HK1220980A1 - 用於治療龐貝氏症的方法和組合物 - Google Patents
用於治療龐貝氏症的方法和組合物Info
- Publication number
- HK1220980A1 HK1220980A1 HK16108966.5A HK16108966A HK1220980A1 HK 1220980 A1 HK1220980 A1 HK 1220980A1 HK 16108966 A HK16108966 A HK 16108966A HK 1220980 A1 HK1220980 A1 HK 1220980A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- compositions
- treatment
- methods
- pompe disease
- pompe
- Prior art date
Links
Classifications
-
- 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
-
- 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
- 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
- A61K47/68—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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6835—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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
- A61K47/6871—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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting an enzyme
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/44—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material not provided for elsewhere, e.g. haptens, metals, DNA, RNA, amino acids
-
- 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
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/10—Immunoglobulins specific features characterized by their source of isolation or production
- C07K2317/14—Specific host cells or culture conditions, e.g. components, pH or temperature
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
- C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/55—Fab or Fab'
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
- C07K2317/565—Complementarity determining region [CDR]
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
- C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
- C07K2317/622—Single chain antibody (scFv)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
- C07K2317/77—Internalization into the cell
-
- 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
- C07K2317/00—Immunoglobulins specific features
- C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
- C07K2317/94—Stability, e.g. half-life, pH, temperature or enzyme-resistance
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
-
- 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)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361767016P | 2013-02-20 | 2013-02-20 | |
US201461926874P | 2014-01-13 | 2014-01-13 | |
PCT/US2014/017483 WO2014130723A1 (en) | 2013-02-20 | 2014-02-20 | Methods and compositions for treatment of pompe disease |
Publications (1)
Publication Number | Publication Date |
---|---|
HK1220980A1 true HK1220980A1 (zh) | 2017-05-19 |
Family
ID=51391819
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HK16108966.5A HK1220980A1 (zh) | 2013-02-20 | 2016-07-27 | 用於治療龐貝氏症的方法和組合物 |
Country Status (8)
Country | Link |
---|---|
US (2) | US10017581B2 (zh) |
EP (1) | EP2981551B1 (zh) |
JP (1) | JP2016514096A (zh) |
CN (1) | CN105189542A (zh) |
AU (1) | AU2014218854B2 (zh) |
CA (1) | CA2901978A1 (zh) |
HK (1) | HK1220980A1 (zh) |
WO (1) | WO2014130723A1 (zh) |
Families Citing this family (23)
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JP6178785B2 (ja) | 2011-04-01 | 2017-08-09 | イェール ユニバーシティーYale University | 細胞浸透性抗dna抗体およびdna修復を阻害するためのその使用 |
US9610362B2 (en) | 2012-03-16 | 2017-04-04 | Valerion Therapeutics, Llc | Antisense conjugates for decreasing expression of DMPK |
US10017581B2 (en) | 2013-02-20 | 2018-07-10 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
CA2936102A1 (en) * | 2014-01-13 | 2015-07-16 | Valerion Therapeutics, Llc | Internalizing moieties |
AU2015274348A1 (en) * | 2014-06-13 | 2017-01-12 | Valerion Therapeutics, Llc | Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders |
WO2016033324A1 (en) | 2014-08-27 | 2016-03-03 | Valerion Therapeutics, Llc | Internalizing moieties for treatment of cancer |
WO2016149508A1 (en) * | 2015-03-19 | 2016-09-22 | Shire Human Genetic Therapies, Inc. | Mrna therapy for pompe disease |
WO2016161086A1 (en) * | 2015-03-31 | 2016-10-06 | Duke University | Methods and compositions for treating glycogen storage diseases using agents that mimic or elevate cyclic amp |
CA2996906A1 (en) * | 2015-08-31 | 2017-03-09 | Duke University | Methods and compositions for the treatment of cytoplasmic glycogen storage disorders |
US10940125B2 (en) | 2015-09-18 | 2021-03-09 | Duke University | Methods and compositions for the treatment of steatosis-associated disorders |
WO2017099579A1 (en) * | 2015-12-07 | 2017-06-15 | Erasmus University Medical Center Rotterdam | Enzymatic replacement therapy and antisense therapy for pompe disease |
CN109803687B (zh) | 2016-06-15 | 2022-11-15 | 耶鲁大学 | 纳米载体向肿瘤的抗体介导的自催化靶向递送 |
EP3509614A4 (en) * | 2016-09-09 | 2020-04-22 | Valerion Therapeutics, LLC | METHODS AND COMPOSITIONS FOR THE TREATMENT OF LAFORA DISEASE |
EP3293259A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
AU2017322376B2 (en) * | 2016-09-12 | 2023-08-17 | Genethon | Acid-alpha glucosidase variants and uses thereof |
KR20200015932A (ko) * | 2017-06-07 | 2020-02-13 | 리제너론 파마슈티칼스 인코포레이티드 | 효소 내재화를 위한 조성물 및 방법 |
EP3730150A4 (en) * | 2017-12-22 | 2021-10-06 | Rophibio, Inc. | ANTI-STAT3 BISPECIFIC ANTIBODY WITH CELL-PENETRATING ABILITY AND THIS PHARMACEUTICAL COMPOSITION |
WO2019152806A1 (en) * | 2018-02-01 | 2019-08-08 | Yale University | Compositions and methods for enhancing nuclear translocation |
SG11202006433PA (en) * | 2018-02-05 | 2020-08-28 | Jcr Pharmaceuticals Co Ltd | Method for delivering drug to muscle |
US20210040464A1 (en) * | 2018-03-15 | 2021-02-11 | Valerion Therapeutics, Llc | Methods and compositions for treatment of polyglucosan disorders |
BR112020023145A2 (pt) * | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
US20220023434A1 (en) | 2018-12-19 | 2022-01-27 | The Board Of Trustees Of The Leland Stanford Junior University | Bifunctional Molecules for Lysosomal Targeting and Related Compositions and Methods |
JP2022546699A (ja) | 2019-08-30 | 2022-11-07 | イェール ユニバーシティー | 核酸を細胞に送達するための組成物および方法 |
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EP2823043A4 (en) | 2012-03-07 | 2016-09-07 | Amicus Therapeutics Inc | HIGHLY CONCENTRATED ALPHA-GLUCOSIDASE COMPOSITIONS FOR THE TREATMENT OF MORBUS POMPE |
US9610362B2 (en) | 2012-03-16 | 2017-04-04 | Valerion Therapeutics, Llc | Antisense conjugates for decreasing expression of DMPK |
WO2013177428A1 (en) | 2012-05-23 | 2013-11-28 | Valerion Therapeutics, Inc. | Methods for increasing muscle contractility |
WO2014130722A1 (en) | 2013-02-20 | 2014-08-28 | Valerion Therapeutics, Llc | Methods and compositions for treatment of forbes-cori disease |
US10017581B2 (en) | 2013-02-20 | 2018-07-10 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
US20140377246A1 (en) | 2013-06-19 | 2014-12-25 | Carol Ann Foundation and International Morquio Organization | Enzyme replacement therapy for treating mps vii related bone lesions using a chemically modified enzyme |
CA2936102A1 (en) | 2014-01-13 | 2015-07-16 | Valerion Therapeutics, Llc | Internalizing moieties |
AU2015274348A1 (en) | 2014-06-13 | 2017-01-12 | Valerion Therapeutics, Llc | Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders |
-
2014
- 2014-02-20 US US14/769,270 patent/US10017581B2/en not_active Expired - Fee Related
- 2014-02-20 CN CN201480018646.0A patent/CN105189542A/zh active Pending
- 2014-02-20 JP JP2015558229A patent/JP2016514096A/ja active Pending
- 2014-02-20 EP EP14753494.5A patent/EP2981551B1/en active Active
- 2014-02-20 AU AU2014218854A patent/AU2014218854B2/en not_active Ceased
- 2014-02-20 WO PCT/US2014/017483 patent/WO2014130723A1/en active Application Filing
- 2014-02-20 CA CA2901978A patent/CA2901978A1/en not_active Abandoned
-
2016
- 2016-07-27 HK HK16108966.5A patent/HK1220980A1/zh unknown
-
2018
- 2018-05-11 US US15/978,075 patent/US20180251571A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
EP2981551A4 (en) | 2016-11-09 |
US20180251571A1 (en) | 2018-09-06 |
US20160108133A1 (en) | 2016-04-21 |
CN105189542A (zh) | 2015-12-23 |
AU2014218854A1 (en) | 2015-08-20 |
EP2981551B1 (en) | 2020-06-03 |
US10017581B2 (en) | 2018-07-10 |
AU2014218854B2 (en) | 2019-01-24 |
JP2016514096A (ja) | 2016-05-19 |
EP2981551A1 (en) | 2016-02-10 |
CA2901978A1 (en) | 2014-08-28 |
WO2014130723A1 (en) | 2014-08-28 |
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