JP2020510429A5 - - Google Patents
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- JP2020510429A5 JP2020510429A5 JP2019547088A JP2019547088A JP2020510429A5 JP 2020510429 A5 JP2020510429 A5 JP 2020510429A5 JP 2019547088 A JP2019547088 A JP 2019547088A JP 2019547088 A JP2019547088 A JP 2019547088A JP 2020510429 A5 JP2020510429 A5 JP 2020510429A5
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- Prior art keywords
- seq
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- nucleic acid
- amino acid
- Prior art date
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- 108090000623 proteins and genes Proteins 0.000 claims 42
- 235000018102 proteins Nutrition 0.000 claims 40
- 102000004169 proteins and genes Human genes 0.000 claims 40
- 150000001413 amino acids Chemical group 0.000 claims 31
- 150000007523 nucleic acids Chemical group 0.000 claims 25
- 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 22
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 17
- 241000702421 Dependoparvovirus Species 0.000 claims 16
- 235000001014 amino acid Nutrition 0.000 claims 15
- 229940024606 amino acid Drugs 0.000 claims 15
- 210000000234 capsid Anatomy 0.000 claims 12
- 239000004471 Glycine Substances 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 102000039446 nucleic acids Human genes 0.000 claims 6
- 108020004705 Codon Proteins 0.000 claims 5
- 230000006240 deamidation Effects 0.000 claims 5
- 239000004220 glutamic acid Substances 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- 230000004048 modification Effects 0.000 claims 4
- 238000012986 modification Methods 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 108090000565 Capsid Proteins Proteins 0.000 claims 3
- 102100023321 Ceruloplasmin Human genes 0.000 claims 3
- CKLJMWTZIZZHCS-REOHCLBHSA-N L-aspartic acid Chemical compound OC(=O)[C@@H](N)CC(O)=O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 claims 3
- 238000004949 mass spectrometry Methods 0.000 claims 3
- DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Natural products OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 claims 2
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims 2
- DCXYFEDJOCDNAF-REOHCLBHSA-N L-asparagine Chemical compound OC(=O)[C@@H](N)CC(N)=O DCXYFEDJOCDNAF-REOHCLBHSA-N 0.000 claims 2
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Natural products CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 claims 2
- 229960001230 asparagine Drugs 0.000 claims 2
- 235000009582 asparagine Nutrition 0.000 claims 2
- 235000003704 aspartic acid Nutrition 0.000 claims 2
- OQFSQFPPLPISGP-UHFFFAOYSA-N beta-carboxyaspartic acid Natural products OC(=O)C(N)C(C(O)=O)C(O)=O OQFSQFPPLPISGP-UHFFFAOYSA-N 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 238000004806 packaging method and process Methods 0.000 claims 2
- 125000006850 spacer group Chemical group 0.000 claims 2
- 239000004474 valine Substances 0.000 claims 2
- 125000002987 valine group Chemical group [H]N([H])C([H])(C(*)=O)C([H])(C([H])([H])[H])C([H])([H])[H] 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 239000004475 Arginine Substances 0.000 claims 1
- 101150044789 Cap gene Proteins 0.000 claims 1
- 108091026890 Coding region Proteins 0.000 claims 1
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 claims 1
- QIVBCDIJIAJPQS-VIFPVBQESA-N L-tryptophane Chemical compound C1=CC=C2C(C[C@H](N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-VIFPVBQESA-N 0.000 claims 1
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 1
- 239000004472 Lysine Substances 0.000 claims 1
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims 1
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 claims 1
- 239000004473 Threonine Substances 0.000 claims 1
- QIVBCDIJIAJPQS-UHFFFAOYSA-N Tryptophan Natural products C1=CC=C2C(CC(N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-UHFFFAOYSA-N 0.000 claims 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 1
- 125000000637 arginyl group Chemical class N[C@@H](CCCNC(N)=N)C(=O)* 0.000 claims 1
- 150000001508 asparagines Chemical class 0.000 claims 1
- 229940009098 aspartate Drugs 0.000 claims 1
- 238000004113 cell culture Methods 0.000 claims 1
- 210000003169 central nervous system Anatomy 0.000 claims 1
- 238000001415 gene therapy Methods 0.000 claims 1
- 229930195712 glutamate Natural products 0.000 claims 1
- WHUUTDBJXJRKMK-VKHMYHEASA-L glutamate group Chemical group N[C@@H](CCC(=O)[O-])C(=O)[O-] WHUUTDBJXJRKMK-VKHMYHEASA-L 0.000 claims 1
- 150000002333 glycines Chemical class 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 210000003734 kidney Anatomy 0.000 claims 1
- 229930182817 methionine Natural products 0.000 claims 1
- 238000013322 recombinant adeno-associated virus production system Methods 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023064895A JP7754872B2 (ja) | 2017-02-28 | 2023-04-12 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| JP2025025694A JP2025081544A (ja) | 2017-02-28 | 2025-02-20 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762464748P | 2017-02-28 | 2017-02-28 | |
| US62/464,748 | 2017-02-28 | ||
| US201762591002P | 2017-11-27 | 2017-11-27 | |
| US62/591,002 | 2017-11-27 | ||
| US201862614002P | 2018-01-05 | 2018-01-05 | |
| US62/614,002 | 2018-01-05 | ||
| PCT/US2018/019992 WO2018160582A1 (en) | 2017-02-28 | 2018-02-27 | Adeno-associated virus (aav) clade f vector and uses therefor |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023064895A Division JP7754872B2 (ja) | 2017-02-28 | 2023-04-12 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020510429A JP2020510429A (ja) | 2020-04-09 |
| JP2020510429A5 true JP2020510429A5 (enExample) | 2021-04-08 |
| JP7455579B2 JP7455579B2 (ja) | 2024-03-26 |
Family
ID=61628475
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019547088A Active JP7455579B2 (ja) | 2017-02-28 | 2018-02-27 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| JP2023064895A Active JP7754872B2 (ja) | 2017-02-28 | 2023-04-12 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| JP2025025694A Pending JP2025081544A (ja) | 2017-02-28 | 2025-02-20 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023064895A Active JP7754872B2 (ja) | 2017-02-28 | 2023-04-12 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| JP2025025694A Pending JP2025081544A (ja) | 2017-02-28 | 2025-02-20 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
Country Status (29)
| Country | Link |
|---|---|
| US (2) | US11827906B2 (enExample) |
| EP (2) | EP3589730B1 (enExample) |
| JP (3) | JP7455579B2 (enExample) |
| KR (1) | KR102775350B1 (enExample) |
| CN (2) | CN110741082B (enExample) |
| AU (2) | AU2018227440B2 (enExample) |
| BR (1) | BR112019017839A2 (enExample) |
| CA (1) | CA3053399A1 (enExample) |
| CL (1) | CL2019002474A1 (enExample) |
| CO (1) | CO2019009525A2 (enExample) |
| DK (1) | DK3589730T5 (enExample) |
| ES (1) | ES2971872T3 (enExample) |
| FI (1) | FI3589730T3 (enExample) |
| HR (1) | HRP20240257T1 (enExample) |
| HU (1) | HUE065885T2 (enExample) |
| IL (1) | IL268644B2 (enExample) |
| LT (1) | LT3589730T (enExample) |
| MD (1) | MD3589730T2 (enExample) |
| MX (2) | MX2019010275A (enExample) |
| PE (1) | PE20191362A1 (enExample) |
| PL (1) | PL3589730T3 (enExample) |
| PT (1) | PT3589730T (enExample) |
| RS (1) | RS65241B1 (enExample) |
| SA (1) | SA519402565B1 (enExample) |
| SG (4) | SG10201912973XA (enExample) |
| SI (1) | SI3589730T1 (enExample) |
| SM (1) | SMT202400080T1 (enExample) |
| WO (1) | WO2018160582A1 (enExample) |
| ZA (1) | ZA201905407B (enExample) |
Families Citing this family (78)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| US10494645B2 (en) | 2013-04-18 | 2019-12-03 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| CA3011529A1 (en) * | 2016-01-15 | 2017-07-20 | American Gene Technologies International Inc. | Methods and compositions for the activation of gamma-delta t-cells |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| US11827906B2 (en) | 2017-02-28 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
| RU2019139555A (ru) | 2017-05-11 | 2021-06-11 | Зе Трастис Оф Зе Юниверсити Оф Пеннсильвания | Генная терапия нейрональных цероидных липофусцинозов |
| CA3083761A1 (en) | 2017-11-30 | 2019-06-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis iiib |
| JP7389744B2 (ja) | 2017-11-30 | 2023-11-30 | 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ｉｉｉａ型のための遺伝子療法 |
| EP3758724A4 (en) * | 2018-02-27 | 2022-07-06 | The Trustees of The University of Pennsylvania | NOVEL VECTORS OF ADENO-ASSOCIATED VIRUS (AAV), AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF |
| MX2020008932A (es) | 2018-02-27 | 2020-10-01 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| KR20210071017A (ko) * | 2018-10-01 | 2021-06-15 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Gm1 강글리오시드증 치료에 유용한 조성물 |
| UY38407A (es) | 2018-10-15 | 2020-05-29 | Novartis Ag | Anticuerpos estabilizadores de trem2 |
| SG11202108614QA (en) | 2019-02-15 | 2021-09-29 | Sangamo Therapeutics Inc | Compositions and methods for producing recombinant aav |
| EP3927381A1 (en) | 2019-02-22 | 2021-12-29 | The Trustees of The University of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| MX2021010266A (es) * | 2019-02-26 | 2021-09-23 | Univ Pennsylvania | Composiciones utiles en el tratamiento de la enfermedad de krabbe. |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| US20220202960A1 (en) * | 2019-04-24 | 2022-06-30 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| PH12021552608A1 (en) * | 2019-04-29 | 2022-06-20 | Univ Pennsylvania | Novel aav capsids and compositions containing same |
| WO2020227166A1 (en) * | 2019-05-03 | 2020-11-12 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療ｃ型尼曼—匹克病之腺相關病毒載體 |
| EP4072572A4 (en) * | 2019-12-10 | 2024-01-03 | Homology Medicines, Inc. | ADENO-ASSOCIATED VIRUS COMPOSITIONS AND METHODS OF USE THEREOF |
| WO2021155337A1 (en) | 2020-02-02 | 2021-08-05 | The Trustees Of The University Of Pennsylvania | Compositions useful for treating gm1 gangliosidosis |
| CN115287350B (zh) * | 2020-03-30 | 2025-07-04 | 中国医学科学院肿瘤医院 | 包括外泌体miR-106b-3p、miR-10a-3p等在肺癌诊断中的应用 |
| IL297635A (en) * | 2020-04-27 | 2022-12-01 | Univ Pennsylvania | Compositions useful in treatment of cdkl5 deficiency disorder (cdd) |
| AU2021270447A1 (en) | 2020-05-12 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Compositions for DRG-specific reduction of transgene expression |
| TW202208622A (zh) * | 2020-05-12 | 2022-03-01 | 賓州大學委員會 | 用於治療克拉培氏病之組成物 |
| WO2021242909A1 (en) | 2020-05-26 | 2021-12-02 | Shape Therapeutics Inc. | High throughput engineering of functional aav capsids |
| TW202216244A (zh) | 2020-07-13 | 2022-05-01 | 賓州大學委員會 | 有用於治療夏馬杜三氏病之組成物 |
| JP2023536148A (ja) | 2020-07-30 | 2023-08-23 | シェイプ セラピューティクス インク． | Ｒａａｖウイルス粒子の誘導産生のための安定な細胞株 |
| 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 |
| MX2023002293A (es) | 2020-08-24 | 2023-05-19 | Univ Pennsylvania | Vectores virales que codifican fusiones de agonistas del receptor de glp-1 y usos de estos en el tratamiento de enfermedades metabólica. |
| MX2023002364A (es) | 2020-08-26 | 2023-05-22 | Univ Pennsylvania | Virus adenoasociado recombinante para el tratamiento de la neurodegeneración iniciada en adultos asociada a grn. |
| KR20230118075A (ko) | 2020-10-09 | 2023-08-10 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 파브리병 치료를 위한 조성물 및 방법 |
| TW202229315A (zh) | 2020-10-18 | 2022-08-01 | 賓州大學委員會 | 改良的腺相關病毒(aav)載體及其用途 |
| CA3199234A1 (en) * | 2020-10-22 | 2022-04-28 | Scout Bio, Inc. | Method of suppressing immune response to vector-delivered therapeutic protein |
| WO2022094078A1 (en) | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| EP4236975A1 (en) * | 2020-10-29 | 2023-09-06 | The Trustees of The University of Pennsylvania | Aav capsids and compositions containing same |
| EP4236974A2 (en) * | 2020-10-29 | 2023-09-06 | RegenxBio Inc. | Vectorized factor xii antibodies and administration thereof |
| TW202233584A (zh) | 2020-11-11 | 2022-09-01 | 歐洲分子生物學實驗室 | 用於基因治療之經修飾病毒顆粒 |
| EP4256065A2 (en) | 2020-12-01 | 2023-10-11 | The Trustees of The University of Pennsylvania | Novel compositions with tissue-specific targeting motifs and compositions containing same |
| CA3200192A1 (en) | 2020-12-01 | 2022-06-09 | Justin PERCIVAL | Compositions and uses thereof for treatment of angelman syndrome |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| US20240191258A1 (en) | 2021-04-12 | 2024-06-13 | The Trustees Of The University Of Pennsylvania | Compositions useful for treating spinal and bulbar muscular atrophy (sbma) |
| EP4334334A1 (en) | 2021-04-23 | 2024-03-13 | The Trustees of The University of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| AU2022274162A1 (en) | 2021-05-12 | 2023-11-30 | Fondazione Telethon | Vector system |
| IL308787A (en) * | 2021-06-08 | 2024-01-01 | Univ Pennsylvania | Recombinant adeno-associated viruses for Leish-Nihan disorders and their uses |
| TW202325845A (zh) | 2021-10-02 | 2023-07-01 | 賓州大學委員會 | 新穎aav衣殼及含其之組成物 |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20250026795A1 (en) | 2021-12-01 | 2025-01-23 | Shape Therapeutics Inc. | Functional aav capsids for intravitreal administration |
| AU2022401548A1 (en) | 2021-12-01 | 2024-07-04 | Shape Therapeutics Inc. | Functional aav capsids for systemic administration |
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| WO2023133584A1 (en) | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| TW202340467A (zh) | 2022-01-10 | 2023-10-16 | 賓州大學委員會 | 有用於治療c9orf72介導之病症之組成物及方法 |
| IL314434A (en) | 2022-01-25 | 2024-09-01 | Univ Pennsylvania | AAV capsids for enhanced cardiac transduction and liver targeting neutralization |
| CA3247109A1 (en) | 2022-04-06 | 2023-10-12 | Univ Pennsylvania | Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers |
| EP4504258A1 (en) | 2022-04-06 | 2025-02-12 | The Trustees of the University of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| US20250353883A1 (en) | 2022-05-06 | 2025-11-20 | Novartis Ag | Novel recombinant aav vp2 fusion polypeptides |
| EP4522757A2 (en) * | 2022-05-13 | 2025-03-19 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| CN121127599A (zh) | 2022-12-17 | 2025-12-12 | 宾夕法尼亚州大学信托人 | 具有心肌和骨骼肌特异性靶向基序的重组aav突变载体及含有其的组合物 |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| AU2024221161A1 (en) | 2023-02-17 | 2025-08-28 | Biogen Ma Inc. | Rgd-containing peptides for delivering payloads |
| WO2024258961A1 (en) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| WO2025035143A1 (en) | 2023-08-10 | 2025-02-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of spinal muscular atrophy |
| WO2025076091A1 (en) * | 2023-10-03 | 2025-04-10 | Brave Bio, Inc. | Cd20 antibody aav vectors and methods of use thereof |
| WO2025090858A1 (en) | 2023-10-27 | 2025-05-01 | Biogen Ma Inc. | Methods for identifying aav capsid variants with desired characteristics |
| WO2025096967A1 (en) | 2023-11-02 | 2025-05-08 | Biogen Ma Inc. | Redirection of aav capsids for central nervous system targeting |
| WO2025102034A1 (en) | 2023-11-10 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
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2023
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2025
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