IL291554B2 - Administering therapeutic agents to the central nervous system - Google Patents
Administering therapeutic agents to the central nervous systemInfo
- Publication number
- IL291554B2 IL291554B2 IL291554A IL29155422A IL291554B2 IL 291554 B2 IL291554 B2 IL 291554B2 IL 291554 A IL291554 A IL 291554A IL 29155422 A IL29155422 A IL 29155422A IL 291554 B2 IL291554 B2 IL 291554B2
- Authority
- IL
- Israel
- Prior art keywords
- composition
- disease
- fold
- brain
- type
- Prior art date
Links
- 239000003814 drug Substances 0.000 title 1
- 229940124597 therapeutic agent Drugs 0.000 title 1
- 210000001519 tissue Anatomy 0.000 claims 23
- 102000004190 Enzymes Human genes 0.000 claims 14
- 108090000790 Enzymes Proteins 0.000 claims 14
- 210000004556 brain Anatomy 0.000 claims 13
- 210000003169 central nervous system Anatomy 0.000 claims 10
- 230000002093 peripheral effect Effects 0.000 claims 8
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 6
- 208000008955 Mucolipidoses Diseases 0.000 claims 6
- 230000002132 lysosomal effect Effects 0.000 claims 5
- 208000005340 mucopolysaccharidosis III Diseases 0.000 claims 5
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 claims 4
- 208000028226 Krabbe disease Diseases 0.000 claims 4
- 210000000278 spinal cord Anatomy 0.000 claims 4
- 102100022548 Beta-hexosaminidase subunit alpha Human genes 0.000 claims 3
- 208000001905 GM2 Gangliosidoses Diseases 0.000 claims 3
- 201000008905 GM2 gangliosidosis Diseases 0.000 claims 3
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 3
- 101001045440 Homo sapiens Beta-hexosaminidase subunit alpha Proteins 0.000 claims 3
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 claims 3
- 206010072927 Mucolipidosis type I Diseases 0.000 claims 3
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 claims 3
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims 3
- 210000001638 cerebellum Anatomy 0.000 claims 3
- 210000004720 cerebrum Anatomy 0.000 claims 3
- 230000000694 effects Effects 0.000 claims 3
- 230000002255 enzymatic effect Effects 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 3
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 3
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 claims 3
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 claims 3
- 210000002569 neuron Anatomy 0.000 claims 3
- 239000004094 surface-active agent Substances 0.000 claims 3
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 2
- 102100022146 Arylsulfatase A Human genes 0.000 claims 2
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 claims 2
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical compound OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 claims 2
- 208000001948 Farber Lipogranulomatosis Diseases 0.000 claims 2
- 208000033149 Farber disease Diseases 0.000 claims 2
- 208000015872 Gaucher disease Diseases 0.000 claims 2
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 claims 2
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 claims 2
- 206010056893 Mucopolysaccharidosis VII Diseases 0.000 claims 2
- 229910019142 PO4 Inorganic materials 0.000 claims 2
- 208000025816 Sanfilippo syndrome type A Diseases 0.000 claims 2
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 claims 2
- 102000005936 beta-Galactosidase Human genes 0.000 claims 2
- 108010005774 beta-Galactosidase Proteins 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 201000004502 glycogen storage disease II Diseases 0.000 claims 2
- 201000008977 glycoproteinosis Diseases 0.000 claims 2
- 108010089932 heparan sulfate sulfatase Proteins 0.000 claims 2
- 239000003018 immunosuppressive agent Substances 0.000 claims 2
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims 2
- 201000007769 mucolipidosis Diseases 0.000 claims 2
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 claims 2
- 208000012226 mucopolysaccharidosis type IIIA Diseases 0.000 claims 2
- 208000012227 mucopolysaccharidosis type IIIB Diseases 0.000 claims 2
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 2
- 239000010452 phosphate Substances 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- 102100035028 Alpha-L-iduronidase Human genes 0.000 claims 1
- 208000029602 Alpha-N-acetylgalactosaminidase deficiency Diseases 0.000 claims 1
- 102100034561 Alpha-N-acetylglucosaminidase Human genes 0.000 claims 1
- 206010068220 Aspartylglucosaminuria Diseases 0.000 claims 1
- 206010011777 Cystinosis Diseases 0.000 claims 1
- 208000011518 Danon disease Diseases 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 201000008892 GM1 Gangliosidosis Diseases 0.000 claims 1
- 208000017462 Galactosialidosis Diseases 0.000 claims 1
- 208000001500 Glycogen Storage Disease Type IIb Diseases 0.000 claims 1
- 208000035148 Glycogen storage disease due to LAMP-2 deficiency Diseases 0.000 claims 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 1
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 claims 1
- 108700037017 Hyaluronidase Deficiency Proteins 0.000 claims 1
- 208000005503 Hyaluronidase deficiency Diseases 0.000 claims 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 1
- 206010072928 Mucolipidosis type II Diseases 0.000 claims 1
- 206010072929 Mucolipidosis type III Diseases 0.000 claims 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims 1
- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 claims 1
- 208000025797 Mucopolysaccharidosis type 4A Diseases 0.000 claims 1
- 208000025923 Mucopolysaccharidosis type 4B Diseases 0.000 claims 1
- 208000025915 Mucopolysaccharidosis type 6 Diseases 0.000 claims 1
- 208000000149 Multiple Sulfatase Deficiency Disease Diseases 0.000 claims 1
- 208000035032 Multiple sulfatase deficiency Diseases 0.000 claims 1
- 102100031688 N-acetylgalactosamine-6-sulfatase Human genes 0.000 claims 1
- 102100027661 N-sulphoglucosamine sulphohydrolase Human genes 0.000 claims 1
- 208000014060 Niemann-Pick disease Diseases 0.000 claims 1
- 201000000788 Niemann-Pick disease type C1 Diseases 0.000 claims 1
- 201000000785 Niemann-Pick disease type C2 Diseases 0.000 claims 1
- 208000013608 Salla disease Diseases 0.000 claims 1
- 208000021811 Sandhoff disease Diseases 0.000 claims 1
- 208000025802 Sanfilippo syndrome type C Diseases 0.000 claims 1
- 208000025804 Sanfilippo syndrome type D Diseases 0.000 claims 1
- 208000000828 Sialic Acid Storage Disease Diseases 0.000 claims 1
- 208000017460 Sialidosis type 2 Diseases 0.000 claims 1
- 201000001828 Sly syndrome Diseases 0.000 claims 1
- 210000001744 T-lymphocyte Anatomy 0.000 claims 1
- 208000022292 Tay-Sachs disease Diseases 0.000 claims 1
- 108700001567 Type I Schindler Disease Proteins 0.000 claims 1
- 208000026589 Wolman disease Diseases 0.000 claims 1
- 230000002411 adverse Effects 0.000 claims 1
- 108010009380 alpha-N-acetyl-D-glucosaminidase Proteins 0.000 claims 1
- 201000008333 alpha-mannosidosis Diseases 0.000 claims 1
- 201000006486 beta-mannosidosis Diseases 0.000 claims 1
- 210000000133 brain stem Anatomy 0.000 claims 1
- 239000006172 buffering agent Substances 0.000 claims 1
- 208000024042 cholesterol ester storage disease Diseases 0.000 claims 1
- 208000013760 cholesteryl ester storage disease Diseases 0.000 claims 1
- 201000008049 fucosidosis Diseases 0.000 claims 1
- 108020001507 fusion proteins Proteins 0.000 claims 1
- 102000037865 fusion proteins Human genes 0.000 claims 1
- 229960003444 immunosuppressant agent Drugs 0.000 claims 1
- 230000001861 immunosuppressant effect Effects 0.000 claims 1
- 229940125721 immunosuppressive agent Drugs 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 210000003734 kidney Anatomy 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 230000004807 localization Effects 0.000 claims 1
- 210000004705 lumbosacral region Anatomy 0.000 claims 1
- 208000020460 mucolipidosis II alpha/beta Diseases 0.000 claims 1
- 208000020468 mucolipidosis III alpha/beta Diseases 0.000 claims 1
- 208000000690 mucopolysaccharidosis VI Diseases 0.000 claims 1
- 208000036710 mucopolysaccharidosis type 3A Diseases 0.000 claims 1
- 208000036709 mucopolysaccharidosis type 3B Diseases 0.000 claims 1
- 208000036707 mucopolysaccharidosis type 3C Diseases 0.000 claims 1
- 208000036725 mucopolysaccharidosis type 3D Diseases 0.000 claims 1
- 208000020004 mucopolysaccharidosis type 9 Diseases 0.000 claims 1
- 208000012224 mucopolysaccharidosis type IIIC Diseases 0.000 claims 1
- 208000027333 mucopolysaccharidosis type IIID Diseases 0.000 claims 1
- 208000012091 mucopolysaccharidosis type IVB Diseases 0.000 claims 1
- 201000008051 neuronal ceroid lipofuscinosis Diseases 0.000 claims 1
- 201000007642 neuronal ceroid lipofuscinosis 1 Diseases 0.000 claims 1
- 210000000056 organ Anatomy 0.000 claims 1
- 238000002203 pretreatment Methods 0.000 claims 1
- 201000010108 pycnodysostosis Diseases 0.000 claims 1
- 208000011985 sialidosis Diseases 0.000 claims 1
- 210000000952 spleen Anatomy 0.000 claims 1
- 238000012385 systemic delivery Methods 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
Classifications
-
- 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)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- 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
- A61K47/02—Inorganic compounds
-
- 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
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
-
- 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
- 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
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
-
- 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
- A61P1/08—Drugs for disorders of the alimentary tract or the digestive system for nausea, cinetosis or vertigo; Antiemetics
-
- 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
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- 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
- 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
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/08—Antiepileptics; Anticonvulsants
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- 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
-
- 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
- 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)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
-
- 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)
-
- 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)
-
- 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)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Mycology (AREA)
- Molecular Biology (AREA)
- Hospice & Palliative Care (AREA)
- Biotechnology (AREA)
- Cardiology (AREA)
- Psychiatry (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Ophthalmology & Optometry (AREA)
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US35885710P | 2010-06-25 | 2010-06-25 | |
| US36078610P | 2010-07-01 | 2010-07-01 | |
| US38786210P | 2010-09-29 | 2010-09-29 | |
| US201161435710P | 2011-01-24 | 2011-01-24 | |
| US201161442115P | 2011-02-11 | 2011-02-11 | |
| US201161476210P | 2011-04-15 | 2011-04-15 | |
| US201161495268P | 2011-06-09 | 2011-06-09 | |
| PCT/US2011/041924 WO2011163648A1 (en) | 2010-06-25 | 2011-06-25 | Cns delivery of therapeutic agents |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| IL291554A IL291554A (en) | 2022-05-01 |
| IL291554B1 IL291554B1 (en) | 2023-10-01 |
| IL291554B2 true IL291554B2 (en) | 2024-02-01 |
Family
ID=46888845
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL291554A IL291554B2 (en) | 2010-06-25 | 2011-06-25 | Administering therapeutic agents to the central nervous system |
| IL223717A IL223717B (en) | 2010-06-25 | 2012-12-18 | Administration of therapeutic agents to the central nervous system |
| IL273889A IL273889B (en) | 2010-06-25 | 2020-04-07 | Administration of therapeutic agents to the central nervous system |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL223717A IL223717B (en) | 2010-06-25 | 2012-12-18 | Administration of therapeutic agents to the central nervous system |
| IL273889A IL273889B (en) | 2010-06-25 | 2020-04-07 | Administration of therapeutic agents to the central nervous system |
Country Status (22)
| Country | Link |
|---|---|
| US (1) | US9283181B2 (https=) |
| EP (3) | EP3103469B1 (https=) |
| JP (1) | JP6045491B2 (https=) |
| KR (6) | KR102283671B1 (https=) |
| CN (1) | CN103096918B (https=) |
| AU (4) | AU2011270668B8 (https=) |
| CA (1) | CA2803166C (https=) |
| CY (2) | CY1118265T1 (https=) |
| DK (1) | DK2588130T3 (https=) |
| ES (1) | ES2650689T3 (https=) |
| HR (1) | HRP20161505T1 (https=) |
| IL (3) | IL291554B2 (https=) |
| LT (1) | LT2588130T (https=) |
| MX (1) | MX389903B (https=) |
| NZ (1) | NZ605865A (https=) |
| PE (1) | PE20230169A1 (https=) |
| PL (1) | PL3103469T3 (https=) |
| RU (1) | RU2626514C2 (https=) |
| SM (1) | SMT201600385T1 (https=) |
| UA (1) | UA115648C2 (https=) |
| WO (1) | WO2011163648A1 (https=) |
| ZA (1) | ZA201300671B (https=) |
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003057179A2 (en) | 2002-01-11 | 2003-07-17 | Biomarin Pharmaceutical, Inc. | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
| HRP20191923T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| KR20230159646A (ko) | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | 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 | ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物 |
| PT3103469T (pt) | 2010-06-25 | 2021-03-04 | Shire Human Genetic Therapies | Administração de agentes terapêuticos no snc |
| LT2588130T (lt) | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| WO2012101671A1 (en) * | 2011-01-25 | 2012-08-02 | Jcr Pharmaceuticals Co., Ltd. | Method for production of human recombinant iduronate 2-sulfatase |
| CN103747807B (zh) | 2011-07-05 | 2016-12-07 | 比奥阿赛斯技术有限公司 | P97‑抗体缀合物和使用方法 |
| SG10201510106PA (en) * | 2011-10-12 | 2016-01-28 | Synageva Biopharma Corp | Recombinant human naglu protein and uses thereof |
| US9682129B2 (en) | 2011-12-23 | 2017-06-20 | Shire Human Genetic Therapies, Inc. | Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| CA3140358A1 (en) * | 2012-07-31 | 2014-02-06 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
| CN112126634A (zh) | 2013-01-09 | 2020-12-25 | 夏尔人类遗传性治疗公司 | 含用于纯化芳基硫酸酯酶a的方法 |
| AU2014218854B2 (en) | 2013-02-20 | 2019-01-24 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
| AU2014243816B2 (en) | 2013-03-13 | 2019-01-31 | Bioasis Technologies Inc. | Fragments of p97 and uses thereof |
| CN115120746A (zh) * | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| JP6603227B2 (ja) | 2014-02-03 | 2019-11-06 | バイオアシス テクノロジーズ インコーポレイテッド | Ｐ９７融合タンパク質 |
| DK3107562T3 (da) | 2014-02-19 | 2019-12-16 | Bioasis Technologies Inc | P97-ids-fusionsproteiner |
| AU2015252906C1 (en) | 2014-05-01 | 2020-09-17 | Bioasis Technologies Inc. | p97-polynucleotide conjugates |
| EP3185889B1 (en) * | 2014-08-11 | 2020-07-15 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| EP3200816A1 (en) | 2014-09-29 | 2017-08-09 | Alexion Pharmaceuticals, Inc. | Methods of treating mucopolysaccharidosis iiib (mpsiiib) |
| WO2016100591A1 (en) * | 2014-12-19 | 2016-06-23 | Biogen Ma Inc. | Apparatus for compound dispersion |
| CA2985235A1 (en) | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Glucocerebrosidase gene therapy for parkinson's disease |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| KR20170004814A (ko) * | 2015-07-02 | 2017-01-11 | 주식회사 녹십자 | 헌터증후군 치료제 |
| WO2017062666A2 (en) * | 2015-10-06 | 2017-04-13 | Patricia Dickson | Enzyme replacement therapy for mucopolysaccharidosis iiid |
| US10123969B2 (en) | 2015-10-15 | 2018-11-13 | Wisconsin Alumni Research Foundation | Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid |
| MX2018007330A (es) * | 2015-12-15 | 2019-03-14 | Genzyme Corp | Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. |
| PL3397270T3 (pl) * | 2015-12-30 | 2024-08-19 | Green Cross Corporation | Kompozycje do zastosowania w leczeniu zespołu huntera |
| JP2019509270A (ja) * | 2016-02-17 | 2019-04-04 | 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 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
| SG10201912761UA (en) | 2016-04-15 | 2020-02-27 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| US11957713B2 (en) | 2016-10-14 | 2024-04-16 | Children's Medical Center Corporation | Compositions and methods for treating diseases and disorders of the central nervous system |
| CA3050690A1 (en) | 2017-01-17 | 2018-07-26 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for diagnosing and treating peroxisomal diseases |
| GB201709459D0 (en) | 2017-06-14 | 2017-07-26 | Intrabio Ltd | Treatment for migraine |
| CN111278453A (zh) * | 2017-09-07 | 2020-06-12 | Jcr制药股份有限公司 | 水性药物组合物 |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| CR20200129A (es) | 2017-10-02 | 2020-08-22 | Denali Therapeutics Inc | Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas |
| US12458614B2 (en) | 2017-10-18 | 2025-11-04 | Intrabio Ltd | Therapeutic agents for improved mobility and cognitive function and for treating neurodegenerative diseases and lysosomal storage disorders |
| IL273929B2 (en) | 2017-10-18 | 2024-11-01 | Intrabio Ltd | Therapeutic agents for neurodegenerative diseases |
| CN111699249A (zh) | 2017-12-19 | 2020-09-22 | 夏尔人类遗传性治疗公司 | 纯化的芳基硫酸酯酶a及其组合物 |
| MA51836B1 (fr) | 2018-02-15 | 2025-01-31 | Intrabio Ltd | Agents thérapeutiques permettant de traiter le syndrome des jambes sans repos |
| US11738068B2 (en) | 2018-06-25 | 2023-08-29 | Jcr Pharmaceuticals Co., Ltd. | Protein-containing aqueous liquid formulation |
| SMT202400334T1 (it) | 2019-03-02 | 2024-09-16 | Intrabio Ltd | (n-)acetil-l-leucina per il trattamento di lesioni cerebrali traumatiche |
| RS67283B1 (sr) | 2019-06-28 | 2025-11-28 | Intrabio Ltd | Kombinovana terapija sa acetil-leucinom i miglustatom za lečenje bolesti lizozomskog skladištenja |
| PT4021575T (pt) * | 2019-08-29 | 2024-07-30 | Biomarin Pharm Inc | Métodos para o tratamento da doença cln2 em sujeitos pediátricos |
| WO2021144720A1 (en) | 2020-01-13 | 2021-07-22 | Intrabio Ltd | Treatment of late-onset neurodegenerative diseases in heterozygous npc1 gene mutation carriers |
| US20230201150A1 (en) | 2020-05-22 | 2023-06-29 | Intrabio Ltd. | The combination of acetyl leucine and 4-aminopyridine or acetazolamide for treating ataxia |
| WO2024102808A2 (en) * | 2022-11-09 | 2024-05-16 | Gmp Biotechnology Limited | Compositions and methods for cns diseases |
| WO2024176112A1 (en) | 2023-02-21 | 2024-08-29 | Takeda Pharmaceutical Company Limited | Large scale production of recombinant arylsulfatase a and compositions thereof |
| WO2025264957A2 (en) | 2024-06-20 | 2025-12-26 | Intrabio Inc. | Acetyl leucine for treating neurodevelopmental disorders |
Family Cites Families (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4743265A (en) | 1986-04-23 | 1988-05-10 | Dij Catheter Corp | Articulated catheter placement device |
| US5863782A (en) | 1995-04-19 | 1999-01-26 | Women's And Children's Hospital | Synthetic mammalian sulphamidase and genetic sequences encoding same |
| US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
| US6063378A (en) | 1997-08-22 | 2000-05-16 | Seikagaku Corporation | Therapeutic agent for herniated intervertebral disc |
| ES2312222T3 (es) | 1998-12-07 | 2009-02-16 | Genzyme Corporation | Tratamiento de la enfermedad de pompe. |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| US20020052311A1 (en) | 1999-09-03 | 2002-05-02 | Beka Solomon | Methods and compostions for the treatment and/or diagnosis of neurological diseases and disorders |
| US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| US20020099025A1 (en) * | 1999-12-30 | 2002-07-25 | Heywood James A. | Treatment of neurological disorders |
| WO2002087510A2 (en) | 2001-04-30 | 2002-11-07 | Symbiontics, Inc. | Subcellular targeting of therapeutic proteins |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, 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 |
| AU2002347910A1 (en) | 2001-10-16 | 2003-04-28 | Symbiontics Inc. | Methods and compositions for targeting proteins across the blood brain barrier |
| DK1503788T3 (da) * | 2002-04-25 | 2011-10-17 | Shire Human Genetic Therapies | Behandling af alpha-galactosidase A-deficiens |
| EP1514106A4 (en) | 2002-05-29 | 2007-05-09 | Zystor Therapeutics Inc | TARGETED THERAPEUTIC PROTEINS |
| ES2371913T3 (es) | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
| EP1638605B1 (en) | 2003-06-20 | 2014-01-08 | Raptor Pharmaceutical, Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050026823A1 (en) * | 2003-06-20 | 2005-02-03 | Biomarin Pharmaceutical Inc. | Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues |
| US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| EP1713904B1 (en) | 2004-01-30 | 2016-06-29 | Shire Pharmaceuticals Ireland Limited | Production and purification of recombinant arylsulfatase a |
| WO2005074888A2 (en) | 2004-02-03 | 2005-08-18 | Biodelivery Sciences International, Inc. | Replacement enzyme cochleates |
| US20080014188A1 (en) | 2004-02-06 | 2008-01-17 | Zankel Todd C | Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof |
| CA2553955C (en) | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| US20090297592A1 (en) | 2005-05-11 | 2009-12-03 | Jcr Pharmaceuticals Co., Ltd. | Lipid Vesicle Composition |
| AU2006254796B2 (en) | 2005-06-08 | 2012-08-16 | Amicus Therapeutics, Inc. | Treatment of CNS disorders associated with mutations in genes encoding lysosomal enzymes |
| AR059089A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| HUE042402T2 (hu) * | 2006-04-04 | 2019-06-28 | Chiesi Farm Spa | Eljárás polipeptid töményítésére |
| GB0611463D0 (en) | 2006-06-09 | 2006-07-19 | Novartis Ag | Organic compounds |
| WO2008070769A1 (en) | 2006-12-06 | 2008-06-12 | Medtronic, Inc. | Intrathecal catheter |
| CA2680189A1 (en) | 2007-03-06 | 2008-09-12 | Saint Louis University | Modified enzyme and treatment method |
| US20110059508A1 (en) * | 2007-06-29 | 2011-03-10 | National University Corporation Nagoya University | Improving agent for dysfunction due to neuropathy and rho kinase activation inhibitor |
| WO2009017005A1 (ja) | 2007-07-27 | 2009-02-05 | Sharp Kabushiki Kaisha | 移動局装置、基地局装置、通信システム及びプログラム |
| JP2010536341A (ja) | 2007-08-15 | 2010-12-02 | アムニクス， インコーポレイテッド | 生物学的に活性なポリペプチドの特性を改変するための組成物および方法 |
| US8420081B2 (en) | 2007-11-30 | 2013-04-16 | Abbvie, Inc. | Antibody formulations and methods of making same |
| US7722865B2 (en) | 2008-01-18 | 2010-05-25 | Biomarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
| JP5627571B2 (ja) | 2008-05-07 | 2014-11-19 | バイオマリン ファーマシューティカル インコーポレイテッド | リソソーム標的化ペプチドおよびその使用 |
| US8436489B2 (en) | 2009-06-29 | 2013-05-07 | Lightsail Energy, Inc. | Compressed air energy storage system utilizing two-phase flow to facilitate heat exchange |
| US20110318324A1 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of b-galactocerebrosidase |
| JP6073783B2 (ja) | 2010-06-25 | 2017-02-01 | 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 | ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物 |
| HRP20191923T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku arilsulfataze a u cns |
| LT2588130T (lt) | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| AU2011270672B2 (en) | 2010-06-25 | 2017-01-12 | Shire Human Genetic Therapies, Inc. | Treatment of Sanfilippo Syndrome type B |
| KR20230159646A (ko) * | 2010-06-25 | 2023-11-21 | 샤이어 휴먼 지네틱 테라피즈 인크. | 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
| PT3103469T (pt) | 2010-06-25 | 2021-03-04 | Shire Human Genetic Therapies | Administração de agentes terapêuticos no snc |
| JP2012062312A (ja) | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
-
2011
- 2011-06-25 LT LTEP11799035.8T patent/LT2588130T/lt unknown
- 2011-06-25 MX MX2018003131A patent/MX389903B/es unknown
- 2011-06-25 KR KR1020217003036A patent/KR102283671B1/ko active Active
- 2011-06-25 ES ES11799035T patent/ES2650689T3/es active Active
- 2011-06-25 WO PCT/US2011/041924 patent/WO2011163648A1/en not_active Ceased
- 2011-06-25 NZ NZ605865A patent/NZ605865A/en unknown
- 2011-06-25 JP JP2013516844A patent/JP6045491B2/ja active Active
- 2011-06-25 AU AU2011270668A patent/AU2011270668B8/en active Active
- 2011-06-25 IL IL291554A patent/IL291554B2/en unknown
- 2011-06-25 HR HRP20161505TT patent/HRP20161505T1/hr unknown
- 2011-06-25 KR KR1020247014519A patent/KR20240068752A/ko not_active Withdrawn
- 2011-06-25 EP EP16179150.4A patent/EP3103469B1/en not_active Revoked
- 2011-06-25 EP EP11799035.8A patent/EP2588130B1/en active Active
- 2011-06-25 PE PE2022001782A patent/PE20230169A1/es unknown
- 2011-06-25 CN CN201180040978.5A patent/CN103096918B/zh active Active
- 2011-06-25 KR KR1020197022037A patent/KR102211960B1/ko active Active
- 2011-06-25 SM SM20160385T patent/SMT201600385T1/it unknown
- 2011-06-25 DK DK11799035.8T patent/DK2588130T3/en active
- 2011-06-25 KR KR1020217042861A patent/KR102663407B1/ko active Active
- 2011-06-25 PL PL16179150T patent/PL3103469T3/pl unknown
- 2011-06-25 CA CA2803166A patent/CA2803166C/en active Active
- 2011-06-25 EP EP20212573.8A patent/EP3875107A1/en active Pending
- 2011-06-25 US US13/168,961 patent/US9283181B2/en active Active
- 2011-06-25 RU RU2012154831A patent/RU2626514C2/ru active
- 2011-06-25 UA UAA201214666A patent/UA115648C2/uk unknown
- 2011-06-25 KR KR1020137001748A patent/KR102007044B1/ko active Active
- 2011-06-25 KR KR1020217023503A patent/KR102345973B1/ko active Active
-
2012
- 2012-12-18 IL IL223717A patent/IL223717B/en active IP Right Grant
-
2013
- 2013-01-25 ZA ZA2013/00671A patent/ZA201300671B/en unknown
-
2016
- 2016-11-17 CY CY20161101188T patent/CY1118265T1/el unknown
-
2017
- 2017-04-10 AU AU2017202349A patent/AU2017202349B2/en active Active
-
2019
- 2019-06-11 AU AU2019204056A patent/AU2019204056B2/en active Active
-
2020
- 2020-04-07 IL IL273889A patent/IL273889B/en unknown
-
2021
- 2021-03-08 CY CY20211100195T patent/CY1124154T1/el unknown
- 2021-10-20 AU AU2021254564A patent/AU2021254564B2/en active Active
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL291554B2 (en) | Administering therapeutic agents to the central nervous system | |
| JP2016037504A5 (https=) | ||
| Parenti et al. | Lysosomal storage diseases: from pathophysiology to therapy | |
| EP1981546B1 (en) | Enzyme replacement therapy for treating lysosomal storage diseases | |
| US10907142B2 (en) | Oligosaccharides comprising an aminooxy group and conjugates thereof | |
| US11065308B2 (en) | Methods and compositions for CNS delivery of heparan n-sulfatase | |
| US8580922B2 (en) | Peptide linkers for polypeptide compositions and methods for using same | |
| Malatack et al. | The status of hematopoietic stem cell transplantation in lysosomal storage disease | |
| JP2013534526A5 (https=) | ||
| JP2007504166A5 (https=) | ||
| US20090022702A1 (en) | Methods for introducing mannose 6-phosphate and other oligosacharides onto glycoproteins | |
| JP2015524662A5 (https=) | ||
| Datta et al. | Enzyme therapy: a forerunner in catalyzing a healthy society? | |
| ES2535461T3 (es) | Método de tratamiento de la enfermedad por almacenamiento de glucógeno | |
| Zimran et al. | A pharmacokinetic analysis of a novel enzyme replacement therapy with Gene-Activated® human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease | |
| JP2010509344A5 (https=) | ||
| Neufeld | Enzyme replacement therapy–a brief history | |
| CN101214249A (zh) | 尼曼-皮克病的基于陪伴分子的治疗 | |
| Caillaud et al. | Gene therapy in lysosomal diseases | |
| Eto et al. | Novel treatment for neuronopathic lysosomal storage diseases-cell therapy/gene therapy | |
| Matzner | Therapy of lysosomal storage diseases | |
| Sestito et al. | Pathobiological insights into the newly targeted therapies of lysosomal storage disorders | |
| HK40021607B (en) | Oligosaccharides comprising an aminooxy group and conjugates thereof | |
| Patel et al. | A Review on: Enzyme Replacement Therapy | |
| Winkel | Enzyme Therapy in Non-classic Pompe’s Disease: Safety and efficacy of recombinant human a-glucosidase from milk of transgenic rabbits and from Chinese hamster ovary cells |