JP2013531471A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2013531471A5 JP2013531471A5 JP2013506330A JP2013506330A JP2013531471A5 JP 2013531471 A5 JP2013531471 A5 JP 2013531471A5 JP 2013506330 A JP2013506330 A JP 2013506330A JP 2013506330 A JP2013506330 A JP 2013506330A JP 2013531471 A5 JP2013531471 A5 JP 2013531471A5
- Authority
- JP
- Japan
- Prior art keywords
- subject
- raav
- administration
- cns
- vector
- 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
- 210000003169 central nervous system Anatomy 0.000 claims 32
- 239000013598 vector Substances 0.000 claims 29
- 210000001519 tissue Anatomy 0.000 claims 21
- 239000008194 pharmaceutical composition Substances 0.000 claims 19
- 241000702421 Dependoparvovirus Species 0.000 claims 16
- 108020004707 nucleic acids Proteins 0.000 claims 15
- 102000039446 nucleic acids Human genes 0.000 claims 15
- 150000007523 nucleic acids Chemical class 0.000 claims 15
- 108090000623 proteins and genes Proteins 0.000 claims 13
- 108700019146 Transgenes Proteins 0.000 claims 12
- 108090000565 Capsid Proteins Proteins 0.000 claims 10
- 102100023321 Ceruloplasmin Human genes 0.000 claims 10
- 108091070501 miRNA Proteins 0.000 claims 9
- 239000002679 microRNA Substances 0.000 claims 9
- 108010005939 Ciliary Neurotrophic Factor Proteins 0.000 claims 8
- 102100031614 Ciliary neurotrophic factor Human genes 0.000 claims 8
- 102000036509 GTP Cyclohydrolase Human genes 0.000 claims 8
- 108010023555 GTP Cyclohydrolase Proteins 0.000 claims 8
- 241000699666 Mus <mouse, genus> Species 0.000 claims 8
- 108010025020 Nerve Growth Factor Proteins 0.000 claims 8
- 102000015336 Nerve Growth Factor Human genes 0.000 claims 8
- 229940053128 nerve growth factor Drugs 0.000 claims 8
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 6
- 241000700159 Rattus Species 0.000 claims 5
- 150000001413 amino acids Chemical class 0.000 claims 5
- 230000002518 glial effect Effects 0.000 claims 5
- 230000002401 inhibitory effect Effects 0.000 claims 5
- 108020004999 messenger RNA Proteins 0.000 claims 5
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 4
- 229940088872 Apoptosis inhibitor Drugs 0.000 claims 4
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 claims 4
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 claims 4
- 208000022526 Canavan disease Diseases 0.000 claims 4
- 102000034615 Glial cell line-derived neurotrophic factor Human genes 0.000 claims 4
- 108091010837 Glial cell line-derived neurotrophic factor Proteins 0.000 claims 4
- 102000008221 Superoxide Dismutase-1 Human genes 0.000 claims 4
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 claims 4
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 4
- 239000000158 apoptosis inhibitor Substances 0.000 claims 4
- 108010046910 brain-derived growth factor Proteins 0.000 claims 4
- 239000003102 growth factor Substances 0.000 claims 4
- 108091006086 inhibitor proteins Proteins 0.000 claims 4
- 238000007913 intrathecal administration Methods 0.000 claims 4
- 210000004962 mammalian cell Anatomy 0.000 claims 4
- 239000000203 mixture Substances 0.000 claims 4
- 230000009223 neuronal apoptosis Effects 0.000 claims 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 3
- 241000282553 Macaca Species 0.000 claims 3
- 238000007914 intraventricular administration Methods 0.000 claims 3
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 108020005544 Antisense RNA Proteins 0.000 claims 2
- 101100222092 Caenorhabditis elegans csp-3 gene Proteins 0.000 claims 2
- 102000004190 Enzymes Human genes 0.000 claims 2
- 108090000790 Enzymes Proteins 0.000 claims 2
- 241000282326 Felis catus Species 0.000 claims 2
- 108010025815 Kanamycin Kinase Proteins 0.000 claims 2
- 108091027967 Small hairpin RNA Proteins 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 239000003184 complementary RNA Substances 0.000 claims 2
- 230000006870 function Effects 0.000 claims 2
- 238000000185 intracerebroventricular administration Methods 0.000 claims 2
- 241001515942 marmosets Species 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 210000000278 spinal cord Anatomy 0.000 claims 2
- 101800000263 Acidic protein Proteins 0.000 claims 1
- 108091010877 Allograft inflammatory factor 1 Proteins 0.000 claims 1
- 102100040121 Allograft inflammatory factor 1 Human genes 0.000 claims 1
- 108700023155 Aspartoacylases Proteins 0.000 claims 1
- 108010035563 Chloramphenicol O-acetyltransferase Proteins 0.000 claims 1
- 102100039289 Glial fibrillary acidic protein Human genes 0.000 claims 1
- 101710193519 Glial fibrillary acidic protein Proteins 0.000 claims 1
- 108060001084 Luciferase Proteins 0.000 claims 1
- 239000005089 Luciferase Substances 0.000 claims 1
- 102000004317 Lyases Human genes 0.000 claims 1
- 108090000856 Lyases Proteins 0.000 claims 1
- 101710160107 Outer membrane protein A Proteins 0.000 claims 1
- 108091000117 Tyrosine 3-Monooxygenase Proteins 0.000 claims 1
- 102000048218 Tyrosine 3-monooxygenases Human genes 0.000 claims 1
- 102000006646 aminoglycoside phosphotransferase Human genes 0.000 claims 1
- 239000003708 ampul Substances 0.000 claims 1
- 102000018919 aspartoacylase Human genes 0.000 claims 1
- 210000000133 brain stem Anatomy 0.000 claims 1
- 208000015114 central nervous system disease Diseases 0.000 claims 1
- 210000001638 cerebellum Anatomy 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 108091006047 fluorescent proteins Proteins 0.000 claims 1
- 102000034287 fluorescent proteins Human genes 0.000 claims 1
- 210000005046 glial fibrillary acidic protein Anatomy 0.000 claims 1
- 210000001320 hippocampus Anatomy 0.000 claims 1
- 210000003016 hypothalamus Anatomy 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000007912 intraperitoneal administration Methods 0.000 claims 1
- 230000003907 kidney function Effects 0.000 claims 1
- 210000005230 lumbar spinal cord Anatomy 0.000 claims 1
- 210000004705 lumbosacral region Anatomy 0.000 claims 1
- 230000001537 neural effect Effects 0.000 claims 1
- 210000004129 prosencephalon Anatomy 0.000 claims 1
- 108010045647 puromycin N-acetyltransferase Proteins 0.000 claims 1
- 239000013608 rAAV vector Substances 0.000 claims 1
- 210000005084 renal tissue Anatomy 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- 210000001103 thalamus Anatomy 0.000 claims 1
- 210000000115 thoracic cavity Anatomy 0.000 claims 1
- 230000002861 ventricular Effects 0.000 claims 1
- 230000004304 visual acuity Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US32762710P | 2010-04-23 | 2010-04-23 | |
| US61/327,627 | 2010-04-23 | ||
| PCT/US2011/033616 WO2011133890A1 (en) | 2010-04-23 | 2011-04-22 | Cns targeting aav vectors and methods of use thereof |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016000310A Division JP6235619B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2016000309A Division JP6422897B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013531471A JP2013531471A (ja) | 2013-08-08 |
| JP2013531471A5 true JP2013531471A5 (enExample) | 2014-08-21 |
| JP5963743B2 JP5963743B2 (ja) | 2016-08-03 |
Family
ID=44834531
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013506330A Active JP5963743B2 (ja) | 2010-04-23 | 2011-04-22 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2016000310A Active JP6235619B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2016000309A Active JP6422897B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2018195573A Active JP6879988B2 (ja) | 2010-04-23 | 2018-10-17 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2019005016A Active JP6941632B2 (ja) | 2010-04-23 | 2019-01-16 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2021077161A Pending JP2021138702A (ja) | 2010-04-23 | 2021-04-30 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
Family Applications After (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016000310A Active JP6235619B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2016000309A Active JP6422897B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2018195573A Active JP6879988B2 (ja) | 2010-04-23 | 2018-10-17 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2019005016A Active JP6941632B2 (ja) | 2010-04-23 | 2019-01-16 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2021077161A Pending JP2021138702A (ja) | 2010-04-23 | 2021-04-30 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (8) | US9102949B2 (enExample) |
| EP (6) | EP3540055A1 (enExample) |
| JP (6) | JP5963743B2 (enExample) |
| CA (3) | CA3049237C (enExample) |
| DK (2) | DK2561073T3 (enExample) |
| ES (2) | ES2698203T3 (enExample) |
| WO (1) | WO2011133890A1 (enExample) |
Families Citing this family (227)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ618298A (en) | 2001-11-13 | 2015-04-24 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| EP2018421B1 (en) | 2006-04-28 | 2012-12-19 | The Trustees of the University of Pennsylvania | Scalable production method for aav |
| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| HRP20161150T1 (hr) | 2007-04-09 | 2016-11-18 | University Of Florida Research Foundation, Inc. | PRIPRAVCI rAAV VEKTORA KOJI IMAJU TIROZIN-MODIFICIRANE CAPSID PROTEINE I POSTUPCI ZA UPORABU |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| JP5963743B2 (ja) | 2010-04-23 | 2016-08-03 | ユニバーシティ オブ マサチューセッツ | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| CA2833912C (en) | 2010-04-23 | 2021-09-21 | University Of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| US9226976B2 (en) | 2011-04-21 | 2016-01-05 | University Of Massachusetts | RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
| WO2013006514A2 (en) * | 2011-07-01 | 2013-01-10 | University Of South Florida | Recombinant adeno-associated virus-mediated expression of fractalkine for treatment of neuroinflammatory and neurodegenerative diseases |
| RU2653444C2 (ru) * | 2012-02-17 | 2018-05-08 | Дзе Чилдрен'З Хоспитал Оф Филадельфия | Композиции вектора aav и способы переноса генов в клетки, органы и ткани |
| US10501513B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides for the production of oncology-related proteins and peptides |
| WO2013151663A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| US9878056B2 (en) | 2012-04-02 | 2018-01-30 | Modernatx, Inc. | Modified polynucleotides for the production of cosmetic proteins and peptides |
| US12358954B2 (en) | 2012-05-15 | 2025-07-15 | University Of Florida Research Foundation, Incorporated | Capsid-modified rAAV vector compositions and methods therefor |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| US9695443B2 (en) * | 2012-05-25 | 2017-07-04 | Commissariat A L'energie Atomique Et Aux Energies Alternatives | Vector for the selective silencing of a gene in astrocytes |
| CN104704123A (zh) * | 2012-06-21 | 2015-06-10 | 肌肉学研究协会 | 基因治疗载体的广泛的基因递送 |
| US20150252384A1 (en) * | 2012-08-01 | 2015-09-10 | National Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| WO2014043480A1 (en) | 2012-09-13 | 2014-03-20 | Crystal Ronald G | Treatment of brain cancers using central nervous system mediated gene transfer of monoclonal antibodies |
| JP2015529685A (ja) | 2012-09-17 | 2015-10-08 | ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルＴｈｅ Ｒｅｓｅａｒｃｈ Ｉｎｓｔｉｔｕｔｅ Ａｔｎａｔｉｏｎｗｉｄｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ | 筋萎縮性側索硬化症の処置のための組成物および方法 |
| PT2922554T (pt) | 2012-11-26 | 2022-06-28 | Modernatx Inc | Arn modificado nas porções terminais |
| HUE063051T2 (hu) | 2013-02-15 | 2023-12-28 | Bioverativ Therapeutics Inc | Optimalizált VIII. faktor gén |
| AU2014244167A1 (en) * | 2013-03-13 | 2015-10-08 | The Children's Hospital Of Philadelphia | Adeno-associated virus vectors and methods of use thereof |
| ES2744833T3 (es) | 2013-03-14 | 2020-02-26 | Univ Laval | Sistema de electrorretinografía (ERG) para la evaluación de trastornos psiquiátricos |
| MX386059B (es) | 2013-03-15 | 2025-03-18 | Univ Pennsylvania | COMPOSICIONES Y SUS usos EN EL TRATAMIENTO DE LA MUCOPOLISACARIDOSIS TIPO I. |
| 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 |
| SI3024498T1 (sl) | 2013-07-22 | 2020-07-31 | The Children's Hospital Of Philadelphia | Različica AAV in sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva |
| RU2664471C2 (ru) * | 2013-07-26 | 2018-08-17 | Юниверсити Оф Айова Рисерч Фаундейшн | Способы и композиции для лечения болезней мозга |
| EP4219727A3 (en) | 2013-08-27 | 2023-09-06 | Research Institute at Nationwide Children's Hospital | Products and methods for treatment of amyotrophic lateral sclerosis |
| EP3044318B1 (en) | 2013-09-13 | 2019-05-01 | California Institute of Technology | Selective recovery |
| WO2015060722A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| WO2015069647A1 (en) | 2013-11-05 | 2015-05-14 | The Research Institute At Nationwide Children's Hospital | COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS |
| EP3075849B1 (en) | 2013-11-29 | 2019-03-13 | Takara Bio Inc. | Method for quantifying adeno-associated virus |
| CA2933258C (en) * | 2013-12-11 | 2023-05-23 | Sumitomo Chemical Company, Limited | Method for manufacturing ciliary marginal zone-like structure |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| US10280418B2 (en) * | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
| EP3134115A4 (en) * | 2014-04-25 | 2017-10-25 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| PT3137497T (pt) | 2014-05-02 | 2021-07-12 | Genzyme Corp | Vetores de aav para terapia genética na retina e snc |
| US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2016004113A1 (en) | 2014-06-30 | 2016-01-07 | Biogen Ma Inc. | Optimized factor ix gene |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| JP6842410B2 (ja) | 2014-10-03 | 2021-03-17 | ユニバーシティ オブ マサチューセッツ | 新規の高効率ライブラリーにより同定されるａａｖベクター |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| WO2016073693A2 (en) * | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| CA2975583A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| CA2967393A1 (en) * | 2014-11-21 | 2016-05-26 | The University Of North Carolina At Chapel Hill | Aav vectors targeted to the central nervous system |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| JP6754361B2 (ja) | 2014-12-16 | 2020-09-09 | ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ | 若年型バッテン病のための遺伝子療法 |
| US20180030096A1 (en) | 2015-02-03 | 2018-02-01 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
| HK1247641A1 (zh) | 2015-02-10 | 2018-09-28 | Genzyme Corporation | 变体RNAi |
| EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions and methods for transient delivery of nucleases |
| AU2016229000B2 (en) * | 2015-03-10 | 2021-11-04 | The Trustees Of Columbia University In The City Of New York | Recombinant Glut1 adeno-associated viral vector constructs and related methods for restoring Glut1 expression |
| WO2016172008A1 (en) * | 2015-04-24 | 2016-10-27 | University Of Massachusetts | Modified aav constructions and uses thereof |
| US20170067028A1 (en) * | 2015-05-15 | 2017-03-09 | Douglas J. Ballon | Radiolabeling of adeno associated virus |
| IL322085A (en) * | 2015-05-29 | 2025-09-01 | Univ Pennsylvania | Compositions and methods for reducing unfolded proteins |
| IL309741A (en) | 2015-06-23 | 2024-02-01 | Childrens Hospital Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| EP3320101B1 (en) | 2015-07-07 | 2021-08-11 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject |
| US10047377B2 (en) * | 2015-09-22 | 2018-08-14 | Loyola University Of Chicago | Methods for modulating KLHL1 levels, methods for modulating current activity in T-type calcium channels, molecules therefor, and methods for identifying molecules therefor |
| US10597655B2 (en) * | 2015-09-22 | 2020-03-24 | Loyola University Chicago | Methods for modulating KLHL1 levels, methods for modulating current activity in T-type calcium channels, molecules therefor, and methods for identifying molecules therefor |
| CA2996420A1 (en) | 2015-09-28 | 2017-04-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| WO2017060510A1 (en) * | 2015-10-09 | 2017-04-13 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for the treatment of alzheimer's disease |
| EP3364996B1 (en) | 2015-10-22 | 2021-08-25 | University of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| DK3364997T5 (da) | 2015-10-22 | 2024-09-30 | Univ Massachusetts | Aspartoacylase genterapi til behandling af canavans sygdom |
| CN105420275A (zh) * | 2015-11-27 | 2016-03-23 | 中国科学院苏州生物医学工程技术研究所 | 制备外源功能基因定点整合的人神经干细胞的方法 |
| CA3006569A1 (en) | 2015-12-02 | 2017-06-08 | Voyager Therapeutics, Inc. | Assays for the detection of aav neutralizing antibodies |
| EP3387118B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| CA3007495C (en) | 2015-12-11 | 2023-04-11 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (aavs) |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| ES2926585T3 (es) | 2016-02-01 | 2022-10-27 | Bioverativ Therapeutics Inc | Genes de Factor VIII optimizados |
| EP3411059A4 (en) | 2016-02-02 | 2019-10-16 | University Of Massachusetts | METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM |
| IL260959B2 (en) | 2016-02-03 | 2024-09-01 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| US11060088B2 (en) | 2016-02-12 | 2021-07-13 | University Of Massachusetts | Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization |
| EP3436078B1 (en) * | 2016-03-31 | 2021-10-27 | University of Cincinnati | Methods and compositions for the treatment of als |
| EP3440210A4 (en) | 2016-04-05 | 2019-11-27 | University of Massachusetts | COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN |
| US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| SG11201808176TA (en) | 2016-04-15 | 2018-10-30 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| GB201608046D0 (en) | 2016-05-09 | 2016-06-22 | Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The | Treatment of complement-mediated disorders |
| RU2725286C2 (ru) | 2016-05-13 | 2020-06-30 | 4Д Молекьюлар Терапьютикс Инк. | Варианты капсидов аденоассоциированного вируса и способы их применения |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| IL262784B2 (en) | 2016-05-18 | 2023-10-01 | Voyager Therapeutics Inc | modulatory polynucleotides |
| US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| SG11201900543SA (en) * | 2016-07-26 | 2019-02-27 | Univ Cornell | Gene therapy for the treatment of aldehyde dehydrogenase deficiency |
| NZ791267A (en) | 2016-08-15 | 2025-11-28 | Genzyme Corp | Methods for detecting AAV |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| MX392374B (es) | 2016-09-28 | 2025-03-24 | Cohbar Inc | Peptidos terapeuticos relacionados con mots-c. |
| TWI773695B (zh) | 2016-10-12 | 2022-08-11 | 美商生物維瑞提夫美國公司 | 抗Ｃ１ｓ抗體及其使用方法 |
| AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
| BR112019009446A2 (pt) | 2016-11-09 | 2019-07-30 | Intrexon Corp | construtos de expressão da frataxina |
| WO2018156892A1 (en) | 2017-02-23 | 2018-08-30 | Adrx, Inc. | Peptide inhibitors of transcription factor aggregation |
| MX2019011772A (es) | 2017-04-03 | 2020-01-09 | Encoded Therapeutics Inc | Expresion transgenica selectiva de tejidos. |
| US10898585B2 (en) | 2017-04-14 | 2021-01-26 | Ptc Therapeutics .Inc. | Gene therapy for AADC deficiency |
| MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
| WO2018204786A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | Methods of treating amyotrophic lateral sclerosis (als) |
| CA3066077A1 (en) | 2017-06-07 | 2018-12-13 | Adrx, Inc. | Tau aggregation inhibitors |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| BR112020000063A2 (pt) | 2017-07-06 | 2020-07-14 | The Trustees Of The University Of Pennsylvania | terapia gênica mediada por aav9 para tratamento de mucopolissacaridose tipo i |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| AU2018311069A1 (en) | 2017-08-03 | 2020-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of AAV |
| KR20250011726A (ko) | 2017-09-20 | 2025-01-21 | 4디 몰레큘러 테라퓨틱스 아이엔씨. | 아데노-관련 바이러스 변이체 캡시드 및 그 용도 |
| AU2018335752B2 (en) | 2017-09-22 | 2025-04-10 | Christian HINDERER | Gene therapy for treating Mucopolysaccharidosis type ii |
| EP3684932A1 (en) | 2017-09-22 | 2020-07-29 | Genzyme Corporation | Variant rnai |
| EP3684937A4 (en) | 2017-09-22 | 2021-06-02 | University of Massachusetts | NEW DUAL SOD1 EXPRESSION VECTORS AND RELATED USES |
| MX2020003042A (es) * | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| MA49923A (fr) * | 2017-10-02 | 2020-06-24 | Res Inst Nationwide Childrens Hospital | Système de déciblage de miarn pour interférence spécifique d'un tissu |
| JP7361037B2 (ja) | 2017-10-03 | 2023-10-13 | プリベイル セラピューティクス，インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| AU2018346102B2 (en) * | 2017-10-03 | 2023-05-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| JP7369121B2 (ja) | 2017-10-11 | 2023-10-25 | バイオベラティブ・ユーエスエイ・インコーポレイテッド | 補体活性を誘発する方法 |
| WO2019075320A1 (en) * | 2017-10-12 | 2019-04-18 | The Trustees Of Columbia University In The City Of New York | INCARN OF SLC2A1 AS BIOLOGICAL SUBSTANCE, TREATMENTS AND ASSOCIATED METHODS |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| CR20200282A (es) | 2017-11-27 | 2021-02-18 | 4D Molecular Therapeutics Inc | Cápsides variantes de virus adenoasociados y su uso para inhibir la angiogénesis |
| JP7384797B2 (ja) | 2017-11-30 | 2023-11-21 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ムコ多糖症ｉｉｉｂ型のための遺伝子療法 |
| WO2019108857A1 (en) | 2017-11-30 | 2019-06-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis iiia |
| CN118345115A (zh) | 2017-12-01 | 2024-07-16 | 编码治疗公司 | 工程化的dna结合蛋白 |
| US12194108B2 (en) | 2018-03-23 | 2025-01-14 | University Of Massachusetts | Gene therapeutics for treating bone disorders |
| CN112272672A (zh) | 2018-04-03 | 2021-01-26 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| SG11202009451VA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Antibody-evading virus vectors |
| US12054724B2 (en) | 2018-04-10 | 2024-08-06 | President And Fellows Of Harvard College | AAV vectors encoding clarin-1 or GJB2 and uses thereof |
| CN112601454B (zh) | 2018-04-16 | 2023-10-27 | 宾夕法尼亚州大学信托人 | 用于治疗杜兴肌营养不良的组合物和方法 |
| WO2019210137A1 (en) | 2018-04-27 | 2019-10-31 | Voyager Therapeutics, Inc. | Methods for measuring the potency of aadc viral vectors |
| WO2019222329A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| WO2019236995A1 (en) | 2018-06-08 | 2019-12-12 | University Of Massachusetts | Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells |
| EP3807404A1 (en) | 2018-06-13 | 2021-04-21 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| US12297241B2 (en) | 2018-07-06 | 2025-05-13 | The Regents Of The University Of California | Method to engineer transplantable human tissues |
| JP2021530218A (ja) * | 2018-07-11 | 2021-11-11 | ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドThe Brigham and Women’s Hospital, Inc. | 血液脳関門を超える薬剤の送達のための方法および組成物 |
| JP2021530548A (ja) | 2018-07-24 | 2021-11-11 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. | 遺伝子治療製剤を生産するための系および方法 |
| US20210301306A1 (en) * | 2018-07-31 | 2021-09-30 | Cornell University | Gene therapy methods to control organ function |
| WO2020028751A2 (en) | 2018-08-03 | 2020-02-06 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
| AR114540A1 (es) | 2018-08-03 | 2020-09-16 | Genzyme Corp | VARIANTE DE ARNi CONTRA a-SINUCLEÍNA |
| EP3844285A4 (en) * | 2018-08-30 | 2022-06-29 | Rowan University | Methods of treating or preventing amyotrophic lateral sclerosis |
| JP7624724B2 (ja) | 2018-09-26 | 2025-01-31 | カリフォルニア インスティテュート オブ テクノロジー | 標的遺伝子療法のためのアデノ随伴ウイルス組成物 |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CN119684417A (zh) | 2018-10-02 | 2025-03-25 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| TW202035689A (zh) | 2018-10-04 | 2020-10-01 | 美商航海家醫療公司 | 測量病毒載體粒子的效價及強度之方法 |
| CN113166731A (zh) | 2018-10-05 | 2021-07-23 | 沃雅戈治疗公司 | 编码aav生产蛋白的工程化核酸构建体 |
| US20210371470A1 (en) | 2018-10-12 | 2021-12-02 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| EP3867389A1 (en) | 2018-10-15 | 2021-08-25 | Voyager Therapeutics, Inc. | Expression vectors for large-scale production of raav in the baculovirus/sf9 system |
| EP3890786A4 (en) | 2018-12-05 | 2022-08-31 | Abeona Therapeutics Inc. | RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR FOR GENE RELEASE |
| WO2020132455A1 (en) * | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| EP3917944A1 (en) | 2019-01-28 | 2021-12-08 | Cohbar Inc. | Therapeutic peptides |
| JP2022521025A (ja) | 2019-02-25 | 2022-04-04 | ノバルティス アーゲー | Ｂｉｅｔｔｉクリスタリン網膜症を治療するための組成物及び方法 |
| US20220154211A1 (en) | 2019-02-25 | 2022-05-19 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
| US12491264B2 (en) | 2019-03-04 | 2025-12-09 | Duke University | Compositions and methods for the diagnosis and treatment of retinopathies |
| SG11202110146VA (en) | 2019-03-21 | 2021-10-28 | Stridebio Inc | Recombinant adeno-associated virus vectors |
| MX2021012184A (es) | 2019-04-10 | 2022-01-24 | Prevail Therapeutics Inc | Terapias genicas para trastornos lisosomales. |
| EP3966231A4 (en) * | 2019-04-10 | 2022-12-21 | University of Massachusetts | Factor h vectors and uses thereof |
| WO2020210592A1 (en) * | 2019-04-12 | 2020-10-15 | University Of Massachusetts | Recombinant aav gene therapy for ngyl1 deficiency |
| JP7644026B2 (ja) * | 2019-04-29 | 2025-03-11 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 新規ａａｖカプシドおよびそれを含む組成物 |
| WO2020223274A1 (en) | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS |
| WO2020223280A1 (en) | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
| EP3966227A1 (en) | 2019-05-07 | 2022-03-16 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| US20220226501A1 (en) * | 2019-05-31 | 2022-07-21 | Universitat Autònoma De Barcelona | Insulin gene therapy |
| WO2020257194A1 (en) * | 2019-06-17 | 2020-12-24 | Alnylam Pharmaceuticals, Inc. | Delivery of oligonucleotides to the striatum |
| US11446084B2 (en) | 2019-07-12 | 2022-09-20 | Neuralink Corp. | Laser drilling of pia mater |
| WO2021025995A1 (en) | 2019-08-02 | 2021-02-11 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
| US20220290182A1 (en) | 2019-08-09 | 2022-09-15 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| GB2587319A (en) * | 2019-08-12 | 2021-03-31 | Healing Genes Llc | Genetic construct |
| TW202122582A (zh) | 2019-08-26 | 2021-06-16 | 美商航海家醫療公司 | 病毒蛋白之控制表現 |
| CA3151087A1 (en) * | 2019-08-29 | 2021-03-04 | The Board Of Trustees Of The Leland Stanford Junior University | Adeno-associated viral vectors for crossing the human blood brain barrier |
| WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| TW202126284A (zh) | 2019-09-30 | 2021-07-16 | 美商百歐維拉提夫治療公司 | 慢病毒載體配製物 |
| EP4045525A1 (en) * | 2019-10-17 | 2022-08-24 | Stridebio, Inc. | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
| WO2021126991A1 (en) * | 2019-12-17 | 2021-06-24 | Nikegen Limited | Parvoviral vectors and methods of making and use thereof |
| IL294608A (en) * | 2020-01-10 | 2022-09-01 | Brigham & Womens Hospital Inc | Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer |
| KR20220133941A (ko) | 2020-01-29 | 2022-10-05 | 젠자임 코포레이션 | 안과 유전자 치료를 위한 변형 아데노-관련 바이러스 캡시드 단백질 및 이의 사용 방법 |
| WO2021177116A1 (ja) | 2020-03-02 | 2021-09-10 | 国立大学法人群馬大学 | ミクログリア選択的遺伝子発現ベクター |
| IL296708A (en) | 2020-03-26 | 2022-11-01 | Diamentis Inc | Systems and methods for processing retinal signal data and identifying conditions |
| KR20230020394A (ko) | 2020-04-15 | 2023-02-10 | 보이저 테라퓨틱스, 인크. | Tau 결합 화합물 |
| GB202005732D0 (en) * | 2020-04-20 | 2020-06-03 | Synpromics Ltd | Regulatory nucleic acid sequences |
| CA3174070A1 (en) * | 2020-04-28 | 2021-11-04 | Pfizer Inc. | Modified nucleic acids encoding aspartoacylase (aspa) and vectors for gene therapy |
| JP2023526310A (ja) | 2020-05-12 | 2023-06-21 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 導入遺伝子発現のｄｒｇ特異的低減のための組成物 |
| IL298001A (en) | 2020-05-13 | 2023-01-01 | Voyager Therapeutics Inc | Redirecting tropism of adeno-associated virus capsids |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| BR112022026127A2 (pt) | 2020-06-24 | 2023-01-17 | Bioverativ Therapeutics Inc | Métodos para remoção de fator viii livre de preparações de vetores lentivirais modificados para expressar referida proteína |
| GB202010981D0 (en) | 2020-07-16 | 2020-09-02 | Ucl Business Ltd | Gene therapy for neuromuscular and neuromotor disorders |
| CA3190601A1 (en) * | 2020-08-05 | 2022-02-10 | Hangzhou Exegenesis Bio Ltd. | Nucleic acid constructs and uses thereof for treating spinal muscular atrophy |
| EP4192514A1 (en) | 2020-08-06 | 2023-06-14 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| JP2023547681A (ja) * | 2020-11-04 | 2023-11-13 | ベーリンガー インゲルハイム インターナショナル ゲゼルシャフト ミット ベシュレンクテル ハフツング | Ｉｌｄ、ｐｆ－ｉｌｄおよびｉｐｆ治療用ウイルスベクターおよび核酸 |
| US20240050593A1 (en) * | 2020-12-18 | 2024-02-15 | Duke University | Compositions For and Methods of Enhancing Spinal Cord Tissue Regeneration |
| US20240141378A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| EP4301768A2 (en) | 2021-03-03 | 2024-01-10 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| US20240158811A1 (en) * | 2021-03-11 | 2024-05-16 | Temple University-Of The Commonwealth System Of Higher Education | Noninvasive AAV Vectors For Highly Efficient Gene Delivery To The Nervous System |
| US20240252679A1 (en) | 2021-05-28 | 2024-08-01 | Shanghai Regenelead Therapies Co., Ltd. | Recombinant adeno-associated virus having variant capsid, and application thereof |
| US20250001008A1 (en) * | 2021-07-01 | 2025-01-02 | Bridgebio Gene Therapy Research, Inc. | Gene therapy for galactosemia |
| EP4405396A2 (en) | 2021-09-20 | 2024-07-31 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| TW202333776A (zh) | 2021-10-12 | 2023-09-01 | 美商阿斯帕醫療公司 | 用於治療腦白質失養症的方法及組合物 |
| WO2023081648A1 (en) | 2021-11-02 | 2023-05-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| JP2024543918A (ja) | 2021-11-29 | 2024-11-26 | 上海瑞宏迪医薬有限公司 | Ａａｄｃ、ｇｄｎｆポリヌクレオチド及びそのパーキンソン病を治療するための使用 |
| WO2023147374A2 (en) | 2022-01-25 | 2023-08-03 | Voyager Therapeutics, Inc. | Baculovirus expression system |
| CA3244192A1 (en) | 2022-02-21 | 2025-01-22 | Shanghai Regenelead Therapies Co Ltd | VEGF-BINDING MOLECULE AND ITS PHARMACEUTICAL USE |
| IL316222A (en) | 2022-04-12 | 2024-12-01 | Genzyme Corp | Using the iRAC4 modulator for gene therapy |
| TW202405430A (zh) | 2022-04-12 | 2024-02-01 | 美商健臻公司 | 對於基因療法藥劑的先天免疫原性之樹突細胞試驗 |
| CA3248481A1 (en) | 2022-04-12 | 2023-10-19 | Genzyme Corp | USE OF INTERLEUKIN 4 RECEPTOR-ASSOCIATED KINASE MODULATORS FOR GENE THERAPY |
| WO2023205397A1 (en) * | 2022-04-22 | 2023-10-26 | The Children's Hospital Of Philadelphia | Human ependyma-specific promoter and uses thereof |
| US20250295809A1 (en) | 2022-05-13 | 2025-09-25 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
| US20250243263A1 (en) | 2022-06-22 | 2025-07-31 | Voyager Therapeutics, Inc. | Tau binding compounds |
| WO2024027632A1 (en) * | 2022-08-01 | 2024-02-08 | Skyline Therapeutics (Shanghai) Co., Ltd. | Novel plasmid backbone to reduce dna impurities in raav preparation |
| WO2024054983A1 (en) | 2022-09-08 | 2024-03-14 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| EP4587470A1 (en) | 2022-09-15 | 2025-07-23 | Voyager Therapeutics, Inc. | Tau binding compounds |
| GB2622629A (en) | 2022-09-23 | 2024-03-27 | Sania Rx Ltd | Method |
| WO2025128817A1 (en) | 2023-12-15 | 2025-06-19 | Genzyme Corporation | Artificial micrornas targeting tau |
| WO2025158385A1 (en) | 2024-01-25 | 2025-07-31 | Genzyme Corporation | Pegylated il-2 for suppressing adaptive immune response to gene therapy |
| WO2025160429A1 (en) | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting snca |
| WO2025160434A1 (en) | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting huntington's disease |
| GB202406331D0 (en) | 2024-05-07 | 2024-06-19 | Sania Rx Ltd | Capsid |
| WO2025235643A1 (en) | 2024-05-08 | 2025-11-13 | Genzyme Corporation | Profiling of gene therapy agents |
Family Cites Families (141)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5043270A (en) | 1989-03-31 | 1991-08-27 | The Board Of Trustees Of The Leland Stanford Junior University | Intronic overexpression vectors |
| US5549910A (en) | 1989-03-31 | 1996-08-27 | The Regents Of The University Of California | Preparation of liposome and lipid complex compositions |
| US5252334A (en) | 1989-09-08 | 1993-10-12 | Cygnus Therapeutic Systems | Solid matrix system for transdermal drug delivery |
| JP3218637B2 (ja) | 1990-07-26 | 2001-10-15 | 大正製薬株式会社 | 安定なリポソーム水懸濁液 |
| JP2958076B2 (ja) | 1990-08-27 | 1999-10-06 | 株式会社ビタミン研究所 | 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法 |
| US5399363A (en) | 1991-01-25 | 1995-03-21 | Eastman Kodak Company | Surface modified anticancer nanoparticles |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| ATE386131T1 (de) | 1994-04-13 | 2008-03-15 | Univ Rockefeller | Aav-vermittelte überbringung von dna in zellen des nervensystems |
| US5741516A (en) | 1994-06-20 | 1998-04-21 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
| US5697899A (en) | 1995-02-07 | 1997-12-16 | Gensia | Feedback controlled drug delivery system |
| IE80468B1 (en) | 1995-04-04 | 1998-07-29 | Elan Corp Plc | Controlled release biodegradable nanoparticles containing insulin |
| US5738868A (en) | 1995-07-18 | 1998-04-14 | Lipogenics Ltd. | Liposome compositions and kits therefor |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| US5656016A (en) | 1996-03-18 | 1997-08-12 | Abbott Laboratories | Sonophoretic drug delivery system |
| US6391607B1 (en) | 1996-06-14 | 2002-05-21 | Genentech, Inc. | Human DNase I hyperactive variants |
| US5797898A (en) | 1996-07-02 | 1998-08-25 | Massachusetts Institute Of Technology | Microchip drug delivery devices |
| US5783208A (en) | 1996-07-19 | 1998-07-21 | Theratech, Inc. | Transdermal drug delivery matrix for coadministering estradiol and another steroid |
| US5779708A (en) | 1996-08-15 | 1998-07-14 | Cyberdent, Inc. | Intraosseous drug delivery device and method |
| CA2264482A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6083716A (en) | 1996-09-06 | 2000-07-04 | The Trustees Of The University Of Pennsylvania | Chimpanzee adenovirus vectors |
| US6177403B1 (en) | 1996-10-21 | 2001-01-23 | The Trustees Of The University Of Pennsylvania | Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal |
| US6544523B1 (en) | 1996-11-13 | 2003-04-08 | Chiron Corporation | Mutant forms of Fas ligand and uses thereof |
| US6475469B1 (en) | 1996-11-21 | 2002-11-05 | Applied Dental Sciences, Inc. | Compositions for removing tooth stains |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6251677B1 (en) | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| AU9397098A (en) | 1997-09-19 | 1999-04-12 | Trustees Of The University Of Pennsylvania, The | Methods and cell line useful for production of recombinant adeno-associated viruses |
| US6346415B1 (en) | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| US6953690B1 (en) | 1998-03-20 | 2005-10-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| AU762220B2 (en) | 1998-05-28 | 2003-06-19 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | AAV5 vector and uses thereof |
| WO2000005415A1 (en) | 1998-07-22 | 2000-02-03 | Immusol Incorporated | Substantially complete ribozyme libraries |
| US20030110526A1 (en) | 1998-08-25 | 2003-06-12 | Robert H. Brown | Dysferlin mutations |
| JP4693244B2 (ja) | 1999-03-18 | 2011-06-01 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法 |
| US6498244B1 (en) | 1999-05-28 | 2002-12-24 | Cell Genesys, Inc. | Adeno-associated virus capsid immunologic determinants |
| US6365394B1 (en) | 1999-09-29 | 2002-04-02 | The Trustees Of The University Of Pennsylvania | Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus |
| WO2001029243A1 (en) | 1999-10-15 | 2001-04-26 | Dalhousie University | Method and vector for producing and transferring trans-spliced peptides |
| US6821512B1 (en) | 1999-12-03 | 2004-11-23 | The Trustees Of The University Of Pennsylvania | Compositions and methods for increasing packaging and yield of recombinant adenoviruses using multiple packaging signals |
| US7638120B2 (en) | 2000-03-14 | 2009-12-29 | Thomas Jefferson University | High transgene expression of a pseudotyped adeno-associated virus type |
| US7749492B2 (en) | 2001-01-05 | 2010-07-06 | Nationwide Children's Hospital, Inc. | AAV vectors and methods |
| US6962815B2 (en) | 2001-01-05 | 2005-11-08 | Children's Hopital Inc. | AAV2 vectors and methods |
| US20040136963A1 (en) | 2001-06-22 | 2004-07-15 | The Trustees Of The University Of Pennsylvania | Simian adenovirus vectors and methods of use |
| CA2450470C (en) | 2001-06-22 | 2012-08-28 | The Trustees Of The University Of Pennsylvania | Method for rapid screening of bacterial transformants and novel simian adenovirus proteins |
| WO2003004660A1 (en) * | 2001-07-03 | 2003-01-16 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
| EP1419245A4 (en) | 2001-07-13 | 2006-04-05 | Univ Iowa Res Found | ADENO-ASSOCIATED PSEUDOTYPE VIRUSES AND USES THEREOF |
| US20030092161A1 (en) | 2001-09-19 | 2003-05-15 | The Trustees Of The University Of Pennsylvania | Compositions and methods for production of recombinant viruses, and uses therefor |
| NZ618298A (en) | 2001-11-13 | 2015-04-24 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| BR0214350A (pt) | 2001-11-21 | 2005-05-10 | Univ Pennsylvania | Sequências de ácido nucleico e aminoácido de adenovìrus de sìmio, vetores contendo as mesmas e métodos de uso |
| JP4810062B2 (ja) | 2001-12-17 | 2011-11-09 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス（ａａｖ）血清型８の配列 |
| PT2573170T (pt) | 2001-12-17 | 2018-03-26 | Univ Pennsylvania | Sequências de um vírus adenoassociado (aav) de serotipo 9, vetor contendo as mesmas, e suas utilizações |
| US20050137153A1 (en) | 2002-02-20 | 2005-06-23 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA) |
| US20060292117A1 (en) | 2002-04-17 | 2006-12-28 | Loiler Scott A | Improved rAAv vectors |
| WO2003089011A1 (en) | 2002-04-19 | 2003-10-30 | University Of Florida | rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES |
| ES2280694T3 (es) | 2002-04-29 | 2007-09-16 | The Trustees Of The University Of Pennsylvania | Metodo para la extraccion directa y la amplificacion de virus integrados del adn celular de tejidos. |
| US20060093589A1 (en) | 2004-02-19 | 2006-05-04 | Warrington Kenneth H | Vp2-modified raav vector compositions and uses therefor |
| US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US20050255086A1 (en) * | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
| JP2006518372A (ja) | 2003-01-28 | 2006-08-10 | セレクティス | 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用 |
| US7387896B2 (en) | 2003-03-26 | 2008-06-17 | The Regents Of The University Of Michigan | MicroRNA vectors |
| US20060228800A1 (en) | 2003-05-15 | 2006-10-12 | Shi-Lung Lin | Novel Transgenic Methods Using intronic RNA |
| EP1633767B1 (en) | 2003-06-02 | 2018-11-21 | University of Massachusetts | Methods and compositions for controlling efficacy of rna silencing |
| HUE035567T2 (en) | 2003-09-30 | 2018-05-28 | Univ Pennsylvania | Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them |
| NZ550284A (en) | 2004-03-05 | 2009-04-30 | Benitec Ltd | Multiple promoter expression cassettes for simultaneous delivery of RNAi agents |
| WO2005096781A2 (en) * | 2004-04-06 | 2005-10-20 | University Of Massachusetts | Methods and compositions for treating gain-of-function disorders using rna interference |
| WO2005116224A2 (en) * | 2004-05-18 | 2005-12-08 | Children's Memorial Hospital | Tetracycline-regulated adeno-associated viral (aav) vectors for gene delivery to the nervous system |
| US7427396B2 (en) | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
| US20060189564A1 (en) * | 2004-10-22 | 2006-08-24 | Medtronic, Inc. | Methods and sequences to suppress pro-inflamatory cytokine actions locally to treat pain |
| NZ555830A (en) | 2004-12-15 | 2009-01-31 | Univ North Carolina | Chimeric vectors |
| AU2006210443B2 (en) | 2005-02-03 | 2011-01-27 | Benitec, Inc. | RNAi expression constructs |
| EP1866422B1 (en) * | 2005-04-07 | 2016-04-06 | The Trustees of The University of Pennsylvania | Method of increasing the function of an aav vector |
| WO2006119137A1 (en) * | 2005-04-29 | 2006-11-09 | The University Of North Carolina At Chapel Hill | Methods and compositions for regulated expression of nucleic acid at post-transcriptional level |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| PL2002003T3 (pl) * | 2005-05-27 | 2016-06-30 | Ospedale San Raffaele Srl | Wektor genetyczny zawierający mi-RNA |
| US20070213292A1 (en) | 2005-08-10 | 2007-09-13 | The Rockefeller University | Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof |
| US7977314B2 (en) * | 2005-12-02 | 2011-07-12 | Amorfix Life Sciences Limited | Methods and compositions to treat and detect misfolded-SOD1 mediated diseases |
| EP1979485A2 (en) | 2006-01-31 | 2008-10-15 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
| SI1986661T1 (sl) * | 2006-02-08 | 2018-12-31 | Genzyme Corporation | Genska terapija za Niemann-Pickovo bolezen tipa A |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| EP2018421B1 (en) | 2006-04-28 | 2012-12-19 | The Trustees of the University of Pennsylvania | Scalable production method for aav |
| ES2434169T3 (es) | 2006-11-08 | 2013-12-13 | Veritas Bio, Llc | Entrega in vivo de ARN bicatenario a una célula objetivo |
| AU2008216147A1 (en) | 2007-02-15 | 2008-08-21 | National Jewish Health | Methods and compositions for the disruption of biofilms |
| HRP20161150T1 (hr) | 2007-04-09 | 2016-11-18 | University Of Florida Research Foundation, Inc. | PRIPRAVCI rAAV VEKTORA KOJI IMAJU TIROZIN-MODIFICIRANE CAPSID PROTEINE I POSTUPCI ZA UPORABU |
| CA2683469C (en) | 2007-04-12 | 2019-11-26 | The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
| WO2008148068A1 (en) | 2007-05-23 | 2008-12-04 | Mannkind Corporation | Multicistronic vectors and methods for their design |
| AU2008256886B2 (en) | 2007-05-23 | 2013-02-28 | Dharmacon, Inc. | Micro-RNA scaffolds and non-naturally occurring micro-RNAs |
| EP2530152B1 (en) | 2007-05-31 | 2017-12-27 | University of Iowa Research Foundation | Reduction of off-target RNA interference toxicity |
| EP3252161B1 (en) | 2007-06-06 | 2021-11-17 | Genzyme Corporation | Gene therapy for lysosomal storage diseases |
| EP2019143A1 (en) * | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| US9090894B2 (en) | 2007-12-17 | 2015-07-28 | California Institute Of Technology | Modulating immune system development and function through microRNA MIR-146 |
| WO2009108274A2 (en) * | 2008-02-26 | 2009-09-03 | The University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| WO2009146178A1 (en) * | 2008-04-15 | 2009-12-03 | President And Fellows Of Harvard College | Angiogenin and amyotrophic lateral sclerosis |
| JP5797551B2 (ja) | 2008-04-22 | 2015-10-21 | フエー・イー・ベー・フエー・ゼツト・ウエー | 肝特異的核酸調節要素ならびにその方法および用途 |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| US8222221B2 (en) | 2008-06-04 | 2012-07-17 | The Board Of Regents Of The University Of Texas System | Modulation of gene expression through endogenous small RNA targeting of gene promoters |
| US20110171262A1 (en) | 2008-06-17 | 2011-07-14 | Andrew Christian Bakker | Parvoviral capsid with incorporated gly-ala repeat region |
| EP2310507A4 (en) | 2008-07-08 | 2013-03-20 | David Gladstone Inst | METHOD AND COMPOSITIONS FOR ANGIOGENIC MODULATION |
| WO2010034314A1 (en) | 2008-09-24 | 2010-04-01 | Aarhus Universitet | Retroviral delivery of synthectic gene cassettes |
| WO2010071454A1 (en) * | 2008-12-17 | 2010-06-24 | Auckland Uniservices Limited | Adeno-associated viral vectors and uses thereof |
| WO2010099383A2 (en) | 2009-02-26 | 2010-09-02 | The Johns Hopkins University | Compositions and methods for ex vivo hepatic nucleic acid delivery |
| DK3421603T3 (da) * | 2009-05-02 | 2022-01-10 | Genzyme Corp | Genterapi for neurodegenerative forstyrrelser |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US20120270930A1 (en) | 2009-10-29 | 2012-10-25 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Methods and compositions for dysferlin exon-skipping |
| MX2012005450A (es) | 2009-11-09 | 2012-08-15 | Genepod Therapeutics Ab | Constructo de vector virico novedoso para sintesis dopa continua optimizada especificamente de neuronas in vivo. |
| CA2781332C (en) | 2009-11-19 | 2018-09-04 | National University Corporation Okayama University | System for increasing gene expression and vector comprising the system |
| WO2011088081A1 (en) | 2010-01-12 | 2011-07-21 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| CN102947453A (zh) | 2010-01-28 | 2013-02-27 | 费城儿童医院 | 用于病毒载体纯化的可扩缩生产平台和用于基因治疗中的如此纯化的病毒载体 |
| AU2011223820B2 (en) | 2010-03-01 | 2016-01-14 | The Children's Hospital Of Philadelphia | Nucleic acids for targeting multiple regions of the HCV genome |
| JP5963743B2 (ja) | 2010-04-23 | 2016-08-03 | ユニバーシティ オブ マサチューセッツ | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| CA2833912C (en) | 2010-04-23 | 2021-09-21 | University Of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| US8785413B2 (en) | 2010-09-27 | 2014-07-22 | Florida Atlantic University Research Corporation | Materials and methods for the treatment of pathological neovascularization in the eye |
| ES2739804T3 (es) | 2011-02-12 | 2020-02-04 | Univ Iowa Res Found | Compuestos terapéuticos |
| SG10201601110VA (en) | 2011-02-17 | 2016-03-30 | Univ Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| EP2699258A4 (en) | 2011-04-15 | 2014-10-29 | Us Sec Dep Of Health And Human Services | AAV-mediated CTLA-4 gene transfer for the treatment of SJÖGREN SYNDROME |
| US9226976B2 (en) | 2011-04-21 | 2016-01-05 | University Of Massachusetts | RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| US8609088B2 (en) | 2011-05-10 | 2013-12-17 | Regents Of The University Of Minnesota | Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases |
| KR20140074997A (ko) | 2011-10-11 | 2014-06-18 | 더 브리검 앤드 우먼즈 하스피털, 인크. | 신경병성 질환에서 마이크로rna |
| US20130142861A1 (en) | 2011-12-05 | 2013-06-06 | National Yang Ming University | Compositions And Method For Detecting And Treating Abnormal Liver Homeostasis And Hepatocarcinogenesis |
| RU2653444C2 (ru) | 2012-02-17 | 2018-05-08 | Дзе Чилдрен'З Хоспитал Оф Филадельфия | Композиции вектора aav и способы переноса генов в клетки, органы и ткани |
| WO2013170078A1 (en) | 2012-05-09 | 2013-11-14 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| CN104704123A (zh) | 2012-06-21 | 2015-06-10 | 肌肉学研究协会 | 基因治疗载体的广泛的基因递送 |
| EP2943060A4 (en) | 2013-01-14 | 2016-11-09 | Recombinetics Inc | LIVESTOCK WITHOUT HORN |
| AU2014244167A1 (en) | 2013-03-13 | 2015-10-08 | The Children's Hospital Of Philadelphia | Adeno-associated virus vectors and methods of use thereof |
| WO2014144229A1 (en) | 2013-03-15 | 2014-09-18 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding aav vectors |
| DK2986635T3 (en) | 2013-04-18 | 2019-01-28 | Fond Telethon | EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS |
| SG11201510284XA (en) | 2013-06-17 | 2016-01-28 | Broad Inst Inc | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
| US10155794B2 (en) | 2013-07-16 | 2018-12-18 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of disorders related to CEP290 |
| CA2919995A1 (en) | 2013-08-05 | 2015-02-12 | Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum | Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp |
| EP4219727A3 (en) | 2013-08-27 | 2023-09-06 | Research Institute at Nationwide Children's Hospital | Products and methods for treatment of amyotrophic lateral sclerosis |
| GB201401707D0 (en) | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| WO2015150922A2 (en) | 2014-03-17 | 2015-10-08 | The Hospital For Sick Children | β-HEXOSAMINIDASE PROTEIN VARIANTS AND ASSOCIATED METHODS FOR TREATING GM2 GANGLIOSIDOSES |
| US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| PT3137497T (pt) | 2014-05-02 | 2021-07-12 | Genzyme Corp | Vetores de aav para terapia genética na retina e snc |
| ES2759317T3 (es) | 2014-05-20 | 2020-05-08 | Univ Iowa Res Found | Compuestos terapéuticos para la enfermedad de Huntington |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| US20190000940A1 (en) | 2015-07-31 | 2019-01-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of aadc deficiency |
-
2011
- 2011-04-22 JP JP2013506330A patent/JP5963743B2/ja active Active
- 2011-04-22 WO PCT/US2011/033616 patent/WO2011133890A1/en not_active Ceased
- 2011-04-22 EP EP19166281.6A patent/EP3540055A1/en active Pending
- 2011-04-22 CA CA3049237A patent/CA3049237C/en active Active
- 2011-04-22 CA CA3066596A patent/CA3066596A1/en active Pending
- 2011-04-22 DK DK11772784.2T patent/DK2561073T3/en active
- 2011-04-22 EP EP18190083.8A patent/EP3536781A1/en active Pending
- 2011-04-22 EP EP11772784.2A patent/EP2561073B1/en active Active
- 2011-04-22 US US13/642,719 patent/US9102949B2/en active Active
- 2011-04-22 DK DK14181861.7T patent/DK2826860T3/en active
- 2011-04-22 ES ES14181861T patent/ES2698203T3/es active Active
- 2011-04-22 ES ES11772784.2T patent/ES2605305T3/es active Active
- 2011-04-22 CA CA2833908A patent/CA2833908C/en active Active
- 2011-04-22 EP EP14181861.7A patent/EP2826860B1/en active Active
- 2011-04-22 EP EP18248259.6A patent/EP3514232A1/en not_active Withdrawn
- 2011-04-22 EP EP18248250.5A patent/EP3567106A1/en not_active Withdrawn
-
2014
- 2014-07-29 US US14/445,670 patent/US9701984B2/en active Active
-
2016
- 2016-01-04 JP JP2016000310A patent/JP6235619B2/ja active Active
- 2016-01-04 JP JP2016000309A patent/JP6422897B2/ja active Active
-
2017
- 2017-06-05 US US15/613,646 patent/US10731178B2/en active Active
-
2018
- 2018-10-17 JP JP2018195573A patent/JP6879988B2/ja active Active
- 2018-12-28 US US16/234,819 patent/US20190194688A1/en not_active Abandoned
- 2018-12-28 US US16/234,834 patent/US20190194689A1/en not_active Abandoned
-
2019
- 2019-01-16 JP JP2019005016A patent/JP6941632B2/ja active Active
- 2019-03-26 US US16/365,065 patent/US20190276849A1/en not_active Abandoned
- 2019-03-26 US US16/364,763 patent/US20190276848A1/en not_active Abandoned
-
2021
- 2021-04-30 JP JP2021077161A patent/JP2021138702A/ja active Pending
-
2022
- 2022-11-08 US US18/053,407 patent/US20230332179A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2013531471A5 (enExample) | ||
| CN115023242B (zh) | 腺相关病毒载体变体 | |
| JP7374119B2 (ja) | 減少した肝臓向性を有するＡＡＶ９とＡＡＶｒｈ７４とのハイブリッド組換えアデノ随伴ウイルス血清型 | |
| JP7452953B2 (ja) | １色覚および他の疾患の治療のためのプロモーター、発現カセット、ベクター、キット、ならびに方法 | |
| US20250161485A1 (en) | Aav capsid variants and uses thereof | |
| CN110606874B (zh) | 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途 | |
| KR20250025387A (ko) | Aav 캡시드 변이체 및 이의 용도 | |
| KR20230051208A (ko) | 신규 aav 캡시드 및 이를 함유하는 조성물 | |
| US20240067984A1 (en) | Triple function adeno-associated virus (aav)vectors for the treatment of c9orf72 associated diseases | |
| JP2022531809A (ja) | ＡＴＰａｓｅ媒介性疾患の処置のための組成物および方法 | |
| WO2024006741A1 (en) | Aav capsid variants and uses thereof | |
| WO2024011112A1 (en) | Aav capsid variants and uses thereof | |
| WO2025038430A1 (en) | Aav capsid variants and uses thereof | |
| WO2024226790A1 (en) | Aav capsid variants and uses thereof | |
| WO2023196967A1 (en) | Compositions and methods for muscle disorders | |
| CN119403823A (zh) | 具有增强的肌肉转导效率的肽修饰的aav衣壳 | |
| CN119677545A (zh) | 用于治疗神经系统疾病的组合物 | |
| JP6473438B2 (ja) | プロモーター組成物 | |
| WO2023288325A2 (en) | Recombinant reelin gene therapy | |
| WO2024154102A1 (en) | Compositions and methods for administration of therapeutic and diagnostic agents to the central nervous system | |
| WO2025019358A2 (en) | Mini-promoter compositions |