent/CN112390871A/en
Priority to US14/390,106 priority patent/US9221891B2/en
Priority to CA2868418A priority patent/CA2868418A1/en
Priority to EP13713306.2A priority patent/EP2833922B1/en
Priority to PCT/US2013/031821 priority patent/WO2013151736A2/en
Priority to CN201380028186.5A priority patent/CN104870022A/en
Priority to EP18204317.4A priority patent/EP3520820A1/en
Publication of WO2013151666A2 publication Critical patent/WO2013151666A2/en
Publication of WO2013151666A3 publication Critical patent/WO2013151666A3/en
Priority to HK15107125.6A priority patent/HK1206601A1/en
Priority to HK15107400.2A priority patent/HK1206639A1/en
Priority to US14/824,247 priority patent/US9572896B2/en
Priority to US15/403,517 priority patent/US10493167B2/en
Priority to JP2017012947A priority patent/JP6953135B6/en
Priority to AU2017232121A priority patent/AU2017232121B2/en
Priority to AU2018200374A priority patent/AU2018200374B2/en
Priority to AU2019202835A priority patent/AU2019202835A1/en
Priority to US16/442,168 priority patent/US10583203B2/en
Priority to JP2019225097A priority patent/JP2020072670A/en
Priority to US16/811,648 priority patent/US20210077634A1/en
Priority to AU2020257150A priority patent/AU2020257150A1/en
Priority to AU2021200507A priority patent/AU2021200507A1/en
Priority to JP2022020499A priority patent/JP2022078081A/en
Priority to AU2023214237A priority patent/AU2023214237A1/en
Priority to AU2023263451A priority patent/AU2023263451A1/en

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
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    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P31/04—Antibacterial agents
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    • A61P31/14—Antivirals for RNA viruses
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P39/00—General protective or antinoxious agents
    • A61P39/02—Antidotes
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/48—Drugs for disorders of the endocrine system of the pancreatic hormones
    • A61P5/50—Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Abstract

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
PCT/US2013/030062 2012-04-02 2013-03-09 Modified polynucleotides for the production of biologics and proteins associated with human disease WO2013151666A2 (en)

Priority Applications (31)

Application Number Priority Date Filing Date Title
AU2013243949A AU2013243949A1 (en) 2012-04-02 2013-03-09 Modified polynucleotides for the production of biologics and proteins associated with human disease
JP2015504565A JP2015518705A (en) 2012-04-02 2013-03-09 Modified polynucleotides for the production of biologics and proteins associated with human diseases
CA2868391A CA2868391A1 (en) 2012-04-02 2013-03-09 Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
CN201380028773.4A CN104411338A (en) 2012-04-02 2013-03-09 Modified polynucleotides for the production of biologics and proteins associated with human disease
EP13712973.0A EP2833920A2 (en) 2012-04-02 2013-03-09 Modified polynucleotides for the production of biologics and proteins associated with human disease
EP18208097.8A EP3520821A1 (en) 2012-04-02 2013-03-09 Modified polynucleotides for the production of biologics and proteins associated with human disease
EP18204317.4A EP3520820A1 (en) 2012-04-02 2013-03-15 In vivo production of proteins
JP2015504587A JP6348482B2 (en) 2012-04-02 2013-03-15 In vivo production of proteins
AU2013243834A AU2013243834A1 (en) 2012-04-02 2013-03-15 In vivo production of proteins
CN202010645626.3A CN112390871A (en) 2012-04-02 2013-03-15 In vivo production of proteins
US14/390,106 US9221891B2 (en) 2012-04-02 2013-03-15 In vivo production of proteins
CA2868418A CA2868418A1 (en) 2012-04-02 2013-03-15 In vivo production of proteins
EP13713306.2A EP2833922B1 (en) 2012-04-02 2013-03-15 In vivo production of proteins
PCT/US2013/031821 WO2013151736A2 (en) 2012-04-02 2013-03-15 In vivo production of proteins
CN201380028186.5A CN104870022A (en) 2012-04-02 2013-03-15 In vivo production of proteins
HK15107125.6A HK1206601A1 (en) 2012-04-02 2015-07-27 Modified polynucleotides for the production of biologics and proteins associated with human disease
HK15107400.2A HK1206639A1 (en) 2012-04-02 2015-08-03 In vivo production of proteins
US14/824,247 US9572896B2 (en) 2012-04-02 2015-08-12 In vivo production of proteins
US15/403,517 US10493167B2 (en) 2012-04-02 2017-01-11 In vivo production of proteins
JP2017012947A JP6953135B6 (en) 2012-04-02 2017-01-27 In vivo production of proteins
AU2017232121A AU2017232121B2 (en) 2012-04-02 2017-09-21 In vivo production of proteins
AU2018200374A AU2018200374B2 (en) 2012-04-02 2018-01-19 Modified polynucleotides for the production of biologics and proteins associated with human disease
AU2019202835A AU2019202835A1 (en) 2012-04-02 2019-04-23 In vivo production of proteins
US16/442,168 US10583203B2 (en) 2012-04-02 2019-06-14 In vivo production of proteins
JP2019225097A JP2020072670A (en) 2012-04-02 2019-12-13 In vivo production of protein
US16/811,648 US20210077634A1 (en) 2012-04-02 2020-03-06 In vivo production of proteins
AU2020257150A AU2020257150A1 (en) 2012-04-02 2020-10-23 In vivo production of proteins
AU2021200507A AU2021200507A1 (en) 2012-04-02 2021-01-28 Modified polynucleotides for the production of biologics and proteins associated with human disease
JP2022020499A JP2022078081A (en) 2012-04-02 2022-02-14 In vivo production of proteins
AU2023214237A AU2023214237A1 (en) 2012-04-02 2023-08-08 Modified polynucleotides for the production of biologics and proteins associated with human disease
AU2023263451A AU2023263451A1 (en) 2012-04-02 2023-11-07 In vivo production of proteins

Applications Claiming Priority (100)

Application Number Priority Date Filing Date Title
US201261618922P 2012-04-02 2012-04-02
US201261618953P 2012-04-02 2012-04-02
US201261618885P 2012-04-02 2012-04-02
US201261618957P 2012-04-02 2012-04-02
US201261618866P 2012-04-02 2012-04-02
US201261618961P 2012-04-02 2012-04-02
US201261618870P 2012-04-02 2012-04-02
US201261618945P 2012-04-02 2012-04-02
US201261618862P 2012-04-02 2012-04-02
US201261618896P 2012-04-02 2012-04-02
US201261618868P 2012-04-02 2012-04-02
US201261618873P 2012-04-02 2012-04-02
US201261618911P 2012-04-02 2012-04-02
US201261618878P 2012-04-02 2012-04-02
US61/618,957 2012-04-02
US61/618,878 2012-04-02
US61/618,885 2012-04-02
US61/618,922 2012-04-02
US61/618,873 2012-04-02
US61/618,862 2012-04-02
US61/618,866 2012-04-02
US61/618,868 2012-04-02
US61/618,945 2012-04-02
US61/618,870 2012-04-02
US61/618,896 2012-04-02
US61/618,953 2012-04-02
US61/618,961 2012-04-02
US61/618,911 2012-04-02
US201261648244P 2012-05-17 2012-05-17
US201261648286P 2012-05-17 2012-05-17
US61/648,286 2012-05-17
US61/648,244 2012-05-17
US201261668157P 2012-07-05 2012-07-05
US61/668,157 2012-07-05
US201261681658P 2012-08-10 2012-08-10
US201261681648P 2012-08-10 2012-08-10
US201261681667P 2012-08-10 2012-08-10
US201261681645P 2012-08-10 2012-08-10
US201261681742P 2012-08-10 2012-08-10
US201261681712P 2012-08-10 2012-08-10
US201261681647P 2012-08-10 2012-08-10
US201261681696P 2012-08-10 2012-08-10
US201261681661P 2012-08-10 2012-08-10
US201261681654P 2012-08-10 2012-08-10
US201261681687P 2012-08-10 2012-08-10
US201261681720P 2012-08-10 2012-08-10
US201261681649P 2012-08-10 2012-08-10
US201261681675P 2012-08-10 2012-08-10
US201261681650P 2012-08-10 2012-08-10
US201261681704P 2012-08-10 2012-08-10
US61/681,647 2012-08-10
US61/681,720 2012-08-10
US61/681,645 2012-08-10
US61/681,704 2012-08-10
US61/681,654 2012-08-10
US61/681,696 2012-08-10
US61/681,687 2012-08-10
US61/681,742 2012-08-10
US61/681,667 2012-08-10
US61/681,675 2012-08-10
US61/681,648 2012-08-10
US61/681,658 2012-08-10
US61/681,661 2012-08-10
US61/681,712 2012-08-10
US61/681,649 2012-08-10
US61/681,650 2012-08-10
US201261696381P 2012-09-04 2012-09-04
US61/696,381 2012-09-04
US201261709303P 2012-10-03 2012-10-03
US61/709,303 2012-10-03
US201261712490P 2012-10-11 2012-10-11
US61/712,490 2012-10-11
US201261737155P 2012-12-14 2012-12-14
US201261737213P 2012-12-14 2012-12-14
US201261737160P 2012-12-14 2012-12-14
US201261737184P 2012-12-14 2012-12-14
US201261737130P 2012-12-14 2012-12-14
US201261737168P 2012-12-14 2012-12-14
US201261737139P 2012-12-14 2012-12-14
US201261737191P 2012-12-14 2012-12-14
US201261737135P 2012-12-14 2012-12-14
US201261737134P 2012-12-14 2012-12-14
US201261737203P 2012-12-14 2012-12-14
US201261737174P 2012-12-14 2012-12-14
US201261737152P 2012-12-14 2012-12-14
US201261737147P 2012-12-14 2012-12-14
US61/737,130 2012-12-14
US61/737,155 2012-12-14
US61/737,134 2012-12-14
US61/737,174 2012-12-14
US61/737,184 2012-12-14
US61/737,191 2012-12-14
US61/737,160 2012-12-14
US61/737,213 2012-12-14
US61/737,203 2012-12-14
US61/737,168 2012-12-14
US61/737,139 2012-12-14
US61/737,135 2012-12-14
US61/737,152 2012-12-14
US61/737,147 2012-12-14

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2013/030063 Continuation WO2013151667A1 (en) 2012-04-02 2013-03-09 Modified polynucleotides

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US14/390,106 Continuation US9221891B2 (en) 2012-04-02 2013-03-15 In vivo production of proteins

Publications (2)

Publication Number Publication Date
WO2013151666A2 WO2013151666A2 (en) 2013-10-10
WO2013151666A3 true WO2013151666A3 (en) 2014-01-09

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Family Applications (1)

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PCT/US2013/030062 WO2013151666A2 (en) 2012-04-02 2013-03-09 Modified polynucleotides for the production of biologics and proteins associated with human disease

Country Status (6)

Country Link
JP (5) JP2015518705A (en)
CN (2) CN108949772A (en)
AU (4) AU2013243949A1 (en)
CA (1) CA2868391A1 (en)
HK (1) HK1206612A1 (en)
WO (1) WO2013151666A2 (en)

Cited By (2)

* Cited by examiner, † Cited by third party
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US11850305B2 (en) 2010-07-06 2023-12-26 Glaxosmithkline Biologicals Sa Method of making lipid formulations with RNA encoding immunogens
US11851660B2 (en) 2010-07-06 2023-12-26 Glaxosmithkline Biologicals Sa Immunisation of large mammals with low doses of RNA

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NZ600616A (en) 2009-12-01 2014-11-28 Shire Human Genetic Therapies Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
LT3243526T (en) 2010-07-06 2020-02-10 Glaxosmithkline Biologicals S.A. Delivery of rna to trigger multiple immune pathways
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
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