MX2020011514A - Construcciones para terapia genica y metodos para su uso. - Google Patents
Construcciones para terapia genica y metodos para su uso.Info
- Publication number
- MX2020011514A MX2020011514A MX2020011514A MX2020011514A MX2020011514A MX 2020011514 A MX2020011514 A MX 2020011514A MX 2020011514 A MX2020011514 A MX 2020011514A MX 2020011514 A MX2020011514 A MX 2020011514A MX 2020011514 A MX2020011514 A MX 2020011514A
- Authority
- MX
- Mexico
- Prior art keywords
- methods
- gene therapy
- therapy constructs
- constructs
- improved
- Prior art date
Links
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 230000001413 cellular effect Effects 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
-
- 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
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- 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
- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- 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
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/38—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against protease inhibitors of peptide structure
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
-
- 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
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- 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)
-
- 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)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- 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)
- C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
- C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
- C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
- C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/02—Thioester hydrolases (3.1.2)
- C12Y301/02022—Palmitoyl-protein hydrolase (3.1.2.22)
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Virology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Se proporcionan en la presente vectores mejorados para terapia génica y métodos de uso, en algunas formas de realización, que comprenden secuencias para la expresión mejorada y la selección celular de una proteína terapéutica.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862664741P | 2018-04-30 | 2018-04-30 | |
US201862688640P | 2018-06-22 | 2018-06-22 | |
US201862744068P | 2018-10-10 | 2018-10-10 | |
PCT/US2019/030076 WO2019213180A1 (en) | 2018-04-30 | 2019-04-30 | Gene therapy constructs and methods of use |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2020011514A true MX2020011514A (es) | 2020-12-09 |
Family
ID=66669057
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2020011514A MX2020011514A (es) | 2018-04-30 | 2019-04-30 | Construcciones para terapia genica y metodos para su uso. |
Country Status (16)
Country | Link |
---|---|
US (3) | US10874750B2 (es) |
EP (1) | EP3788064A1 (es) |
JP (1) | JP2021521851A (es) |
KR (1) | KR20210005154A (es) |
CN (1) | CN112513071A (es) |
AU (1) | AU2019261982A1 (es) |
BR (1) | BR112020021962A2 (es) |
CA (1) | CA3098674A1 (es) |
CL (1) | CL2020002809A1 (es) |
CO (1) | CO2020014729A2 (es) |
MX (1) | MX2020011514A (es) |
PH (1) | PH12020551818A1 (es) |
SG (1) | SG11202010533WA (es) |
TW (2) | TW202344691A (es) |
WO (1) | WO2019213180A1 (es) |
ZA (1) | ZA202006905B (es) |
Families Citing this family (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
SG11202010533WA (en) * | 2018-04-30 | 2020-11-27 | Amicus Therapeutics Inc | Gene therapy constructs and methods of use |
CN113631182A (zh) | 2018-10-10 | 2021-11-09 | 阿米库斯治疗学公司 | 二硫键稳定的多肽组合物和使用方法 |
CA3154189A1 (en) * | 2019-10-10 | 2021-04-15 | Hung Do | Variant igf2 constructs |
MX2022005916A (es) * | 2019-11-19 | 2022-08-04 | Asklepios Biopharmaceutical Inc | Virus adenoasociado terapeutico que comprende promotores especificos del higado para el tratamiento de la enfermedad de pompe y los trastornos liposomales. |
EP4149519A4 (en) * | 2020-05-14 | 2024-06-12 | The Trustees of The University of Pennsylvania | COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE |
BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
WO2022026410A2 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
WO2022104186A1 (en) * | 2020-11-16 | 2022-05-19 | Tavotek Biotherapeutics (Hong Kong) Limited | A shielded and homing bispecific antibody that simultaneously inhibits angiogenic pathway targets and her2 family proteins and uses thereof |
AU2021392642A1 (en) | 2020-12-01 | 2023-06-22 | The Trustees Of The University Of Pennsylvania | Compositions and uses thereof for treatment of angelman syndrome |
WO2023086939A1 (en) * | 2021-11-12 | 2023-05-19 | Amicus Therapeutics, Inc. | Compositions and methods for treating mucopolysaccharidosis iiia |
WO2023091949A2 (en) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
WO2024151982A1 (en) | 2023-01-13 | 2024-07-18 | Amicus Therapeutics, Inc. | Gene therapy constructs for the treatment of pompe disease |
Family Cites Families (33)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6087164A (en) * | 1997-10-03 | 2000-07-11 | Yissum Research Development Company Of The Hebrew University Of Jerusalem | Methods and compositions for inducing tumor-specific cytotoxicity |
AU1086501A (en) | 1999-10-15 | 2001-04-30 | Carnegie Institution Of Washington | Rna interference pathway genes as tools for targeted genetic interference |
US6326193B1 (en) | 1999-11-05 | 2001-12-04 | Cambria Biosciences, Llc | Insect control agent |
EP1118334A1 (en) | 2000-01-11 | 2001-07-25 | Aventis Behring Gesellschaft mit beschränkter Haftung | Method for the production of conjugates and uses thereof for the prevention and treatment of allergic reactions and autoimmune diseases |
WO2001096584A2 (en) | 2000-06-12 | 2001-12-20 | Akkadix Corporation | Materials and methods for the control of nematodes |
IL158623A0 (en) | 2001-04-30 | 2004-05-12 | Symbiontics Inc | Subcellular targeting of therapeutic proteins |
US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
WO2003032727A1 (en) * | 2001-10-16 | 2003-04-24 | Symbiontics Inc. | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
DE10237639A1 (de) * | 2002-08-13 | 2004-02-26 | Martin-Luther-Universität Halle-Wittenberg | Verfahren zur Herstellung von virusanalogen Partikeln durch in vitro-Assemblierung von Kapsomeren oder von Kapsomeren, an die biologisch aktive Makromoleküle gebunden sind, bei gleichzeitiger Verpackung der Makromoleküle |
EP1587923B1 (en) | 2003-01-22 | 2011-08-24 | Duke University | Improved constructs for expressing lysosomal polypeptides |
WO2004098648A1 (en) | 2003-05-01 | 2004-11-18 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
GB0314856D0 (en) * | 2003-06-25 | 2003-07-30 | Unitargeting Res As | Protein expression system |
WO2005033134A2 (en) | 2003-09-30 | 2005-04-14 | Regeneron Pharmaceuticals, Inc. | Secreted protein therapeutics and uses thereof |
US20050244400A1 (en) | 2004-02-10 | 2005-11-03 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
ES2242542B1 (es) * | 2004-04-30 | 2006-12-16 | Consejo Superior De Investigaciones Cientificas | Procedimiento para la produccion en levaduras de capsidas virales vacias compuestas por proteinas derivadas de pvp2 del virus causante de la enfermedad de la bursitis infecciosa (ibdv). |
WO2006007560A2 (en) | 2004-07-01 | 2006-01-19 | University Of Pennsylvania | Targeted protein replacement for the treatment of lysosomal storage disorders |
CN1903374A (zh) * | 2005-07-27 | 2007-01-31 | 中国人民解放军军事医学科学院生物医学分析中心 | 大肠杆菌在制备肿瘤诊断或治疗药物中的用途 |
CA2669347A1 (en) | 2006-11-13 | 2008-05-29 | Zystor Therapeutics, Inc. | Methods for treating pompe disease |
HUE034850T2 (en) | 2008-05-07 | 2018-03-28 | Biomarin Pharm Inc | Lysosomal targeting peptides and their use |
PT3912643T (pt) | 2009-02-13 | 2023-01-12 | Immunomedics Inc | Imunoconjugados com uma ligação intracelular clivável |
US9279007B2 (en) * | 2010-11-22 | 2016-03-08 | Amicus Therapeutics, Inc. | Signal sequences to improve protein expressions and secretion of recombinant enzymes and other proteins |
PL2646044T3 (pl) * | 2010-11-30 | 2020-03-31 | Orphazyme A/S | Sposoby zwiększenia aktywności wewnątrzkomórkowej Hsp70 |
US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
EP2687597B1 (en) | 2011-03-16 | 2018-12-26 | Amano Enzyme Inc. | Modified alpha-glucosidase and applications of same |
DK2714752T3 (en) | 2011-05-27 | 2018-02-26 | Amicus Therapeutics Inc | PROCEDURES FOR THE CONNECTION OF TARGETING PEPTIDES TO RECOMBINANT LYSOSOMAL ENZYMES FOR IMPROVED TREATMENT OF LYSOSOMAL DISPOSAL DISEASES |
US9387236B2 (en) * | 2011-06-10 | 2016-07-12 | Prothera Inc. | Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases |
LT3115372T (lt) | 2012-11-27 | 2019-06-25 | Biomarin Pharmaceutical Inc. | Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas |
ES2804594T3 (es) * | 2013-03-15 | 2021-02-08 | Amicus Therapeutics Inc | Reticuladores químicos |
WO2015060722A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
WO2015085238A1 (en) | 2013-12-05 | 2015-06-11 | The Regents Of The University Of California, A California Corporation | Inhibitors of lpxc |
SG11202010533WA (en) | 2018-04-30 | 2020-11-27 | Amicus Therapeutics Inc | Gene therapy constructs and methods of use |
-
2019
- 2019-04-30 SG SG11202010533WA patent/SG11202010533WA/en unknown
- 2019-04-30 TW TW112122938A patent/TW202344691A/zh unknown
- 2019-04-30 CN CN201980029113.5A patent/CN112513071A/zh active Pending
- 2019-04-30 EP EP19727170.3A patent/EP3788064A1/en active Pending
- 2019-04-30 WO PCT/US2019/030076 patent/WO2019213180A1/en unknown
- 2019-04-30 CA CA3098674A patent/CA3098674A1/en active Pending
- 2019-04-30 BR BR112020021962-2A patent/BR112020021962A2/pt unknown
- 2019-04-30 US US16/399,979 patent/US10874750B2/en active Active
- 2019-04-30 JP JP2020560355A patent/JP2021521851A/ja active Pending
- 2019-04-30 TW TW108115137A patent/TWI808169B/zh active
- 2019-04-30 MX MX2020011514A patent/MX2020011514A/es unknown
- 2019-04-30 KR KR1020207033891A patent/KR20210005154A/ko unknown
- 2019-04-30 AU AU2019261982A patent/AU2019261982A1/en active Pending
-
2020
- 2020-10-29 CL CL2020002809A patent/CL2020002809A1/es unknown
- 2020-10-30 PH PH12020551818A patent/PH12020551818A1/en unknown
- 2020-11-05 ZA ZA2020/06905A patent/ZA202006905B/en unknown
- 2020-11-27 CO CONC2020/0014729A patent/CO2020014729A2/es unknown
- 2020-12-18 US US17/127,001 patent/US11491243B2/en active Active
-
2022
- 2022-11-07 US US18/053,160 patent/US20230233711A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
TW202344691A (zh) | 2023-11-16 |
TWI808169B (zh) | 2023-07-11 |
CN112513071A (zh) | 2021-03-16 |
SG11202010533WA (en) | 2020-11-27 |
ZA202006905B (en) | 2022-03-30 |
PH12020551818A1 (en) | 2021-07-26 |
CO2020014729A2 (es) | 2020-12-21 |
EP3788064A1 (en) | 2021-03-10 |
US20190343968A1 (en) | 2019-11-14 |
CL2020002809A1 (es) | 2021-04-30 |
CA3098674A1 (en) | 2019-11-07 |
TW202003053A (zh) | 2020-01-16 |
AU2019261982A1 (en) | 2020-10-15 |
US10874750B2 (en) | 2020-12-29 |
US11491243B2 (en) | 2022-11-08 |
KR20210005154A (ko) | 2021-01-13 |
US20210162075A1 (en) | 2021-06-03 |
WO2019213180A1 (en) | 2019-11-07 |
US20230233711A1 (en) | 2023-07-27 |
JP2021521851A (ja) | 2021-08-30 |
BR112020021962A2 (pt) | 2021-01-26 |
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