CO2019013332A2 - Productos terapéuticos para la enfermedad de almacenamiento de glucógeno de tipo iii - Google Patents

Productos terapéuticos para la enfermedad de almacenamiento de glucógeno de tipo iii

Info

Publication number
CO2019013332A2
CO2019013332A2 CONC2019/0013332A CO2019013332A CO2019013332A2 CO 2019013332 A2 CO2019013332 A2 CO 2019013332A2 CO 2019013332 A CO2019013332 A CO 2019013332A CO 2019013332 A2 CO2019013332 A2 CO 2019013332A2
Authority
CO
Colombia
Prior art keywords
alpha
lga
therapeutics
activity
type iii
Prior art date
Application number
CONC2019/0013332A
Other languages
English (en)
Spanish (es)
Inventor
Kiyoshi Tachikawa
Carlos Gustavo Perez-Garcia
Padmanabh Chivukula
Hari Prakash Bhaskaran
Christian W Cobaugh
Sean Christopher Daugherty
Original Assignee
Ultragenyx Pharmaceutical Inc
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 Ultragenyx Pharmaceutical Inc filed Critical Ultragenyx Pharmaceutical Inc
Publication of CO2019013332A2 publication Critical patent/CO2019013332A2/es

Links

Classifications

    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
    • C12N9/1051—Hexosyltransferases (2.4.1)
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2451—Glucanases acting on alpha-1,6-glucosidic bonds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01025—4-Alpha-glucanotransferase (2.4.1.25)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01033—Amylo-alpha-1,6-glucosidase (3.2.1.33)
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CONC2019/0013332A 2017-05-31 2019-11-27 Productos terapéuticos para la enfermedad de almacenamiento de glucógeno de tipo iii CO2019013332A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762513350P 2017-05-31 2017-05-31
PCT/US2018/035477 WO2018222926A1 (en) 2017-05-31 2018-05-31 Therapeutics for glycogen storage disease type iii

Publications (1)

Publication Number Publication Date
CO2019013332A2 true CO2019013332A2 (es) 2020-02-18

Family

ID=64456114

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2019/0013332A CO2019013332A2 (es) 2017-05-31 2019-11-27 Productos terapéuticos para la enfermedad de almacenamiento de glucógeno de tipo iii

Country Status (14)

Country Link
US (2) US11377643B2 (https=)
EP (1) EP3630964B1 (https=)
JP (2) JP7284101B2 (https=)
KR (1) KR102636537B1 (https=)
CN (1) CN110719954B (https=)
AR (1) AR112706A1 (https=)
AU (1) AU2018278315B2 (https=)
BR (1) BR112019025224A2 (https=)
CA (1) CA3063907A1 (https=)
CO (1) CO2019013332A2 (https=)
ES (1) ES3048960T3 (https=)
MX (1) MX2019014412A (https=)
TW (1) TWI794237B (https=)
WO (1) WO2018222926A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
BR112019025224A2 (pt) * 2017-05-31 2020-12-08 Ultragenyx Pharmaceutical Inc. Terapêutica para doença de armazenamento de glicogênio tipo iii
AU2019394996B2 (en) 2018-12-06 2025-11-06 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
CN110314407A (zh) * 2019-08-01 2019-10-11 山东新希望六和集团有限公司 一种脂肪快速提取装置
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
IT202000003371A1 (it) * 2020-02-19 2021-08-19 Enea Agenzia Naz Per Le Nuove Tecnologie Lenergia E Lo Sviluppo Economico Sostenibile Composto per il trattamento di una glicogenosi
US12351834B2 (en) 2020-03-03 2025-07-08 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
AU2021236068A1 (en) * 2020-03-09 2022-10-06 Arcturus Therapeutics, Inc. Compositions and methods for inducing immune responses
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
EP4196128A4 (en) 2020-08-14 2024-06-12 Arcturus Therapeutics, Inc. METHOD FOR FREEZE DILIZATION OF LIPID NANOPARTICLES
JP2024517427A (ja) * 2021-04-20 2024-04-22 アンジャリウム バイオサイエンシズ エージー アミロ－α－１，６－グルコシダーゼ、４－α－グルカノトランスフェラーゼをコードするＤＮＡ分子の組成物、その作製の方法、及びその使用の方法
CN118369335A (zh) * 2022-01-06 2024-07-19 上海吉量医药工程有限公司 N1位修饰假尿嘧啶核苷及其在mRNA合成中的应用
CN115960926A (zh) * 2022-12-30 2023-04-14 南方科技大学 一种mRNA及含有mRNA的药物
WO2025080939A1 (en) 2023-10-13 2025-04-17 Ultragenyx Pharmaceutical, Inc. Compositions and methods for treating conditions associated with cartilage oligomeric matrix protein (comp) mutations
WO2025128853A2 (en) 2023-12-13 2025-06-19 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with ube3a overexpression

Family Cites Families (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2340499T3 (es) 2001-06-05 2010-06-04 Curevac Gmbh Arnm de antigeno tumoral estabilizado con un contenido de g/c aumentado.
JP2006508939A (ja) 2002-10-29 2006-03-16 エンジーン， インコーポレイテッド 癌処置のための組成物
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
US8304529B2 (en) 2006-07-28 2012-11-06 Life Technologies Corporation Dinucleotide MRNA cap analogs
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
ES2535419T3 (es) 2007-12-27 2015-05-11 Protiva Biotherapeutics Inc. Silenciamiento de expresión de quinasa tipo polo usando ARN interferente
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
ES2535461T3 (es) 2008-07-08 2015-05-11 Duke University Método de tratamiento de la enfermedad por almacenamiento de glucógeno
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
CA2740000C (en) 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
WO2010048536A2 (en) 2008-10-23 2010-04-29 Alnylam Pharmaceuticals, Inc. Processes for preparing lipids
CN111808084A (zh) 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
SG10201911942UA (en) 2009-05-05 2020-02-27 Muthiah Manoharan Lipid compositions
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
KR20190039347A (ko) 2010-06-03 2019-04-10 알닐람 파마슈티칼스 인코포레이티드 활성제의 전달을 위한 생분해성 지질
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
RU2013154295A (ru) * 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
DE19216461T1 (de) 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
EP3144389B1 (en) 2011-12-30 2018-05-09 Cellscript, Llc Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
WO2014093924A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
EP4331620A3 (en) * 2012-12-07 2024-12-04 Translate Bio, Inc. Lipidic nanoparticles for mrna delivery
WO2014130722A1 (en) * 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of forbes-cori disease
US20160022840A1 (en) 2013-03-09 2016-01-28 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
EP3750903A1 (en) 2013-03-14 2020-12-16 Translate Bio, Inc. Ribonucleic acids with 4'-thio-modified nucleotides and related methods
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
JP6506749B2 (ja) 2013-10-22 2019-04-24 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのｍＲＮＡ療法
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2015192092A1 (en) * 2014-06-13 2015-12-17 Valerion Therapeutics, Llc Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders
AU2015338923B2 (en) * 2014-11-02 2021-10-21 Arcturus Therapeutics, Inc. Messenger UNA molecules and uses thereof
WO2016077125A1 (en) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
WO2016077123A1 (en) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Multiparametric nucleic acid optimization
US9709490B2 (en) 2014-12-08 2017-07-18 Canon Kabushiki Kaisha Refractive index distribution measuring method, refractive index distribution measuring apparatus, and optical element manufacturing method
WO2017054086A1 (en) * 2015-10-01 2017-04-06 Exerkine Corporation Treatment of genetic myopathies using bioengineered exosomes
WO2017100551A1 (en) 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION
IL308824A (en) 2016-08-17 2024-01-01 Factor Bioscience Inc Nucleic acid products and methods of administration thereof
CA3040337A1 (en) 2016-10-26 2018-05-03 Curevac Ag Lipid nanoparticle mrna vaccines
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10227302B2 (en) 2017-02-09 2019-03-12 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
BR112019025224A2 (pt) * 2017-05-31 2020-12-08 Ultragenyx Pharmaceutical Inc. Terapêutica para doença de armazenamento de glicogênio tipo iii

Also Published As

Publication number Publication date
BR112019025224A2 (pt) 2020-12-08
CA3063907A1 (en) 2018-12-06
US11377643B2 (en) 2022-07-05
AU2018278315B2 (en) 2024-01-18
EP3630964C0 (en) 2025-08-13
US20220340886A1 (en) 2022-10-27
JP2023075248A (ja) 2023-05-30
JP7284101B2 (ja) 2023-05-30
CN110719954A (zh) 2020-01-21
KR102636537B1 (ko) 2024-02-15
TWI794237B (zh) 2023-03-01
TW201903151A (zh) 2019-01-16
MX2019014412A (es) 2020-02-10
AU2018278315A1 (en) 2019-12-12
EP3630964A4 (en) 2021-03-03
WO2018222926A1 (en) 2018-12-06
AR112706A1 (es) 2019-12-04
EP3630964A1 (en) 2020-04-08
ES3048960T3 (en) 2025-12-12
JP2020522244A (ja) 2020-07-30
CN110719954B (zh) 2023-12-26
US20200149017A1 (en) 2020-05-14
EP3630964B1 (en) 2025-08-13
KR20200014319A (ko) 2020-02-10

Similar Documents

Publication Publication Date Title
CO2019013332A2 (es) Productos terapéuticos para la enfermedad de almacenamiento de glucógeno de tipo iii
CO2020015255A2 (es) Métodos y composiciones para tratar el cáncer
MX2024009571A (es) Vectores virales que codifican variantes del factor de coagulacion ix (fix) recombinantes con mayor expresion para la terapia genica de hemofilia b.
CL2019003390A1 (es) Proteínas de anticuerpo injertadas con citocina y métodos de uso en el tratamiento del cáncer
MX2021001837A (es) Constructos del receptor de celulas t y usos de los mismos.
CO2019011450A2 (es) Expresión transgénica selectiva de tejidos
EA202091995A1 (ru) Композиции и способы лечения не связанного с возрастом нарушения слуха у субъекта-человека
TN2016000553A1 (en) Methods and compositions for treating ulcers
WO2018068008A8 (en) T cells expressing membrane-anchored il-12 for the treatment of cancer
MY194175A (en) Variant rnai
MX2017000821A (es) Moleculas que activan selectivamente celulas t reguladoras para el tratamiento de enfermedades autoinmunes.
MX2015012176A (es) Metodos de usar zscan4 para rejuvenecer celulas humanas.
MX2020005849A (es) Células madre y progenitoras hematopoyéticas humanas cd34+ modificadas con crispr-cas9 y sus usos.
PE20151893A1 (es) Tratamiento del cancer usando anticuerpos que se unen a grp78 en la superficie celular
AR118980A1 (es) Moléculas de unión a tigit y pd-1 / tigit
UY37967A (es) Compuestos y métodos para la reducción de la expresión de snca
MX2017011794A (es) Composiciones y metodos para diagnosticos y tratamiento del cancer.
NI201500057A (es) Métodos para reducir riesgos asociados con insuficiencia cardiaca y factores asociados
BR112016029558A2 (pt) terapia de células-tronco em patologias endometriais
CL2023003260A1 (es) Composiciones y métodos para el tratamiento de la hipoacusia neurosensorial mediante sistemas de vector dual de estereocilina.
MX2021010543A (es) Receptores de linfocitos t y metodos de uso de estos.
CO2020013050A2 (es) Enzimas quinureninasa humanas y sus usos
BR112018003928A2 (pt) métodos para o tratamento de doenças
CL2020000734A1 (es) Expansión y uso de fracciones de células nk ampliadas.
MX2019000235A (es) Eliminacion de la cistina mediada por enzimas humanas.