JP2018531965A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018531965A5 JP2018531965A5 JP2018521617A JP2018521617A JP2018531965A5 JP 2018531965 A5 JP2018531965 A5 JP 2018531965A5 JP 2018521617 A JP2018521617 A JP 2018521617A JP 2018521617 A JP2018521617 A JP 2018521617A JP 2018531965 A5 JP2018531965 A5 JP 2018531965A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutically acceptable
- acceptable composition
- agent
- therapeutic agent
- composition according
- 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.)
- Granted
Links
- 239000000203 mixture Substances 0.000 claims description 67
- 239000003814 drug Substances 0.000 claims description 50
- 229940124597 therapeutic agent Drugs 0.000 claims description 46
- 238000000034 method Methods 0.000 claims description 44
- 239000000412 dendrimer Substances 0.000 claims description 32
- 229920000736 dendritic polymer Polymers 0.000 claims description 32
- 208000036546 leukodystrophy Diseases 0.000 claims description 30
- 208000034800 Leukoencephalopathies Diseases 0.000 claims description 22
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 22
- 210000002824 peroxisome Anatomy 0.000 claims description 21
- 239000003795 chemical substances by application Substances 0.000 claims description 20
- 208000035475 disorder Diseases 0.000 claims description 17
- 201000011452 Adrenoleukodystrophy Diseases 0.000 claims description 16
- AUYYCJSJGJYCDS-LBPRGKRZSA-N Thyrolar Chemical class IC1=CC(C[C@H](N)C(O)=O)=CC(I)=C1OC1=CC=C(O)C(I)=C1 AUYYCJSJGJYCDS-LBPRGKRZSA-N 0.000 claims description 16
- 210000004885 white matter Anatomy 0.000 claims description 16
- 102000004190 Enzymes Human genes 0.000 claims description 14
- 108090000790 Enzymes Proteins 0.000 claims description 14
- 230000002490 cerebral effect Effects 0.000 claims description 12
- 229940039227 diagnostic agent Drugs 0.000 claims description 11
- 239000000032 diagnostic agent Substances 0.000 claims description 11
- 102100024643 ATP-binding cassette sub-family D member 1 Human genes 0.000 claims description 10
- 108010049137 Member 1 Subfamily D ATP Binding Cassette Transporter Proteins 0.000 claims description 10
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 claims description 10
- 229920000962 poly(amidoamine) Polymers 0.000 claims description 10
- 210000000278 spinal cord Anatomy 0.000 claims description 10
- 150000004669 very long chain fatty acids Chemical class 0.000 claims description 10
- 208000020547 Peroxisomal disease Diseases 0.000 claims description 9
- 239000002260 anti-inflammatory agent Substances 0.000 claims description 9
- 229940121363 anti-inflammatory agent Drugs 0.000 claims description 9
- 102100020970 ATP-binding cassette sub-family D member 2 Human genes 0.000 claims description 8
- 102100022146 Arylsulfatase A Human genes 0.000 claims description 8
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 claims description 8
- 102100028496 Galactocerebrosidase Human genes 0.000 claims description 8
- 108010042681 Galactosylceramidase Proteins 0.000 claims description 8
- 101000783774 Homo sapiens ATP-binding cassette sub-family D member 2 Proteins 0.000 claims description 8
- 238000004519 manufacturing process Methods 0.000 claims description 8
- 230000035755 proliferation Effects 0.000 claims description 8
- 208000011580 syndromic disease Diseases 0.000 claims description 8
- 230000007659 motor function Effects 0.000 claims description 7
- 210000002569 neuron Anatomy 0.000 claims description 7
- 239000005495 thyroid hormone Substances 0.000 claims description 7
- 229940036555 thyroid hormone Drugs 0.000 claims description 7
- 206010058314 Dysplasia Diseases 0.000 claims description 6
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 claims description 6
- 102000006386 Myelin Proteins Human genes 0.000 claims description 6
- 108010083674 Myelin Proteins Proteins 0.000 claims description 6
- 208000018909 Neonatal adrenoleukodystrophy Diseases 0.000 claims description 6
- 208000010796 X-linked adrenoleukodystrophy Diseases 0.000 claims description 6
- 230000003376 axonal effect Effects 0.000 claims description 6
- 210000000133 brain stem Anatomy 0.000 claims description 6
- 230000002950 deficient Effects 0.000 claims description 6
- 210000005012 myelin Anatomy 0.000 claims description 6
- SENLDUJVTGGYIH-UHFFFAOYSA-N n-(2-aminoethyl)-3-[[3-(2-aminoethylamino)-3-oxopropyl]-[2-[bis[3-(2-aminoethylamino)-3-oxopropyl]amino]ethyl]amino]propanamide Chemical compound NCCNC(=O)CCN(CCC(=O)NCCN)CCN(CCC(=O)NCCN)CCC(=O)NCCN SENLDUJVTGGYIH-UHFFFAOYSA-N 0.000 claims description 6
- 210000005250 spinal neuron Anatomy 0.000 claims description 6
- 102100026198 Aspartate-tRNA ligase, mitochondrial Human genes 0.000 claims description 5
- 101000630206 Homo sapiens Aspartate-tRNA ligase, mitochondrial Proteins 0.000 claims description 5
- 208000025121 Leukoencephalopathy with brain stem and spinal cord involvement-high lactate syndrome Diseases 0.000 claims description 5
- 201000010099 disease Diseases 0.000 claims description 5
- 230000004064 dysfunction Effects 0.000 claims description 5
- 150000004668 long chain fatty acids Chemical class 0.000 claims description 5
- OBKXEAXTFZPCHS-UHFFFAOYSA-M 4-phenylbutyrate Chemical group [O-]C(=O)CCCC1=CC=CC=C1 OBKXEAXTFZPCHS-UHFFFAOYSA-M 0.000 claims description 4
- 206010003591 Ataxia Diseases 0.000 claims description 4
- 208000014644 Brain disease Diseases 0.000 claims description 4
- 206010012289 Dementia Diseases 0.000 claims description 4
- 208000032274 Encephalopathy Diseases 0.000 claims description 4
- 208000028226 Krabbe disease Diseases 0.000 claims description 4
- JVTAAEKCZFNVCJ-UHFFFAOYSA-M Lactate Chemical compound CC(O)C([O-])=O JVTAAEKCZFNVCJ-UHFFFAOYSA-M 0.000 claims description 4
- QNVSXXGDAPORNA-UHFFFAOYSA-N Resveratrol Natural products OC1=CC=CC(C=CC=2C=C(O)C(O)=CC=2)=C1 QNVSXXGDAPORNA-UHFFFAOYSA-N 0.000 claims description 4
- LUKBXSAWLPMMSZ-OWOJBTEDSA-N Trans-resveratrol Chemical group C1=CC(O)=CC=C1\C=C\C1=CC(O)=CC(O)=C1 LUKBXSAWLPMMSZ-OWOJBTEDSA-N 0.000 claims description 4
- 230000005856 abnormality Effects 0.000 claims description 4
- 208000002552 acute disseminated encephalomyelitis Diseases 0.000 claims description 4
- 239000003963 antioxidant agent Substances 0.000 claims description 4
- 235000006708 antioxidants Nutrition 0.000 claims description 4
- 230000007850 degeneration Effects 0.000 claims description 4
- OBKXEAXTFZPCHS-UHFFFAOYSA-N gamma-phenylbutyric acid Natural products OC(=O)CCCC1=CC=CC=C1 OBKXEAXTFZPCHS-UHFFFAOYSA-N 0.000 claims description 4
- 230000013632 homeostatic process Effects 0.000 claims description 4
- 230000006540 mitochondrial respiration Effects 0.000 claims description 4
- 230000000069 prophylactic effect Effects 0.000 claims description 4
- 235000021283 resveratrol Nutrition 0.000 claims description 4
- 229940016667 resveratrol Drugs 0.000 claims description 4
- 208000024891 symptom Diseases 0.000 claims description 4
- ZYTXTXAMMDTYDQ-DGEXFFLYSA-N vamorolone Chemical group C([C@H]12)CC3=CC(=O)C=C[C@]3(C)C1=CC[C@@]1(C)[C@H]2C[C@@H](C)[C@]1(O)C(=O)CO ZYTXTXAMMDTYDQ-DGEXFFLYSA-N 0.000 claims description 4
- XUIIKFGFIJCVMT-GFCCVEGCSA-N D-thyroxine Chemical compound IC1=CC(C[C@@H](N)C(O)=O)=CC(I)=C1OC1=CC(I)=C(O)C(I)=C1 XUIIKFGFIJCVMT-GFCCVEGCSA-N 0.000 claims description 3
- 208000021236 Hereditary diffuse leukoencephalopathy with axonal spheroids and pigmented glia Diseases 0.000 claims description 3
- 210000003169 central nervous system Anatomy 0.000 claims description 3
- 208000031513 cyst Diseases 0.000 claims description 3
- 230000006738 locomotor deficit Effects 0.000 claims description 3
- 102000039446 nucleic acids Human genes 0.000 claims description 3
- 108020004707 nucleic acids Proteins 0.000 claims description 3
- 150000007523 nucleic acids Chemical class 0.000 claims description 3
- 230000002739 subcortical effect Effects 0.000 claims description 3
- 229940034208 thyroxine Drugs 0.000 claims description 3
- XUIIKFGFIJCVMT-UHFFFAOYSA-N thyroxine-binding globulin Natural products IC1=CC(CC([NH3+])C([O-])=O)=CC(I)=C1OC1=CC(I)=C(O)C(I)=C1 XUIIKFGFIJCVMT-UHFFFAOYSA-N 0.000 claims description 3
- 206010068533 18q minus syndrome Diseases 0.000 claims description 2
- KISWVXRQTGLFGD-UHFFFAOYSA-N 2-[[2-[[6-amino-2-[[2-[[2-[[5-amino-2-[[2-[[1-[2-[[6-amino-2-[(2,5-diamino-5-oxopentanoyl)amino]hexanoyl]amino]-5-(diaminomethylideneamino)pentanoyl]pyrrolidine-2-carbonyl]amino]-3-hydroxypropanoyl]amino]-5-oxopentanoyl]amino]-5-(diaminomethylideneamino)p Chemical compound C1CCN(C(=O)C(CCCN=C(N)N)NC(=O)C(CCCCN)NC(=O)C(N)CCC(N)=O)C1C(=O)NC(CO)C(=O)NC(CCC(N)=O)C(=O)NC(CCCN=C(N)N)C(=O)NC(CO)C(=O)NC(CCCCN)C(=O)NC(C(=O)NC(CC(C)C)C(O)=O)CC1=CC=C(O)C=C1 KISWVXRQTGLFGD-UHFFFAOYSA-N 0.000 claims description 2
- HVCOBJNICQPDBP-UHFFFAOYSA-N 3-[3-[3,5-dihydroxy-6-methyl-4-(3,4,5-trihydroxy-6-methyloxan-2-yl)oxyoxan-2-yl]oxydecanoyloxy]decanoic acid;hydrate Chemical compound O.OC1C(OC(CC(=O)OC(CCCCCCC)CC(O)=O)CCCCCCC)OC(C)C(O)C1OC1C(O)C(O)C(O)C(C)O1 HVCOBJNICQPDBP-UHFFFAOYSA-N 0.000 claims description 2
- 208000032194 Acute haemorrhagic leukoencephalitis Diseases 0.000 claims description 2
- 208000034014 Adult-onset autosomal dominant leukodystrophy Diseases 0.000 claims description 2
- 208000011403 Alexander disease Diseases 0.000 claims description 2
- DPUOLQHDNGRHBS-UHFFFAOYSA-N Brassidinsaeure Natural products CCCCCCCCC=CCCCCCCCCCCCC(O)=O DPUOLQHDNGRHBS-UHFFFAOYSA-N 0.000 claims description 2
- 208000010482 CADASIL Diseases 0.000 claims description 2
- 206010008025 Cerebellar ataxia Diseases 0.000 claims description 2
- 208000033221 Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy Diseases 0.000 claims description 2
- 208000033935 Cerebral autosomal dominant arteriopathy-subcortical infarcts-leukoencephalopathy Diseases 0.000 claims description 2
- ACTIUHUUMQJHFO-UHFFFAOYSA-N Coenzym Q10 Natural products COC1=C(OC)C(=O)C(CC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)CCC=C(C)C)=C(C)C1=O ACTIUHUUMQJHFO-UHFFFAOYSA-N 0.000 claims description 2
- URXZXNYJPAJJOQ-UHFFFAOYSA-N Erucic acid Natural products CCCCCCC=CCCCCCCCCCCCC(O)=O URXZXNYJPAJJOQ-UHFFFAOYSA-N 0.000 claims description 2
- 229930186217 Glycolipid Natural products 0.000 claims description 2
- 101000728860 Homo sapiens Ribonuclease T2 Proteins 0.000 claims description 2
- 206010058359 Hypogonadism Diseases 0.000 claims description 2
- 102000047918 Myelin Basic Human genes 0.000 claims description 2
- 101710107068 Myelin basic protein Proteins 0.000 claims description 2
- 208000008425 Protein deficiency Diseases 0.000 claims description 2
- 101000713578 Rattus norvegicus Tubulin beta-3 chain Proteins 0.000 claims description 2
- 102100029683 Ribonuclease T2 Human genes 0.000 claims description 2
- 208000021386 Sjogren Syndrome Diseases 0.000 claims description 2
- 201000004525 Zellweger Syndrome Diseases 0.000 claims description 2
- 230000002159 abnormal effect Effects 0.000 claims description 2
- 239000013543 active substance Substances 0.000 claims description 2
- 230000001154 acute effect Effects 0.000 claims description 2
- 230000001919 adrenal effect Effects 0.000 claims description 2
- 201000001452 adult-onset autosomal dominant demyelinating leukodystrophy Diseases 0.000 claims description 2
- 230000002715 bioenergetic effect Effects 0.000 claims description 2
- 208000016886 cerebral arteriopathy with subcortical infarcts and leukoencephalopathy Diseases 0.000 claims description 2
- 206010008118 cerebral infarction Diseases 0.000 claims description 2
- 208000007118 chronic progressive multiple sclerosis Diseases 0.000 claims description 2
- YNNUSGIPVFPVBX-NHCUHLMSSA-N clemastine Chemical group CN1CCC[C@@H]1CCO[C@@](C)(C=1C=CC(Cl)=CC=1)C1=CC=CC=C1 YNNUSGIPVFPVBX-NHCUHLMSSA-N 0.000 claims description 2
- 229960002881 clemastine Drugs 0.000 claims description 2
- 235000017471 coenzyme Q10 Nutrition 0.000 claims description 2
- ACTIUHUUMQJHFO-UPTCCGCDSA-N coenzyme Q10 Chemical group COC1=C(OC)C(=O)C(C\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CC\C=C(/C)CCC=C(C)C)=C(C)C1=O ACTIUHUUMQJHFO-UPTCCGCDSA-N 0.000 claims description 2
- 229940110767 coenzyme Q10 Drugs 0.000 claims description 2
- 230000003111 delayed effect Effects 0.000 claims description 2
- 239000000839 emulsion Substances 0.000 claims description 2
- DPUOLQHDNGRHBS-KTKRTIGZSA-N erucic acid Chemical group CCCCCCCC\C=C/CCCCCCCCCCCC(O)=O DPUOLQHDNGRHBS-KTKRTIGZSA-N 0.000 claims description 2
- 210000004884 grey matter Anatomy 0.000 claims description 2
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 2
- 201000000770 hypomyelinating leukodystrophy 6 Diseases 0.000 claims description 2
- 201000010828 metaphyseal dysplasia Diseases 0.000 claims description 2
- 238000012544 monitoring process Methods 0.000 claims description 2
- 230000023105 myelination Effects 0.000 claims description 2
- 201000001119 neuropathy Diseases 0.000 claims description 2
- 230000007823 neuropathy Effects 0.000 claims description 2
- 230000002611 ovarian Effects 0.000 claims description 2
- 208000033808 peripheral neuropathy Diseases 0.000 claims description 2
- 239000000049 pigment Substances 0.000 claims description 2
- 210000003625 skull Anatomy 0.000 claims description 2
- 239000000243 solution Substances 0.000 claims description 2
- 239000000725 suspension Substances 0.000 claims description 2
- 230000008685 targeting Effects 0.000 claims description 2
- 108700023155 Aspartoacylases Proteins 0.000 claims 2
- 241000282412 Homo Species 0.000 claims 2
- 102000018919 aspartoacylase Human genes 0.000 claims 2
- DEFLNOSTNCSZRB-IDTAVKCVSA-N 9-[(2r,3r,4r,5r)-3,4-dimethoxy-5-(methoxymethyl)oxolan-2-yl]-n-methoxypurin-6-amine Chemical compound CO[C@@H]1[C@H](OC)[C@@H](COC)O[C@H]1N1C2=NC=NC(NOC)=C2N=C1 DEFLNOSTNCSZRB-IDTAVKCVSA-N 0.000 claims 1
- BSYNRYMUTXBXSQ-UHFFFAOYSA-N Aspirin Chemical compound CC(=O)OC1=CC=CC=C1C(O)=O BSYNRYMUTXBXSQ-UHFFFAOYSA-N 0.000 claims 1
- 206010008027 Cerebellar atrophy Diseases 0.000 claims 1
- 208000003098 Ganglion Cysts Diseases 0.000 claims 1
- 208000008498 Infantile Refsum disease Diseases 0.000 claims 1
- 208000005400 Synovial Cyst Diseases 0.000 claims 1
- 201000004810 Vascular dementia Diseases 0.000 claims 1
- 230000002567 autonomic effect Effects 0.000 claims 1
- 230000001413 cellular effect Effects 0.000 claims 1
- 230000031736 central nervous system myelination Effects 0.000 claims 1
- 230000004807 localization Effects 0.000 claims 1
- 230000003137 locomotive effect Effects 0.000 claims 1
- 210000002241 neurite Anatomy 0.000 claims 1
- 210000004498 neuroglial cell Anatomy 0.000 claims 1
- 208000020930 peroxisome biogenesis disorder 1B Diseases 0.000 claims 1
- 230000001737 promoting effect Effects 0.000 claims 1
- GVJHHUAWPYXKBD-UHFFFAOYSA-N (±)-α-Tocopherol Chemical compound OC1=C(C)C(C)=C2OC(CCCC(C)CCCC(C)CCCC(C)C)(C)CCC2=C1C GVJHHUAWPYXKBD-UHFFFAOYSA-N 0.000 description 6
- HYAFETHFCAUJAY-UHFFFAOYSA-N pioglitazone Chemical compound N1=CC(CC)=CC=C1CCOC(C=C1)=CC=C1CC1C(=O)NC(=O)S1 HYAFETHFCAUJAY-UHFFFAOYSA-N 0.000 description 6
- 208000036110 Neuroinflammatory disease Diseases 0.000 description 5
- 230000003959 neuroinflammation Effects 0.000 description 5
- 229940079593 drug Drugs 0.000 description 4
- 229930003427 Vitamin E Natural products 0.000 description 3
- 230000003078 antioxidant effect Effects 0.000 description 3
- 238000003745 diagnosis Methods 0.000 description 3
- WIGCFUFOHFEKBI-UHFFFAOYSA-N gamma-tocopherol Natural products CC(C)CCCC(C)CCCC(C)CCCC1CCC2C(C)C(O)C(C)C(C)C2O1 WIGCFUFOHFEKBI-UHFFFAOYSA-N 0.000 description 3
- 230000036542 oxidative stress Effects 0.000 description 3
- 229960005095 pioglitazone Drugs 0.000 description 3
- 235000019165 vitamin E Nutrition 0.000 description 3
- 229940046009 vitamin E Drugs 0.000 description 3
- 239000011709 vitamin E Substances 0.000 description 3
- YBJHBAHKTGYVGT-ZKWXMUAHSA-N (+)-Biotin Chemical compound N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-N 0.000 description 2
- PWKSKIMOESPYIA-BYPYZUCNSA-N L-N-acetyl-Cysteine Chemical group CC(=O)N[C@@H](CS)C(O)=O PWKSKIMOESPYIA-BYPYZUCNSA-N 0.000 description 2
- 208000005587 Refsum Disease Diseases 0.000 description 2
- 229960004308 acetylcysteine Drugs 0.000 description 2
- 208000030597 adult Refsum disease Diseases 0.000 description 2
- 230000006378 damage Effects 0.000 description 2
- 230000007812 deficiency Effects 0.000 description 2
- 238000001514 detection method Methods 0.000 description 2
- 150000002344 gold compounds Chemical class 0.000 description 2
- 238000012423 maintenance Methods 0.000 description 2
- 230000003961 neuronal insult Effects 0.000 description 2
- 239000000041 non-steroidal anti-inflammatory agent Substances 0.000 description 2
- 229940021182 non-steroidal anti-inflammatory drug Drugs 0.000 description 2
- 239000002294 steroidal antiinflammatory agent Substances 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- 206010003694 Atrophy Diseases 0.000 description 1
- 208000022526 Canavan disease Diseases 0.000 description 1
- 206010053684 Cerebrohepatorenal syndrome Diseases 0.000 description 1
- 206010011732 Cyst Diseases 0.000 description 1
- 208000018175 Hypomyelination-hypogonadotropic hypogonadism-hypodontia syndrome Diseases 0.000 description 1
- 108700010229 Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation Proteins 0.000 description 1
- 206010033892 Paraplegia Diseases 0.000 description 1
- 208000032930 Spastic paraplegia Diseases 0.000 description 1
- 208000027418 Wounds and injury Diseases 0.000 description 1
- 206010048215 Xanthomatosis Diseases 0.000 description 1
- 208000036813 Zellweger spectrum disease Diseases 0.000 description 1
- 230000037444 atrophy Effects 0.000 description 1
- -1 benzafibrate Substances 0.000 description 1
- 235000020958 biotin Nutrition 0.000 description 1
- 239000011616 biotin Substances 0.000 description 1
- 229960002685 biotin Drugs 0.000 description 1
- 210000004027 cell Anatomy 0.000 description 1
- 210000001638 cerebellum Anatomy 0.000 description 1
- 235000014113 dietary fatty acids Nutrition 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 206010015037 epilepsy Diseases 0.000 description 1
- 229930195729 fatty acid Natural products 0.000 description 1
- 239000000194 fatty acid Substances 0.000 description 1
- 150000004665 fatty acids Chemical class 0.000 description 1
- 210000000609 ganglia Anatomy 0.000 description 1
- 230000002518 glial effect Effects 0.000 description 1
- 208000014674 injury Diseases 0.000 description 1
- 210000003007 myelin sheath Anatomy 0.000 description 1
- 230000009707 neogenesis Effects 0.000 description 1
- 238000001228 spectrum Methods 0.000 description 1
- 210000002435 tendon Anatomy 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022087580A JP2022105767A (ja) | 2015-10-29 | 2022-05-30 | ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562248163P | 2015-10-29 | 2015-10-29 | |
| US62/248,163 | 2015-10-29 | ||
| PCT/US2016/059697 WO2017075580A1 (en) | 2015-10-29 | 2016-10-31 | Compositions and methods for treatment of peroxisomal disorders and leukodystrophies |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022087580A Division JP2022105767A (ja) | 2015-10-29 | 2022-05-30 | ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018531965A JP2018531965A (ja) | 2018-11-01 |
| JP2018531965A5 true JP2018531965A5 (enExample) | 2019-12-12 |
| JP7088473B2 JP7088473B2 (ja) | 2022-06-21 |
Family
ID=57233968
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521617A Active JP7088473B2 (ja) | 2015-10-29 | 2016-10-31 | ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 |
| JP2022087580A Pending JP2022105767A (ja) | 2015-10-29 | 2022-05-30 | ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022087580A Pending JP2022105767A (ja) | 2015-10-29 | 2022-05-30 | ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US20170119899A1 (enExample) |
| EP (1) | EP3368088B1 (enExample) |
| JP (2) | JP7088473B2 (enExample) |
| CN (1) | CN108430514B (enExample) |
| AU (1) | AU2016343855B2 (enExample) |
| CA (1) | CA3003589C (enExample) |
| WO (1) | WO2017075580A1 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6342575B2 (ja) * | 2014-08-13 | 2018-06-13 | ザ・ジョンズ・ホプキンス・ユニバーシティー | 脳腫瘍への選択的デンドリマー送達 |
| EP3615086A4 (en) | 2017-04-27 | 2021-01-06 | The Johns Hopkins University | COMPOSITIONS OF DENDRIMERS FOR USE IN ANGIOGRAPHY |
| WO2018208707A1 (en) * | 2017-05-08 | 2018-11-15 | Neurovia, Inc. | Methods and compositions for treating demyelinating diseases |
| CN111615528A (zh) | 2017-11-10 | 2020-09-01 | 约翰霍普金斯大学 | 树枝状聚合物递送系统和其使用方法 |
| US11679104B2 (en) | 2017-12-15 | 2023-06-20 | Duke University | Compositions and methods of enhancing the homing and/or engraftment of hematopoietic cells in the central nervous system |
| EP3866811A4 (en) * | 2018-10-19 | 2022-11-02 | Disarm Therapeutics, Inc. | INHIBITORS OF SARM1 IN COMBINATION WITH NAD+ OR A NAD+ PRECURSOR |
| WO2020142373A1 (en) * | 2019-01-02 | 2020-07-09 | Mayo Foundation For Medical Education And Research | Methods and materials for treating leukodystrophies |
| JP7759322B2 (ja) | 2019-12-04 | 2025-10-23 | アシュバッタ セラピューティクス, インコーポレイテッド | 眼に薬物送達するためのデンドリマー組成物および方法 |
| CN116075322A (zh) * | 2020-04-24 | 2023-05-05 | 阿什瓦塔治疗股份有限公司 | 用于治疗重度急性呼吸窘迫综合征的树枝状大分子组合物和方法 |
| PH12023550133A1 (en) * | 2020-07-17 | 2024-06-24 | Ashvattha Therapeutics Inc | Dendrimer compositions and methods for drug delivery to injured kidney |
| WO2023081750A1 (en) * | 2021-11-04 | 2023-05-11 | The Children's Hospital Of Philadelphia | Compositions and methods for treating metachromatic leukodystrophy disease and related disorders |
| WO2024081687A2 (en) * | 2022-10-12 | 2024-04-18 | The Johns Hopkins University | Method of treating lbsl by enhancing dars2 expression |
| CN116082153A (zh) * | 2022-12-09 | 2023-05-09 | 长沙贝塔医药科技有限公司 | 一种丙烯酸酯类衍生物-13c的制备方法 |
| EP4410284A1 (en) * | 2023-02-02 | 2024-08-07 | Apteeus | Compound and method for the treatment of zellweger spectrum disorder |
| CN117643581A (zh) * | 2023-11-29 | 2024-03-05 | 徐州医科大学 | 白藜芦醇在制备激活sirt1基因的药物中的应用 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2212047C (en) | 1995-02-07 | 2010-11-16 | Saul W. Brusilow | Triglycerides and ethyl esters of phenylalkanoic acid and phenylalkenoic acid useful in treatment of various disorders |
| CA2344717A1 (en) * | 1998-09-28 | 2000-04-06 | The Johns Hopkins University | Adrenoleukodystrophy treatments and drug screening |
| US8148356B2 (en) | 2005-08-24 | 2012-04-03 | Cumberland Pharmaceuticals, Inc. | Acetylcysteine composition and uses therefor |
| TW200817369A (en) * | 2006-05-22 | 2008-04-16 | Elan Pharm Inc | Preparation of polymer conjugates of therapeutic, agricultural and food additive compounds |
| JP5484339B2 (ja) * | 2007-10-05 | 2014-05-07 | ウェイン ステート ユニバーシティー | 合成物の持続的な放出のためのデンドリマー |
| US8642012B2 (en) | 2008-04-29 | 2014-02-04 | Hyperion Therapeutics, Inc. | Methods of treatment using ammonia-scavenging drugs |
| WO2009142754A1 (en) * | 2008-05-22 | 2009-11-26 | Goverment Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Dendritic conjugates and methods of use |
| EP2442797B1 (en) * | 2009-06-15 | 2020-01-01 | Wayne State University | Dendrimer based nanodevices for therapeutic and imaging purposes |
| WO2012012640A1 (en) | 2010-07-21 | 2012-01-26 | Cumberland Pharmaceuticals, Inc. | Acetycysteine compositions and methods of use thereof |
| BR112014007357B1 (pt) | 2011-09-30 | 2022-12-06 | Immedica Pharma Ab | Método para determinar o ajuste da dosagem de um fármaco de varredura de nitrogênio |
| JP2016517883A (ja) * | 2013-05-03 | 2016-06-20 | オレゴン ヘルス アンド サイエンス ユニバーシティー | X連鎖副腎白質ジストロフィーの処置におけるソベチロムの使用 |
| US20160122406A1 (en) * | 2013-06-07 | 2016-05-05 | The Administrators Of The Tulane Educational Fund | Analogs of pituitary adenylate cyclase-activating polypeptide (pacap) and methods for their use |
| WO2015027068A1 (en) * | 2013-08-21 | 2015-02-26 | Concordia University | Dendrimer-resveratrol complex |
| US20170232120A1 (en) * | 2014-08-13 | 2017-08-17 | The Johns Hopkins University | Dendrimer compositions and use in treatment of neurological and cns disorders |
-
2016
- 2016-10-31 WO PCT/US2016/059697 patent/WO2017075580A1/en not_active Ceased
- 2016-10-31 CA CA3003589A patent/CA3003589C/en active Active
- 2016-10-31 AU AU2016343855A patent/AU2016343855B2/en active Active
- 2016-10-31 CN CN201680073817.9A patent/CN108430514B/zh active Active
- 2016-10-31 EP EP16791282.3A patent/EP3368088B1/en active Active
- 2016-10-31 JP JP2018521617A patent/JP7088473B2/ja active Active
- 2016-10-31 US US15/339,486 patent/US20170119899A1/en not_active Abandoned
-
2021
- 2021-09-20 US US17/479,400 patent/US20220071923A1/en not_active Abandoned
-
2022
- 2022-05-30 JP JP2022087580A patent/JP2022105767A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018531965A5 (enExample) | ||
| Sun et al. | Promotion of astrocyte-neuron glutamate-glutamine shuttle by SCFA contributes to the alleviation of Alzheimer's disease | |
| Cepeda et al. | The role of dopamine in Huntington's disease | |
| Ward et al. | Neurodegenerative diseases and therapeutic strategies using iron chelators | |
| CN102764279B (zh) | 改变细胞功能的方法和组合物 | |
| JP5616631B2 (ja) | 細胞機能を変化させるための方法および組成物 | |
| US20110038889A1 (en) | Methods and compositions for altering cell function | |
| Kato et al. | Protection of dopaminergic neurons with a novel astrocyte modulating agent (R)-(−)-2-propyloctanoic acid (ONO-2506) in an MPTP-mouse model of Parkinson's disease | |
| Lapchak | Therapeutic potentials for glial cell line-derived neurotrophic factor (GDNF) based upon pharmacological activities in the CNS | |
| US8865763B2 (en) | Methods and compositions for altering cell function | |
| Shaw | Dopamine excess and/or norepinephrine and epinephrine deficiency in autistic patients due to prenatal and/or postnatal deficiency of dopamine beta-hydroxylase | |
| Latimer | Tardive dyskinesia: a review | |
| US8871715B2 (en) | Use of selenium compounds, especially selenium yeasts for altering cognitive function | |
| US20070155687A1 (en) | Methods and compositions for altering cell function | |
| Lv et al. | Research progress of kynurenine pathway in Alzheimer's disease. | |
| CA2524551C (en) | Use of selenium yeasts in the treatment of alzheimer's disease | |
| Groves et al. | Adult vitamin D deficiency and adverse brain outcomes | |
| IMRAN et al. | Experimental animal models of Parkinson’s disease: an overview | |
| Ogawa et al. | Parkinson’s disease, dopamine and free radicals | |
| Carlotta et al. | Ketogenic diet and neurodegenerative diseases: A focus on Alzheimer’s disease, Parkinson’s disease and amyotrofic lateral sclerosis | |
| Okafor | Design of copper-selective peptide shuttles to prevent toxicity of the Cu-amyloid-β complex linked to Alzheimer's disease | |
| AU2013205053A1 (en) | Method and compositions for altering cell function | |
| De Marchi et al. | Co-occurrence of OCD and HD in a nuclear family | |
| Kirubakaran et al. | and Alzheimer’s Disease | |
| Pavese et al. | P3. 024 Progression of monoaminergic dysfunction in Parkinson's disease: a longitudinal 18F-Dopa PET study |