ES2643015T3 - Tratamiento del Síndrome de Sanfilippo Tipo B - Google Patents
Tratamiento del Síndrome de Sanfilippo Tipo B Download PDFInfo
- Publication number
- ES2643015T3 ES2643015T3 ES11799039.0T ES11799039T ES2643015T3 ES 2643015 T3 ES2643015 T3 ES 2643015T3 ES 11799039 T ES11799039 T ES 11799039T ES 2643015 T3 ES2643015 T3 ES 2643015T3
- Authority
- ES
- Spain
- Prior art keywords
- protein
- brain
- naglu
- tissues
- therapeutic
- Prior art date
- Legal status (The legal status 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 status listed.)
- Active
Links
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 title 1
- 208000036709 mucopolysaccharidosis type 3B Diseases 0.000 title 1
- 208000012227 mucopolysaccharidosis type IIIB Diseases 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 description 46
- 108090000790 Enzymes Proteins 0.000 description 45
- 102000004190 Enzymes Human genes 0.000 description 45
- 102000004169 proteins and genes Human genes 0.000 description 43
- 210000001519 tissue Anatomy 0.000 description 37
- 239000000203 mixture Substances 0.000 description 27
- 210000004556 brain Anatomy 0.000 description 25
- 239000003814 drug Substances 0.000 description 25
- 230000001225 therapeutic effect Effects 0.000 description 25
- 229940124597 therapeutic agent Drugs 0.000 description 24
- 238000009472 formulation Methods 0.000 description 18
- 210000004027 cell Anatomy 0.000 description 17
- 238000007913 intrathecal administration Methods 0.000 description 16
- 125000003275 alpha amino acid group Chemical group 0.000 description 11
- 150000001413 amino acids Chemical class 0.000 description 11
- 238000000034 method Methods 0.000 description 11
- 230000002490 cerebral effect Effects 0.000 description 10
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 10
- 230000002132 lysosomal effect Effects 0.000 description 9
- 108020001507 fusion proteins Proteins 0.000 description 8
- 102000037865 fusion proteins Human genes 0.000 description 8
- 239000008194 pharmaceutical composition Substances 0.000 description 8
- 150000007523 nucleic acids Chemical group 0.000 description 7
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 6
- 230000004071 biological effect Effects 0.000 description 6
- 210000000133 brain stem Anatomy 0.000 description 6
- 201000010099 disease Diseases 0.000 description 6
- 239000012530 fluid Substances 0.000 description 6
- 108090000765 processed proteins & peptides Proteins 0.000 description 6
- 210000001638 cerebellum Anatomy 0.000 description 5
- 210000003734 kidney Anatomy 0.000 description 5
- 239000002243 precursor Substances 0.000 description 5
- 241000282693 Cercopithecidae Species 0.000 description 4
- 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 description 4
- 108010076504 Protein Sorting Signals Proteins 0.000 description 4
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 4
- 210000005013 brain tissue Anatomy 0.000 description 4
- 208000035475 disorder Diseases 0.000 description 4
- 238000001802 infusion Methods 0.000 description 4
- 238000003780 insertion Methods 0.000 description 4
- 230000037431 insertion Effects 0.000 description 4
- 210000003292 kidney cell Anatomy 0.000 description 4
- 210000004962 mammalian cell Anatomy 0.000 description 4
- 229920001542 oligosaccharide Polymers 0.000 description 4
- 150000002482 oligosaccharides Chemical class 0.000 description 4
- 238000006467 substitution reaction Methods 0.000 description 4
- 239000002699 waste material Substances 0.000 description 4
- 102100037182 Cation-independent mannose-6-phosphate receptor Human genes 0.000 description 3
- 101710145225 Cation-independent mannose-6-phosphate receptor Proteins 0.000 description 3
- 241000699666 Mus <mouse, genus> Species 0.000 description 3
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 description 3
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 description 3
- 150000001720 carbohydrates Chemical group 0.000 description 3
- 230000001054 cortical effect Effects 0.000 description 3
- 230000000694 effects Effects 0.000 description 3
- 230000013595 glycosylation Effects 0.000 description 3
- 238000006206 glycosylation reaction Methods 0.000 description 3
- 210000003016 hypothalamus Anatomy 0.000 description 3
- 210000001259 mesencephalon Anatomy 0.000 description 3
- 108020004707 nucleic acids Proteins 0.000 description 3
- 102000039446 nucleic acids Human genes 0.000 description 3
- 230000008569 process Effects 0.000 description 3
- 239000011780 sodium chloride Substances 0.000 description 3
- 239000000243 solution Substances 0.000 description 3
- 210000000278 spinal cord Anatomy 0.000 description 3
- 238000003860 storage Methods 0.000 description 3
- 239000000126 substance Substances 0.000 description 3
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 3
- 210000004885 white matter Anatomy 0.000 description 3
- 206010067484 Adverse reaction Diseases 0.000 description 2
- 241000894006 Bacteria Species 0.000 description 2
- 241000699800 Cricetinae Species 0.000 description 2
- 241000699802 Cricetulus griseus Species 0.000 description 2
- 102000048143 Insulin-Like Growth Factor II Human genes 0.000 description 2
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 description 2
- 108091028043 Nucleic acid sequence Proteins 0.000 description 2
- 108010033276 Peptide Fragments Proteins 0.000 description 2
- 102000007079 Peptide Fragments Human genes 0.000 description 2
- 240000004808 Saccharomyces cerevisiae Species 0.000 description 2
- 244000000188 Vaccinium ovalifolium Species 0.000 description 2
- 230000009471 action Effects 0.000 description 2
- 230000006838 adverse reaction Effects 0.000 description 2
- 238000004458 analytical method Methods 0.000 description 2
- 238000002869 basic local alignment search tool Methods 0.000 description 2
- 238000003766 bioinformatics method Methods 0.000 description 2
- 230000015572 biosynthetic process Effects 0.000 description 2
- 238000009395 breeding Methods 0.000 description 2
- 230000001488 breeding effect Effects 0.000 description 2
- 159000000007 calcium salts Chemical class 0.000 description 2
- 230000015556 catabolic process Effects 0.000 description 2
- 210000003169 central nervous system Anatomy 0.000 description 2
- 210000004978 chinese hamster ovary cell Anatomy 0.000 description 2
- 238000004590 computer program Methods 0.000 description 2
- 238000006731 degradation reaction Methods 0.000 description 2
- 238000012217 deletion Methods 0.000 description 2
- 230000037430 deletion Effects 0.000 description 2
- 210000002451 diencephalon Anatomy 0.000 description 2
- 239000003085 diluting agent Substances 0.000 description 2
- 238000006073 displacement reaction Methods 0.000 description 2
- 210000002472 endoplasmic reticulum Anatomy 0.000 description 2
- 230000002255 enzymatic effect Effects 0.000 description 2
- 239000012634 fragment Substances 0.000 description 2
- 238000004108 freeze drying Methods 0.000 description 2
- 238000007710 freezing Methods 0.000 description 2
- 230000008014 freezing Effects 0.000 description 2
- 210000001320 hippocampus Anatomy 0.000 description 2
- 239000007924 injection Substances 0.000 description 2
- 238000002347 injection Methods 0.000 description 2
- 210000005229 liver cell Anatomy 0.000 description 2
- 230000007246 mechanism Effects 0.000 description 2
- 210000001672 ovary Anatomy 0.000 description 2
- 229920001184 polypeptide Polymers 0.000 description 2
- 102000004196 processed proteins & peptides Human genes 0.000 description 2
- 230000004952 protein activity Effects 0.000 description 2
- 210000000449 purkinje cell Anatomy 0.000 description 2
- 230000000979 retarding effect Effects 0.000 description 2
- 230000000087 stabilizing effect Effects 0.000 description 2
- 208000024891 symptom Diseases 0.000 description 2
- 208000011580 syndromic disease Diseases 0.000 description 2
- 210000001103 thalamus Anatomy 0.000 description 2
- 238000010257 thawing Methods 0.000 description 2
- 230000009261 transgenic effect Effects 0.000 description 2
- 230000002861 ventricular Effects 0.000 description 2
- NHBKXEKEPDILRR-UHFFFAOYSA-N 2,3-bis(butanoylsulfanyl)propyl butanoate Chemical compound CCCC(=O)OCC(SC(=O)CCC)CSC(=O)CCC NHBKXEKEPDILRR-UHFFFAOYSA-N 0.000 description 1
- 241000193830 Bacillus <bacterium> Species 0.000 description 1
- 235000014469 Bacillus subtilis Nutrition 0.000 description 1
- 206010006187 Breast cancer Diseases 0.000 description 1
- 241000282465 Canis Species 0.000 description 1
- 241000283707 Capra Species 0.000 description 1
- 241000282552 Chlorocebus aethiops Species 0.000 description 1
- SHZGCJCMOBCMKK-UHFFFAOYSA-N D-mannomethylose Chemical group CC1OC(O)C(O)C(O)C1O SHZGCJCMOBCMKK-UHFFFAOYSA-N 0.000 description 1
- 102100024746 Dihydrofolate reductase Human genes 0.000 description 1
- 101150110503 END3 gene Proteins 0.000 description 1
- 241000196324 Embryophyta Species 0.000 description 1
- 241000588724 Escherichia coli Species 0.000 description 1
- PNNNRSAQSRJVSB-SLPGGIOYSA-N Fucose Chemical group C[C@H](O)[C@@H](O)[C@H](O)[C@H](O)C=O PNNNRSAQSRJVSB-SLPGGIOYSA-N 0.000 description 1
- 241000233866 Fungi Species 0.000 description 1
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 1
- 241000238631 Hexapoda Species 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 102000003839 Human Proteins Human genes 0.000 description 1
- 108090000144 Human Proteins Proteins 0.000 description 1
- 241000235058 Komagataella pastoris Species 0.000 description 1
- SHZGCJCMOBCMKK-DHVFOXMCSA-N L-fucopyranose Chemical group C[C@@H]1OC(O)[C@@H](O)[C@H](O)[C@@H]1O SHZGCJCMOBCMKK-DHVFOXMCSA-N 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 108010031099 Mannose Receptor Proteins 0.000 description 1
- 208000007650 Meningeal Carcinomatosis Diseases 0.000 description 1
- 206010051696 Metastases to meninges Diseases 0.000 description 1
- 101000822667 Mus musculus Something about silencing protein 10 Proteins 0.000 description 1
- 241000699670 Mus sp. Species 0.000 description 1
- OVRNDRQMDRJTHS-UHFFFAOYSA-N N-acelyl-D-glucosamine Natural products CC(=O)NC1C(O)OC(CO)C(O)C1O OVRNDRQMDRJTHS-UHFFFAOYSA-N 0.000 description 1
- OVRNDRQMDRJTHS-FMDGEEDCSA-N N-acetyl-beta-D-glucosamine Chemical compound CC(=O)N[C@H]1[C@H](O)O[C@H](CO)[C@@H](O)[C@@H]1O OVRNDRQMDRJTHS-FMDGEEDCSA-N 0.000 description 1
- MBLBDJOUHNCFQT-LXGUWJNJSA-N N-acetylglucosamine Natural products CC(=O)N[C@@H](C=O)[C@@H](O)[C@H](O)[C@H](O)CO MBLBDJOUHNCFQT-LXGUWJNJSA-N 0.000 description 1
- 244000061176 Nicotiana tabacum Species 0.000 description 1
- 235000002637 Nicotiana tabacum Nutrition 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
- 229910019142 PO4 Inorganic materials 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 description 1
- 206010035226 Plasma cell myeloma Diseases 0.000 description 1
- 206010062519 Poor quality sleep Diseases 0.000 description 1
- 241000589516 Pseudomonas Species 0.000 description 1
- 241000700157 Rattus norvegicus Species 0.000 description 1
- 241000187747 Streptomyces Species 0.000 description 1
- 229930006000 Sucrose Natural products 0.000 description 1
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 description 1
- 241000282887 Suidae Species 0.000 description 1
- LFTYTUAZOPRMMI-CFRASDGPSA-N UDP-N-acetyl-alpha-D-glucosamine Chemical compound O1[C@H](CO)[C@@H](O)[C@H](O)[C@@H](NC(=O)C)[C@H]1OP(O)(=O)OP(O)(=O)OC[C@@H]1[C@@H](O)[C@@H](O)[C@H](N2C(NC(=O)C=C2)=O)O1 LFTYTUAZOPRMMI-CFRASDGPSA-N 0.000 description 1
- LFTYTUAZOPRMMI-UHFFFAOYSA-N UNPD164450 Natural products O1C(CO)C(O)C(O)C(NC(=O)C)C1OP(O)(=O)OP(O)(=O)OCC1C(O)C(O)C(N2C(NC(=O)C=C2)=O)O1 LFTYTUAZOPRMMI-UHFFFAOYSA-N 0.000 description 1
- 230000036626 alertness Effects 0.000 description 1
- 239000008228 bacteriostatic water for injection Substances 0.000 description 1
- 210000004227 basal ganglia Anatomy 0.000 description 1
- 230000033228 biological regulation Effects 0.000 description 1
- 210000004204 blood vessel Anatomy 0.000 description 1
- 210000004958 brain cell Anatomy 0.000 description 1
- 210000004899 c-terminal region Anatomy 0.000 description 1
- 239000011575 calcium Substances 0.000 description 1
- 210000001159 caudate nucleus Anatomy 0.000 description 1
- 238000004113 cell culture Methods 0.000 description 1
- 235000013339 cereals Nutrition 0.000 description 1
- 210000003591 cerebellar nuclei Anatomy 0.000 description 1
- 210000004720 cerebrum Anatomy 0.000 description 1
- 208000019065 cervical carcinoma Diseases 0.000 description 1
- 238000004891 communication Methods 0.000 description 1
- 210000003792 cranial nerve Anatomy 0.000 description 1
- 230000002950 deficient Effects 0.000 description 1
- 230000001934 delay Effects 0.000 description 1
- 108020001096 dihydrofolate reductase Proteins 0.000 description 1
- 229940079593 drug Drugs 0.000 description 1
- 210000000647 epithalamus Anatomy 0.000 description 1
- 230000001747 exhibiting effect Effects 0.000 description 1
- 239000013022 formulation composition Substances 0.000 description 1
- 210000000609 ganglia Anatomy 0.000 description 1
- 239000008103 glucose Substances 0.000 description 1
- 210000004884 grey matter Anatomy 0.000 description 1
- 206010073071 hepatocellular carcinoma Diseases 0.000 description 1
- 239000012456 homogeneous solution Substances 0.000 description 1
- 230000002209 hydrophobic effect Effects 0.000 description 1
- 239000000819 hypertonic solution Substances 0.000 description 1
- 229940021223 hypertonic solution Drugs 0.000 description 1
- 238000007917 intracranial administration Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 239000000644 isotonic solution Substances 0.000 description 1
- 235000021374 legumes Nutrition 0.000 description 1
- 239000007788 liquid Substances 0.000 description 1
- 239000006193 liquid solution Substances 0.000 description 1
- 238000009593 lumbar puncture Methods 0.000 description 1
- 210000005265 lung cell Anatomy 0.000 description 1
- 210000003712 lysosome Anatomy 0.000 description 1
- 230000001868 lysosomic effect Effects 0.000 description 1
- 238000007726 management method Methods 0.000 description 1
- 125000000311 mannosyl group Chemical group C1([C@@H](O)[C@@H](O)[C@H](O)[C@H](O1)CO)* 0.000 description 1
- 210000002418 meninge Anatomy 0.000 description 1
- 210000002891 metencephalon Anatomy 0.000 description 1
- 239000008267 milk Substances 0.000 description 1
- 210000004080 milk Anatomy 0.000 description 1
- 235000013336 milk Nutrition 0.000 description 1
- 108091005573 modified proteins Proteins 0.000 description 1
- 102000035118 modified proteins Human genes 0.000 description 1
- 239000002052 molecular layer Substances 0.000 description 1
- 208000005340 mucopolysaccharidosis III Diseases 0.000 description 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- 201000000050 myeloid neoplasm Diseases 0.000 description 1
- 229950006780 n-acetylglucosamine Drugs 0.000 description 1
- 210000000478 neocortex Anatomy 0.000 description 1
- 210000002475 olfactory pathway Anatomy 0.000 description 1
- 210000001328 optic nerve Anatomy 0.000 description 1
- 230000020477 pH reduction Effects 0.000 description 1
- 210000002741 palatine tonsil Anatomy 0.000 description 1
- 229940021222 peritoneal dialysis isotonic solution Drugs 0.000 description 1
- 239000010452 phosphate Substances 0.000 description 1
- 239000011574 phosphorus Substances 0.000 description 1
- 229910052698 phosphorus Inorganic materials 0.000 description 1
- 210000003446 pia mater Anatomy 0.000 description 1
- 230000002035 prolonged effect Effects 0.000 description 1
- 210000002637 putamen Anatomy 0.000 description 1
- 210000000463 red nucleus Anatomy 0.000 description 1
- 150000003839 salts Chemical class 0.000 description 1
- 210000004761 scalp Anatomy 0.000 description 1
- 230000003248 secreting effect Effects 0.000 description 1
- 239000011734 sodium Substances 0.000 description 1
- 229910000162 sodium phosphate Inorganic materials 0.000 description 1
- 239000001488 sodium phosphate Substances 0.000 description 1
- 239000007787 solid Substances 0.000 description 1
- 239000002904 solvent Substances 0.000 description 1
- 239000008223 sterile water Substances 0.000 description 1
- 210000000495 subthalamus Anatomy 0.000 description 1
- 239000005720 sucrose Substances 0.000 description 1
- 238000004114 suspension culture Methods 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 210000004062 tegmentum mesencephali Anatomy 0.000 description 1
- 238000012546 transfer Methods 0.000 description 1
- 230000014616 translation Effects 0.000 description 1
- RYFMWSXOAZQYPI-UHFFFAOYSA-K trisodium phosphate Chemical compound [Na+].[Na+].[Na+].[O-]P([O-])([O-])=O RYFMWSXOAZQYPI-UHFFFAOYSA-K 0.000 description 1
- 210000004785 virchow-robin space Anatomy 0.000 description 1
Classifications
-
- 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
- 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
- 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
- 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
- A61K9/14—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
- 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
- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
-
- 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
- A61P25/20—Hypnotics; Sedatives
-
- 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
- A61P27/16—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
-
- 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)
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Inorganic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Psychiatry (AREA)
- General Engineering & Computer Science (AREA)
- Psychology (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Anesthesiology (AREA)
- Hospice & Palliative Care (AREA)
- Hematology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (15)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US35885710P | 2010-06-25 | 2010-06-25 | |
| US358857P | 2010-06-25 | ||
| US36078610P | 2010-07-01 | 2010-07-01 | |
| US360786P | 2010-07-01 | ||
| US38786210P | 2010-09-29 | 2010-09-29 | |
| US387862P | 2010-09-29 | ||
| US201161435710P | 2011-01-24 | 2011-01-24 | |
| US201161435710P | 2011-01-24 | ||
| US201161442115P | 2011-02-11 | 2011-02-11 | |
| US201161442115P | 2011-02-11 | ||
| US201161476210P | 2011-04-15 | 2011-04-15 | |
| US201161476210P | 2011-04-15 | ||
| US201161495268P | 2011-06-09 | 2011-06-09 | |
| US201161495268P | 2011-06-09 | ||
| PCT/US2011/041928 WO2011163652A2 (en) | 2010-06-25 | 2011-06-25 | Treatment of sanfilippo syndrome type b |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2643015T3 true ES2643015T3 (es) | 2017-11-21 |
Family
ID=46888842
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES11799039.0T Active ES2643015T3 (es) | 2010-06-25 | 2011-06-25 | Tratamiento del Síndrome de Sanfilippo Tipo B |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20110318327A1 (enExample) |
| EP (1) | EP2585105B1 (enExample) |
| JP (1) | JP6063380B2 (enExample) |
| KR (1) | KR20130043166A (enExample) |
| CN (1) | CN103269709A (enExample) |
| AU (2) | AU2011270672B2 (enExample) |
| CA (1) | CA2805449A1 (enExample) |
| ES (1) | ES2643015T3 (enExample) |
| MX (1) | MX2013000324A (enExample) |
| NZ (2) | NZ605871A (enExample) |
| WO (1) | WO2011163652A2 (enExample) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7741446B2 (en) | 2006-08-18 | 2010-06-22 | Armagen Technologies, Inc. | Fusion antibodies that cross the blood-brain barrier in both directions |
| EP2997976A1 (en) | 2007-07-27 | 2016-03-23 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-l-iduronidase activity in the cns |
| HUE034850T2 (en) * | 2008-05-07 | 2018-03-28 | Biomarin Pharm Inc | Lysosomal targeting peptides and their use |
| WO2011044542A1 (en) | 2009-10-09 | 2011-04-14 | Armagen Technologies, Inc. | Methods and compositions for increasing iduronate 2-sulfatase activity in the cns |
| IL291554B2 (en) | 2010-06-25 | 2024-02-01 | Shire Human Genetic Therapies | Administering therapeutic agents to the central nervous system |
| MX382443B (es) | 2010-06-25 | 2025-03-11 | Shire Human Genetic Therapies | Una formulación estable para usarse en el tratamiento de la enfermedad por leucodistrofia metacromática (mld). |
| PE20130589A1 (es) | 2010-06-25 | 2013-05-19 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de arilsulfatasa a |
| MX2013000320A (es) | 2010-06-25 | 2013-06-05 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa. |
| EP2975134A3 (en) | 2010-07-22 | 2016-02-24 | Shire Human Genetic Therapies, Inc. | Crystal structure of human alpha-n-acetylglucosaminidase |
| CN103764162B (zh) * | 2011-03-04 | 2017-03-08 | 夏尔人类遗传治疗公司 | 用于多肽组成物的肽连接物及其使用方法 |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| JP6329483B2 (ja) | 2011-10-12 | 2018-05-23 | シナジェバ・バイオファーマ・コーポレイションＳｙｎａｇｅｖａ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐ． | 組換えヒトｎａｇｌｕタンパク質およびその利用 |
| JP6266529B2 (ja) | 2011-12-02 | 2018-01-24 | アーマジェン・インコーポレイテッドＡｒｍａｇｅｎ， Ｉｎｃ． | Ｃｎｓにおけるアリールスルファターゼａ活性を増加するための方法および組成物 |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| KR101380740B1 (ko) * | 2012-06-29 | 2014-04-11 | 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 | 이듀로네이트-2-설파타제의 정제 |
| KR102521039B1 (ko) * | 2012-11-27 | 2023-04-12 | 바이오마린 파머수티컬 인크. | 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들 |
| FR3011468B1 (fr) * | 2013-10-04 | 2015-12-04 | Inventiva | Utilisation de l'odiparcil dans le traitement d'une mucopolysaccharidose |
| EP3160494B1 (en) * | 2014-06-25 | 2019-11-20 | Shire Human Genetic Therapies, Inc. | Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase |
| WO2016025523A1 (en) | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
| MX2017001898A (es) | 2014-08-11 | 2017-04-11 | Shire Human Genetic Therapies | Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales. |
| JP2017531632A (ja) * | 2014-09-29 | 2017-10-26 | アレクシオン ファーマシューティカルズ， インコーポレイテッド | ムコ多糖症ｉｉｉｂ型（ｍｐｓｉｉｉｂ）を治療する方法 |
| EP3220958A1 (en) * | 2014-10-24 | 2017-09-27 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting of enzymes, and uses thereof |
| MA41022A (fr) | 2014-11-24 | 2017-10-03 | Shire Human Genetic Therapies | Ciblage lysosomial et utilisations correspondantes |
| US10538589B2 (en) * | 2015-01-14 | 2020-01-21 | Armagen Inc. | Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU |
| AU2017222620B2 (en) * | 2016-02-24 | 2022-06-16 | Biomarin Pharmaceutical Inc. | Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof |
| CN112566652A (zh) | 2018-06-25 | 2021-03-26 | Jcr制药股份有限公司 | 含有蛋白的水性液体制剂 |
| WO2022045273A1 (ja) * | 2020-08-28 | 2022-03-03 | Ｊｃｒファーマ株式会社 | α－Ｎ－アセチルグルコサミニダーゼの変異体 |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
| ES2677343T3 (es) | 1998-12-07 | 2018-08-01 | 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 |
| US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| IL158623A0 (en) | 2001-04-30 | 2004-05-12 | Symbiontics Inc | Subcellular targeting of therapeutic proteins |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | 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 |
| AU2002362930A2 (en) * | 2001-10-16 | 2003-04-28 | Zystor Therapeutics, Inc. | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
| AU2003237314B2 (en) | 2002-05-29 | 2010-03-04 | Biomarin Pharmaceutical Inc. | Targeted therapeutic proteins |
| ES2371913T3 (es) | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
| JP5624256B2 (ja) * | 2003-06-20 | 2014-11-12 | ラプター・ファーマシューティカル・インコーポレイテッド | 脳および他の組織への治療化合物の送達 |
| US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050244400A1 (en) | 2004-02-10 | 2005-11-03 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| GB0611463D0 (en) * | 2006-06-09 | 2006-07-19 | Novartis Ag | Organic compounds |
| CA2680189A1 (en) | 2007-03-06 | 2008-09-12 | Saint Louis University | Modified enzyme and treatment method |
| HUE034850T2 (en) * | 2008-05-07 | 2018-03-28 | Biomarin Pharm Inc | Lysosomal targeting peptides and their use |
| MX2013000320A (es) * | 2010-06-25 | 2013-06-05 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa. |
-
2011
- 2011-06-25 WO PCT/US2011/041928 patent/WO2011163652A2/en not_active Ceased
- 2011-06-25 MX MX2013000324A patent/MX2013000324A/es unknown
- 2011-06-25 NZ NZ605871A patent/NZ605871A/en not_active IP Right Cessation
- 2011-06-25 NZ NZ702801A patent/NZ702801A/en not_active IP Right Cessation
- 2011-06-25 CN CN2011800407953A patent/CN103269709A/zh active Pending
- 2011-06-25 KR KR1020137001751A patent/KR20130043166A/ko not_active Ceased
- 2011-06-25 ES ES11799039.0T patent/ES2643015T3/es active Active
- 2011-06-25 US US13/168,969 patent/US20110318327A1/en not_active Abandoned
- 2011-06-25 EP EP11799039.0A patent/EP2585105B1/en active Active
- 2011-06-25 JP JP2013516847A patent/JP6063380B2/ja not_active Expired - Fee Related
- 2011-06-25 AU AU2011270672A patent/AU2011270672B2/en not_active Ceased
- 2011-06-25 CA CA2805449A patent/CA2805449A1/en not_active Abandoned
-
2017
- 2017-04-10 AU AU2017202344A patent/AU2017202344A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| WO2011163652A3 (en) | 2013-07-11 |
| NZ605871A (en) | 2015-02-27 |
| CA2805449A1 (en) | 2011-12-29 |
| CN103269709A (zh) | 2013-08-28 |
| AU2011270672A1 (en) | 2013-02-14 |
| JP6063380B2 (ja) | 2017-01-18 |
| US20110318327A1 (en) | 2011-12-29 |
| AU2017202344A1 (en) | 2017-04-27 |
| EP2585105B1 (en) | 2017-08-09 |
| WO2011163652A2 (en) | 2011-12-29 |
| MX2013000324A (es) | 2013-02-01 |
| KR20130043166A (ko) | 2013-04-29 |
| JP2013542913A (ja) | 2013-11-28 |
| EP2585105A4 (en) | 2014-07-09 |
| EP2585105A2 (en) | 2013-05-01 |
| NZ702801A (en) | 2016-08-26 |
| AU2011270672B2 (en) | 2017-01-12 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| ES2643015T3 (es) | Tratamiento del Síndrome de Sanfilippo Tipo B | |
| US11065307B2 (en) | Therapeutic fusion protein comprising an alpha-n-acetylglucosaminidase and a lysosomal targeting moiety | |
| RU2671503C2 (ru) | Способы и композиции для доставки в цнс арилсульфатазы а | |
| AU2017202219B2 (en) | Methods and compositions for cns delivery of iduronate-2-sulfatase | |
| EP2793922B1 (en) | Stable formulations for cns delivery of arylsulfatase a | |
| JP2025521612A (ja) | ヒアルロニダーゼポリペプチドおよびその用途 | |
| US20220133863A1 (en) | Treatment of sanfilippo syndrome type b | |
| RU2783380C2 (ru) | Способы и композиции для доставки в цнс арилсульфатазы а | |
| RU2774112C2 (ru) | Способы и композиции для доставки в цнс идуронат-2-сульфатазы | |
| HK1230922A1 (en) | Methods and compositions for cns delivery of iduronate-2-sulfatase | |
| HK40022682A (en) | Methods and compositions for cns delivery of arylsulfatase a | |
| HK40022682B (en) | Methods and compositions for cns delivery of arylsulfatase a |