JP2021500029A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2021500029A5 JP2021500029A5 JP2020522007A JP2020522007A JP2021500029A5 JP 2021500029 A5 JP2021500029 A5 JP 2021500029A5 JP 2020522007 A JP2020522007 A JP 2020522007A JP 2020522007 A JP2020522007 A JP 2020522007A JP 2021500029 A5 JP2021500029 A5 JP 2021500029A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- raav
- polypeptide
- composition
- tmck
- 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.)
- Pending
Links
- 102100029268 Neurotrophin-3 Human genes 0.000 claims description 140
- 150000007523 nucleic acids Chemical class 0.000 claims description 126
- 108020004707 nucleic acids Proteins 0.000 claims description 119
- 102000039446 nucleic acids Human genes 0.000 claims description 119
- 108090000742 Neurotrophin 3 Proteins 0.000 claims description 81
- 101000634196 Homo sapiens Neurotrophin-3 Proteins 0.000 claims description 69
- 239000000203 mixture Substances 0.000 claims description 65
- 241000702421 Dependoparvovirus Species 0.000 claims description 63
- 239000002773 nucleotide Substances 0.000 claims description 41
- 125000003729 nucleotide group Chemical group 0.000 claims description 41
- 238000007918 intramuscular administration Methods 0.000 claims description 38
- 210000003205 muscle Anatomy 0.000 claims description 30
- 238000000034 method Methods 0.000 claims description 26
- 208000033808 peripheral neuropathy Diseases 0.000 claims description 19
- 238000010255 intramuscular injection Methods 0.000 claims description 18
- 239000007927 intramuscular injection Substances 0.000 claims description 18
- 238000002347 injection Methods 0.000 claims description 17
- 239000007924 injection Substances 0.000 claims description 17
- 201000001119 neuropathy Diseases 0.000 claims description 17
- 230000007823 neuropathy Effects 0.000 claims description 17
- 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 claims description 13
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 13
- 201000000585 muscular atrophy Diseases 0.000 claims description 13
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 9
- 239000002245 particle Substances 0.000 claims description 8
- 206010028289 Muscle atrophy Diseases 0.000 claims description 7
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 7
- 201000010099 disease Diseases 0.000 claims description 7
- 230000002459 sustained effect Effects 0.000 claims description 7
- 102000057714 human NTF3 Human genes 0.000 claims description 6
- 230000037257 muscle growth Effects 0.000 claims description 6
- 230000007614 genetic variation Effects 0.000 claims description 5
- 208000015181 infectious disease Diseases 0.000 claims description 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 4
- 208000030939 Chronic inflammatory demyelinating polyneuropathy Diseases 0.000 claims description 4
- 206010061818 Disease progression Diseases 0.000 claims description 4
- 241000725303 Human immunodeficiency virus Species 0.000 claims description 4
- 208000021642 Muscular disease Diseases 0.000 claims description 4
- 230000001363 autoimmune Effects 0.000 claims description 4
- 201000005795 chronic inflammatory demyelinating polyneuritis Diseases 0.000 claims description 4
- 239000002131 composite material Substances 0.000 claims description 4
- 230000005750 disease progression Effects 0.000 claims description 4
- 201000009623 Myopathy Diseases 0.000 claims description 3
- 230000004936 stimulating effect Effects 0.000 claims description 3
- 208000011580 syndromic disease Diseases 0.000 claims description 3
- 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
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 2
- 208000003808 Amyloid Neuropathies Diseases 0.000 claims description 2
- 208000000412 Avitaminosis Diseases 0.000 claims description 2
- 235000008733 Citrus aurantifolia Nutrition 0.000 claims description 2
- 208000015943 Coeliac disease Diseases 0.000 claims description 2
- 206010010957 Copper deficiency Diseases 0.000 claims description 2
- 108020004414 DNA Proteins 0.000 claims description 2
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 2
- 229930186217 Glycolipid Natural products 0.000 claims description 2
- 206010019663 Hepatic failure Diseases 0.000 claims description 2
- 206010020631 Hypergammaglobulinaemia benign monoclonal Diseases 0.000 claims description 2
- 206010020880 Hypertrophy Diseases 0.000 claims description 2
- 208000013016 Hypoglycemia Diseases 0.000 claims description 2
- 206010021135 Hypovitaminosis Diseases 0.000 claims description 2
- 206010024229 Leprosy Diseases 0.000 claims description 2
- 108010059343 MM Form Creatine Kinase Proteins 0.000 claims description 2
- 208000023137 Myotoxicity Diseases 0.000 claims description 2
- 206010028980 Neoplasm Diseases 0.000 claims description 2
- 206010053869 POEMS syndrome Diseases 0.000 claims description 2
- 206010036105 Polyneuropathy Diseases 0.000 claims description 2
- 208000001647 Renal Insufficiency Diseases 0.000 claims description 2
- 208000024799 Thyroid disease Diseases 0.000 claims description 2
- 235000011941 Tilia x europaea Nutrition 0.000 claims description 2
- 206010047115 Vasculitis Diseases 0.000 claims description 2
- 206010047631 Vitamin E deficiency Diseases 0.000 claims description 2
- 230000002159 abnormal effect Effects 0.000 claims description 2
- 230000001154 acute effect Effects 0.000 claims description 2
- 206010002022 amyloidosis Diseases 0.000 claims description 2
- 201000011510 cancer Diseases 0.000 claims description 2
- FDJOLVPMNUYSCM-UVKKECPRSA-L cobalt(3+);[(2r,3s,4r,5s)-5-(5,6-dimethylbenzimidazol-1-yl)-4-hydroxy-2-(hydroxymethyl)oxolan-3-yl] [(2r)-1-[3-[(2r,3r,4z,7s,9z,12s,13s,14z,17s,18s,19r)-2,13,18-tris(2-amino-2-oxoethyl)-7,12,17-tris(3-amino-3-oxopropyl)-3,5,8,8,13,15,18,19-octamethyl-2,7, Chemical compound [Co+3].N#[C-].C1([C@H](CC(N)=O)[C@@]2(C)CCC(=O)NC[C@@H](C)OP([O-])(=O)O[C@H]3[C@H]([C@H](O[C@@H]3CO)N3C4=CC(C)=C(C)C=C4N=C3)O)[N-]\C2=C(C)/C([C@H](C\2(C)C)CCC(N)=O)=N/C/2=C\C([C@H]([C@@]/2(CC(N)=O)C)CCC(N)=O)=N\C\2=C(C)/C2=N[C@]1(C)[C@@](C)(CC(N)=O)[C@@H]2CCC(N)=O FDJOLVPMNUYSCM-UVKKECPRSA-L 0.000 claims description 2
- 208000018631 connective tissue disease Diseases 0.000 claims description 2
- 230000007812 deficiency Effects 0.000 claims description 2
- 206010061811 demyelinating polyneuropathy Diseases 0.000 claims description 2
- 206010012601 diabetes mellitus Diseases 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 239000003623 enhancer Substances 0.000 claims description 2
- 230000002218 hypoglycaemic effect Effects 0.000 claims description 2
- 208000003532 hypothyroidism Diseases 0.000 claims description 2
- 230000002989 hypothyroidism Effects 0.000 claims description 2
- 230000001771 impaired effect Effects 0.000 claims description 2
- 230000002458 infectious effect Effects 0.000 claims description 2
- 230000002757 inflammatory effect Effects 0.000 claims description 2
- 201000006370 kidney failure Diseases 0.000 claims description 2
- 239000004571 lime Substances 0.000 claims description 2
- 150000002632 lipids Chemical class 0.000 claims description 2
- 208000019423 liver disease Diseases 0.000 claims description 2
- 230000005976 liver dysfunction Effects 0.000 claims description 2
- 230000004060 metabolic process Effects 0.000 claims description 2
- 208000012268 mitochondrial disease Diseases 0.000 claims description 2
- 201000005328 monoclonal gammopathy of uncertain significance Diseases 0.000 claims description 2
- 208000018360 neuromuscular disease Diseases 0.000 claims description 2
- 230000002093 peripheral effect Effects 0.000 claims description 2
- 230000008488 polyadenylation Effects 0.000 claims description 2
- 230000007824 polyneuropathy Effects 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 102200150628 rs151220873 Human genes 0.000 claims description 2
- 102200079768 rs2233463 Human genes 0.000 claims description 2
- 208000001076 sarcopenia Diseases 0.000 claims description 2
- 210000002966 serum Anatomy 0.000 claims description 2
- 230000008359 toxicosis Effects 0.000 claims description 2
- 230000002485 urinary effect Effects 0.000 claims description 2
- 208000030401 vitamin deficiency disease Diseases 0.000 claims description 2
- 206010003694 Atrophy Diseases 0.000 claims 1
- 206010012335 Dependence Diseases 0.000 claims 1
- 208000003098 Ganglion Cysts Diseases 0.000 claims 1
- 208000000857 Hepatic Insufficiency Diseases 0.000 claims 1
- 208000019758 Hypergammaglobulinemia Diseases 0.000 claims 1
- 208000005400 Synovial Cyst Diseases 0.000 claims 1
- 230000037444 atrophy Effects 0.000 claims 1
- 201000010901 lateral sclerosis Diseases 0.000 claims 1
- 206010025482 malaise Diseases 0.000 claims 1
- 208000005264 motor neuron disease Diseases 0.000 claims 1
- 230000020763 muscle atrophy Effects 0.000 claims 1
- 230000003387 muscular Effects 0.000 claims 1
- 210000001087 myotubule Anatomy 0.000 claims 1
- 235000016709 nutrition Nutrition 0.000 claims 1
- 230000035764 nutrition Effects 0.000 claims 1
- 208000025223 poliovirus infection Diseases 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 description 15
- 239000013603 viral vector Substances 0.000 description 6
- 102000004420 Creatine Kinase Human genes 0.000 description 3
- 108010042126 Creatine kinase Proteins 0.000 description 3
- 230000002146 bilateral effect Effects 0.000 description 3
- 210000003141 lower extremity Anatomy 0.000 description 3
- 210000001364 upper extremity Anatomy 0.000 description 3
- 239000013598 vector Substances 0.000 description 3
- 208000028389 Nerve injury Diseases 0.000 description 2
- 239000003814 drug Substances 0.000 description 2
- 229940079593 drug Drugs 0.000 description 2
- 238000004519 manufacturing process Methods 0.000 description 2
- 230000008764 nerve damage Effects 0.000 description 2
- 206010060880 Monoclonal gammopathy Diseases 0.000 description 1
- 206010027910 Mononeuritis Diseases 0.000 description 1
- 206010029240 Neuritis Diseases 0.000 description 1
- 238000002266 amputation Methods 0.000 description 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 description 1
- 230000009286 beneficial effect Effects 0.000 description 1
- 230000006835 compression Effects 0.000 description 1
- 238000007906 compression Methods 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 208000007903 liver failure Diseases 0.000 description 1
- 231100000835 liver failure Toxicity 0.000 description 1
- 208000013734 mononeuritis simplex Diseases 0.000 description 1
- 201000005518 mononeuropathy Diseases 0.000 description 1
- 210000002161 motor neuron Anatomy 0.000 description 1
- 201000006938 muscular dystrophy Diseases 0.000 description 1
- 235000015097 nutrients Nutrition 0.000 description 1
- 150000004032 porphyrins Chemical class 0.000 description 1
- 230000000472 traumatic effect Effects 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023151106A JP2023166580A (ja) | 2017-10-20 | 2023-09-19 | Ｎｔ－３遺伝子治療のための方法及び材料 |
| JP2025127206A JP2025157575A (ja) | 2017-10-20 | 2025-07-30 | Ｎｔ－３遺伝子治療のための方法及び材料 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762574828P | 2017-10-20 | 2017-10-20 | |
| US62/574,828 | 2017-10-20 | ||
| US201862676687P | 2018-05-25 | 2018-05-25 | |
| US62/676,687 | 2018-05-25 | ||
| US201862741335P | 2018-10-04 | 2018-10-04 | |
| US62/741,335 | 2018-10-04 | ||
| PCT/US2018/056765 WO2019079755A1 (en) | 2017-10-20 | 2018-10-19 | METHODS AND MATERIALS FOR NT-3 GENE THERAPY |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023151106A Division JP2023166580A (ja) | 2017-10-20 | 2023-09-19 | Ｎｔ－３遺伝子治療のための方法及び材料 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021500029A JP2021500029A (ja) | 2021-01-07 |
| JP2021500029A5 true JP2021500029A5 (enExample) | 2021-12-02 |
Family
ID=66173875
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020522007A Pending JP2021500029A (ja) | 2017-10-20 | 2018-10-19 | Ｎｔ−３遺伝子治療のための方法及び材料 |
| JP2023151106A Pending JP2023166580A (ja) | 2017-10-20 | 2023-09-19 | Ｎｔ－３遺伝子治療のための方法及び材料 |
| JP2025127206A Pending JP2025157575A (ja) | 2017-10-20 | 2025-07-30 | Ｎｔ－３遺伝子治療のための方法及び材料 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023151106A Pending JP2023166580A (ja) | 2017-10-20 | 2023-09-19 | Ｎｔ－３遺伝子治療のための方法及び材料 |
| JP2025127206A Pending JP2025157575A (ja) | 2017-10-20 | 2025-07-30 | Ｎｔ－３遺伝子治療のための方法及び材料 |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US11926653B2 (enExample) |
| EP (1) | EP3697916A4 (enExample) |
| JP (3) | JP2021500029A (enExample) |
| KR (2) | KR20240164969A (enExample) |
| CN (2) | CN119193705A (enExample) |
| AU (2) | AU2018351528B2 (enExample) |
| BR (1) | BR112020007790A2 (enExample) |
| CA (1) | CA3079416A1 (enExample) |
| CL (1) | CL2020001060A1 (enExample) |
| CO (1) | CO2020006148A2 (enExample) |
| IL (2) | IL321310A (enExample) |
| MA (1) | MA50412A (enExample) |
| MX (2) | MX2020004035A (enExample) |
| MY (1) | MY207563A (enExample) |
| SA (1) | SA520411798B1 (enExample) |
| SG (1) | SG11202003285PA (enExample) |
| TW (2) | TWI879721B (enExample) |
| WO (1) | WO2019079755A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102877920B1 (ko) | 2015-11-16 | 2025-10-30 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법 |
| SG11202006722RA (en) | 2018-01-31 | 2020-08-28 | Res Inst Nationwide Childrens Hospital | Gene therapy for limb-girdle muscular dystrophy type 2c |
| KR20210028162A (ko) | 2018-06-29 | 2021-03-11 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 |
| CA3131390A1 (en) | 2019-02-26 | 2020-09-03 | Research Institute Of Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
| GB201907882D0 (en) * | 2019-06-03 | 2019-07-17 | Cyprus Foundation For Muscular Dystrophy Res | Methods |
| HRP20240505T1 (hr) | 2019-08-21 | 2024-07-05 | Research Institute At Nationwide Children's Hospital | Dostava alfa-sarkoglikana vektorom povezanim s adenovirusom i liječenje mišićne distrofije |
| EP4025258A4 (en) * | 2019-09-03 | 2023-09-06 | University of Cincinnati | METHODS AND COMPOSITIONS FOR THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS |
| WO2021211705A1 (en) * | 2020-04-14 | 2021-10-21 | Research Institute At Nationwide Children's Hospital | Treatment of sarcopenia using nt-3 gene therapy |
| WO2021211713A1 (en) * | 2020-04-14 | 2021-10-21 | Research Institute At Nationwide Children's Hospital | Treatment of charcot-marie-tooth axonal type 2d using nt-3 gene therapy |
| CN116440252A (zh) * | 2020-06-16 | 2023-07-18 | 温州医科大学附属第二医院（温州医科大学附属育英儿童医院） | 神经营养因子3在制备提高睾丸中睾酮含量的药物中的应用 |
| EP4108263A3 (en) * | 2021-06-02 | 2023-03-22 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| CN115838725B (zh) * | 2022-12-30 | 2023-09-08 | 广州派真生物技术有限公司 | 在哺乳动物心脏中特异性启动基因的启动子序列及其应用 |
| AU2024265705A1 (en) | 2023-05-02 | 2025-11-20 | Research Institute At Nationwide Children's Hospital | Gene therapy for treatment of protein misfolding diseases |
| WO2024229115A1 (en) * | 2023-05-02 | 2024-11-07 | Research Institute At Nationwide Children's Hospital | Treatment of multiple sclerosis using nt-3 gene therapy |
| CN119899809A (zh) * | 2025-01-07 | 2025-04-29 | 广州派真生物技术有限公司 | 一种提高重组腺相关病毒生产效率的方法及应用 |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| ATE272123T1 (de) | 1993-11-09 | 2004-08-15 | Ohio Med College | Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren |
| EP0733103B1 (en) | 1993-11-09 | 2004-03-03 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| JPH11511326A (ja) | 1995-08-30 | 1999-10-05 | ジエンザイム コーポレイション | アデノウィルスおよびａａｖの精製 |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| ATE465267T1 (de) | 1996-09-06 | 2010-05-15 | Univ Pennsylvania | Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen |
| WO1999011764A2 (en) | 1997-09-05 | 1999-03-11 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of recombinant aav vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| WO2010089706A1 (en) * | 2009-02-04 | 2010-08-12 | Ecole Polytechnique Federale De Lausanne (Epfl) | Use of igf-1 and nt-3 in the treatment of x-linked adrenoleukodystrophy |
| PT2529020T (pt) | 2010-01-28 | 2018-07-30 | Childrens Hospital Philadelphia | Plataforma de fabrico escalável para purificação de vetor viral e vetores virais assim purificados para utilização na terapia génica |
| ES2990023T3 (es) | 2011-07-25 | 2024-11-28 | Nationwide Childrens Hospital Inc | Productos de virus recombinantes y métodos para inhibición de la expresión de DUX4 |
| KR20160130392A (ko) | 2014-02-18 | 2016-11-11 | 듀크 유니버시티 | 바이러스 복제의 불활성화를 위한 조성물 및 그의 제조 및 사용 방법 |
| KR20160068558A (ko) * | 2014-12-05 | 2016-06-15 | 삼성바이오에피스 주식회사 | 마우스 cmv 프로모터를 포함하는 융합 폴리뉴클레오타이드 및 이를 이용한 목적 폴리펩타이드의 생산 방법 |
| WO2016100963A1 (en) | 2014-12-19 | 2016-06-23 | Research Institute At Nationwide Children's Hospital | Pyruvate compounds for treatment of peripheral neuropathy |
| IL258005B2 (en) | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
| GB2547179A (en) | 2015-10-26 | 2017-08-16 | Quethera Ltd | Genetic construct |
| IL259178B (en) | 2015-11-12 | 2022-08-01 | Res Inst Nationwide Childrens Hospital | A vector encoding ano5 for the treatment of muscular dystrophy |
| WO2017165806A1 (en) | 2016-03-25 | 2017-09-28 | Periphagen, Inc. | Hsv vectors for delivery of nt3 and treatment of cipn |
| MX2018011928A (es) | 2016-03-30 | 2019-03-28 | Spark Therapeutics Inc | Linea celular para produccion de proteina recombinante y/o vector viral. |
| MA45477A (fr) * | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
| HUE066442T2 (hu) | 2016-04-15 | 2024-08-28 | Res Institute At Nationwide | ß-szarkoglikán és mikroRNS-29 adenoasszociáltvírus-vektorral történõ bejuttatása és izomdisztrófia kezelése |
| WO2018170408A1 (en) | 2017-03-17 | 2018-09-20 | Research Institute At Nationwide Children's Hospital, Inc. | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
-
2018
- 2018-10-19 EP EP18867716.5A patent/EP3697916A4/en active Pending
- 2018-10-19 KR KR1020247037712A patent/KR20240164969A/ko active Pending
- 2018-10-19 KR KR1020207014064A patent/KR20200083495A/ko not_active Ceased
- 2018-10-19 JP JP2020522007A patent/JP2021500029A/ja active Pending
- 2018-10-19 MX MX2020004035A patent/MX2020004035A/es unknown
- 2018-10-19 AU AU2018351528A patent/AU2018351528B2/en active Active
- 2018-10-19 US US16/757,176 patent/US11926653B2/en active Active
- 2018-10-19 IL IL321310A patent/IL321310A/en unknown
- 2018-10-19 CA CA3079416A patent/CA3079416A1/en active Pending
- 2018-10-19 SG SG11202003285PA patent/SG11202003285PA/en unknown
- 2018-10-19 BR BR112020007790-9A patent/BR112020007790A2/pt unknown
- 2018-10-19 MA MA050412A patent/MA50412A/fr unknown
- 2018-10-19 WO PCT/US2018/056765 patent/WO2019079755A1/en not_active Ceased
- 2018-10-19 CN CN202411339680.XA patent/CN119193705A/zh active Pending
- 2018-10-19 MY MYPI2020001966A patent/MY207563A/en unknown
- 2018-10-19 CN CN201880068238.4A patent/CN111433367A/zh active Pending
- 2018-10-22 TW TW107137220A patent/TWI879721B/zh active
- 2018-10-22 TW TW114113642A patent/TW202532645A/zh unknown
-
2020
- 2020-04-16 IL IL273976A patent/IL273976B2/en unknown
- 2020-04-19 SA SA520411798A patent/SA520411798B1/ar unknown
- 2020-04-20 CL CL2020001060A patent/CL2020001060A1/es unknown
- 2020-05-19 CO CONC2020/0006148A patent/CO2020006148A2/es unknown
- 2020-07-13 MX MX2025010166A patent/MX2025010166A/es unknown
-
2023
- 2023-09-19 JP JP2023151106A patent/JP2023166580A/ja active Pending
-
2024
- 2024-03-08 US US18/600,267 patent/US20240383956A1/en active Pending
-
2025
- 2025-01-31 AU AU2025200649A patent/AU2025200649A1/en active Pending
- 2025-07-30 JP JP2025127206A patent/JP2025157575A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021500029A5 (enExample) | ||
| US20240109938A1 (en) | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof | |
| AU2018304194B2 (en) | Apheresis methods and uses | |
| IL320714A (en) | Gene therapy for limb-girdle muscular dystrophy type 2c | |
| EP3113787A1 (en) | Improved raav vectors and methods for transduction of photoreceptors and rpe cells | |
| EP3134113A1 (en) | Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus | |
| RU2751592C2 (ru) | Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе | |
| JP2023501263A (ja) | 第ｖｉｉｉ因子ポリペプチド | |
| JP2024511024A (ja) | ２１－ヒドロキシラーゼ欠損症の遺伝子治療 | |
| JP2022519596A (ja) | Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達 | |
| CN115836128A (zh) | 用于基因疗法的编码葡萄糖-6-磷酸酶(G6Pase-a)的载体 | |
| US20250207126A1 (en) | Modified muscle-specific promoters | |
| JP7684296B2 (ja) | Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法 | |
| CA3207161A1 (en) | Methods and compositions for treatment of friedreich's ataxia | |
| US20220136005A1 (en) | One-step gene therapy for duchenne muscular dystrophy via gene replacement and anti-inflammation | |
| US20250144243A1 (en) | Aav capsids for improved heart transduction and detargeting of liver | |
| JP2025534651A (ja) | 呼吸困難を伴う脊髄性筋萎縮１型（ｓｍａｒｄ１）及びシャルコー・マリー・トゥース２ｓ型（ｃｍｔ２ｓ）を治療するためのアデノ随伴ウイルス送達 | |
| JP2024515623A (ja) | 髄腔内送達によってピット・ホプキンス症候群を治療するためのメチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルス | |
| JP2024513533A (ja) | 血友病における血友病性関節症の予防及び／又は治療のための方法及び手段 | |
| MX2024010002A (es) | Metodo para reducir la fluctuacion del cst en amd neovascular mediante un virus adenoasociado recombinante | |
| JP2024156872A (ja) | ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法 | |
| RU2021102985A (ru) | Генная терапия нейродегенеративных нарушений | |
| NZ748736B2 (en) | Treatment of complement-mediated disorders |