JP2016525356A5 - - Google Patents

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JP2016525356A5
JP2016525356A5 JP2016529835A JP2016529835A JP2016525356A5 JP 2016525356 A5 JP2016525356 A5 JP 2016525356A5 JP 2016529835 A JP2016529835 A JP 2016529835A JP 2016529835 A JP2016529835 A JP 2016529835A JP 2016525356 A5 JP2016525356 A5 JP 2016525356A5
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recombinant aav
amino acid
aav particle
acid position
capsid
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JP2016529835A
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JP2016525356A (ja
JP6495273B2 (ja
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Priority claimed from PCT/US2014/047670 external-priority patent/WO2015013313A2/en
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JP2016529835A 2013-07-22 2014-07-22 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法 Active JP6495273B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361857161P 2013-07-22 2013-07-22
US61/857,161 2013-07-22
US201461985365P 2014-04-28 2014-04-28
US61/985,365 2014-04-28
PCT/US2014/047670 WO2015013313A2 (en) 2013-07-22 2014-07-22 Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues

Related Child Applications (1)

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JP2019041018A Division JP7094236B2 (ja) 2013-07-22 2019-03-06 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法

Publications (3)

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JP2016525356A JP2016525356A (ja) 2016-08-25
JP2016525356A5 true JP2016525356A5 (enExample) 2017-09-28
JP6495273B2 JP6495273B2 (ja) 2019-04-03

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JP2016529835A Active JP6495273B2 (ja) 2013-07-22 2014-07-22 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
JP2019041018A Active JP7094236B2 (ja) 2013-07-22 2019-03-06 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
JP2022099592A Active JP7607007B2 (ja) 2013-07-22 2022-06-21 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
JP2024116303A Pending JP2024150613A (ja) 2013-07-22 2024-07-19 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法

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JP2019041018A Active JP7094236B2 (ja) 2013-07-22 2019-03-06 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
JP2022099592A Active JP7607007B2 (ja) 2013-07-22 2022-06-21 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
JP2024116303A Pending JP2024150613A (ja) 2013-07-22 2024-07-19 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法

Country Status (24)

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US (2) US9840719B2 (enExample)
EP (2) EP3024498B1 (enExample)
JP (4) JP6495273B2 (enExample)
KR (3) KR102380265B1 (enExample)
CN (2) CN105579465B (enExample)
AU (4) AU2014293253B2 (enExample)
CA (2) CA3209883A1 (enExample)
DK (1) DK3024498T3 (enExample)
ES (1) ES2774966T3 (enExample)
HU (1) HUE047996T2 (enExample)
IL (6) IL297919A (enExample)
MX (2) MX2016001044A (enExample)
MY (1) MY175477A (enExample)
NZ (1) NZ716102A (enExample)
PE (1) PE20160188A1 (enExample)
PH (2) PH12016500162B1 (enExample)
PL (1) PL3024498T3 (enExample)
PT (1) PT3024498T (enExample)
RU (1) RU2697444C2 (enExample)
SA (1) SA516370442B1 (enExample)
SG (2) SG11201600518WA (enExample)
SI (1) SI3024498T1 (enExample)
WO (1) WO2015013313A2 (enExample)
ZA (1) ZA201600481B (enExample)

Families Citing this family (203)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3409296A1 (en) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
PH12016500162B1 (en) * 2013-07-22 2024-02-21 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues
PL3851537T3 (pl) 2014-04-25 2024-09-16 Genethon Leczenie hiperbilirubinemii
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016077689A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
US11103597B2 (en) * 2015-05-14 2021-08-31 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers outside ITR
CA2990193A1 (en) * 2015-06-23 2016-12-29 The Children's Hospital Of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
PT3356390T (pt) 2015-09-28 2021-04-21 Univ Florida Métodos e composições para vetores virais que se evadem a anticorpos
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
WO2017077451A1 (en) 2015-11-05 2017-05-11 Bamboo Therapeutics, Inc. Modified friedreich ataxia genes and vectors for gene therapy
MX2018006682A (es) * 2015-12-01 2018-09-26 Spark Therapeutics Inc Metodos escalables para producir vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension libre de suero adecuado para su uso clinico.
WO2017096164A1 (en) 2015-12-02 2017-06-08 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
US20180363000A1 (en) * 2015-12-14 2018-12-20 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
JP7061067B2 (ja) 2015-12-14 2022-04-27 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 クリグラー・ナジャー症候群の処置のための組成物
US10179176B2 (en) 2016-02-16 2019-01-15 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
CA3019126A1 (en) * 2016-03-30 2017-10-05 Spark Therapeutics, Inc. Cell line for recombinant protein and/or viral vector production
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics Inc composições e métodos de tratamento da doença de huntington
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
EP3491008A2 (en) 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
US11339406B2 (en) 2016-09-12 2022-05-24 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293260A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3534892A4 (en) * 2016-11-04 2020-05-27 The Children's Hospital of Philadelphia GENE TRANSFER COMPOSITIONS, METHODS AND USE FOR TREATING NEURODEGENERATIVE DISEASES
US20230190958A1 (en) 2017-01-13 2023-06-22 Jichi Medical University AAV Vector for Disrupting Coagulation Factor-Related Gene on Liver Genome
CN110914419A (zh) 2017-03-10 2020-03-24 吉尼松公司 糖原贮积病iii的治疗
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
CN110770346B (zh) * 2017-03-15 2024-01-12 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US20200172927A1 (en) * 2017-05-15 2020-06-04 The Trustees Of Columbia University In The City Of New York Reprogramming metabolism by inhibiting vhl for treatment of neurodegeneration
CA3066358A1 (en) 2017-06-07 2018-12-13 Spark Therapeutics, Inc. Enhancing agents for improved cell transfection and/or raav vector production
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3069880A1 (en) * 2017-07-17 2019-01-24 Spark Therapeutics, Inc. Apheresis methods and uses
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
CN111163811A (zh) * 2017-08-25 2020-05-15 奥维德医疗公司 重组腺相关载体
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
KR102892243B1 (ko) 2018-01-17 2025-11-27 아드레나스 테라퓨틱스, 인코포레이티드 21-하이드록실라제 결핍을 위한 아데노-관련 바이러스 유전자 요법
WO2019152843A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN111902539B (zh) * 2018-02-07 2025-05-16 吉尼松公司 杂合调控元件
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
AU2019227726B2 (en) * 2018-02-27 2025-09-04 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (AAV) vectors, aav vectors having reduced capsid deamidation and uses therefor
EP3765624A4 (en) * 2018-03-16 2022-05-25 Research Institute at Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
JP7378417B2 (ja) 2018-03-30 2023-11-13 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 増強されたヒト膵臓トロピズムを有する新規な組換えアデノ随伴ウイルスキャプシド
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ,インコーポレイテッド 抗体を回避するウイルスベクター
CA3093347A1 (en) * 2018-04-05 2019-10-10 Genethon Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
WO2019210267A2 (en) * 2018-04-27 2019-10-31 Spark Therapeutics, Inc. Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
WO2019212922A1 (en) 2018-04-29 2019-11-07 Regenxbio Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
US12365865B2 (en) 2018-04-29 2025-07-22 Regenxbio Inc. Scalable clarification process for recombinant AAV production
NZ759802A (en) * 2018-05-07 2025-08-29 Univ North Carolina Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
JP2021523914A (ja) 2018-05-15 2021-09-09 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. パーキンソン病を治療するための組成物および方法
US12070702B2 (en) 2018-06-14 2024-08-27 Regenxbio Inc. Anion exchange chromatography for recombinant AAV production
DK3833746T5 (da) 2018-08-08 2024-08-19 Genethon Mini-gde til behandlingen af glykogenlagringssygdom iii
AU2019319976B2 (en) 2018-08-10 2025-08-14 Regenxbio Inc. Scalable method for recombinant AAV production
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
WO2020081415A1 (en) 2018-10-15 2020-04-23 Regenxbio Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
US12383587B2 (en) 2018-10-25 2025-08-12 Takeda Pharmaceutical Company Limited AAV triple-plasmid system
EP3880830A4 (en) * 2018-11-16 2022-08-24 Asklepios Biopharmaceutical, Inc. VECTORS COMPRISING NUCLEIC ACID ENCODING LYSOSOMAL ENZYMES FUSED TO A LYSOSOMAL TARGETING SEQUENCE
AU2020241888A1 (en) 2019-03-21 2021-09-30 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors
BR112021019436A2 (pt) * 2019-03-28 2021-12-07 Harvard College Vetores de vírus adeno-associados (aav) modificados para expressão transgênica
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
HUE064411T2 (hu) 2019-04-11 2024-03-28 Regenxbio Inc Méretkizárásos kromatográfiás módszerek rekombináns adeno-asszociált víruskészítmények jellemzésére
CN113993552A (zh) 2019-04-19 2022-01-28 再生生物股份有限公司 腺相关病毒载体制剂和方法
CN113692411A (zh) 2019-04-19 2021-11-23 吉尼松公司 成纤维细胞生长因子23相关的低磷酸盐血症的基因治疗
SG11202111414RA (en) 2019-04-24 2021-11-29 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
WO2020223236A1 (en) * 2019-04-29 2020-11-05 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
EP3967706A4 (en) * 2019-05-08 2022-12-28 Center For Excellence In Brain Science And Intelligence Technology, Chinese Academy Of Sciences AAV MUTANT FOR EFFICIENT INFECTION OF SUPPORT CELLS
CN110205297A (zh) * 2019-05-15 2019-09-06 华北理工大学 一种重组脂肪干细胞及其重组方法
CA3144864A1 (en) 2019-06-27 2020-12-30 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
EP3996696A1 (en) 2019-07-09 2022-05-18 Genethon Treatment of glycogen storage disease (gsd)
US20220251567A1 (en) 2019-07-10 2022-08-11 Inserm (Institut National De La Santè Et De La Recherche Médicale) Methods for the treatment of epilepsy
WO2021009805A1 (ja) 2019-07-12 2021-01-21 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2021021661A1 (en) 2019-07-26 2021-02-04 Regenxbio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
RU2751592C2 (ru) * 2019-08-22 2021-07-15 Общество С Ограниченной Ответственностью "Анабион" Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
US20220298514A1 (en) * 2019-09-11 2022-09-22 Aav Gene Therapeutics, Inc. Aav-zyme and use for infusion replacement therapy
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
WO2021078834A1 (en) 2019-10-22 2021-04-29 Genethon Chimeric acid-alpha glucosidase polypeptides and uses thereof
US20230033268A1 (en) 2019-10-22 2023-02-02 Genethon Chimeric polypeptides and uses thereof
AU2020384294A1 (en) * 2019-11-14 2022-06-02 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with liver-specific gene therapy vectors
US20230016983A1 (en) 2019-11-19 2023-01-19 lNSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) Antisense oligonucleotides and thier use for the treatment of cancer
JP2023503637A (ja) 2019-11-28 2023-01-31 リジェネックスバイオ インコーポレイテッド マイクロジストロフィン遺伝子治療コンストラクト及びその使用
KR20220118421A (ko) 2019-12-18 2022-08-25 제네똥 식물 모상근으로부터 재조합 바이러스 벡터의 제조
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
JP2023512043A (ja) 2020-01-29 2023-03-23 レジェンクスバイオ インコーポレーテッド ムコ多糖症ivaの治療
EP3892720A1 (en) 2020-04-06 2021-10-13 Innovative Cellular Therapeutics Holdings, Ltd. Presenting cell and use thereof in cell therapy
WO2021229255A1 (en) 2020-05-14 2021-11-18 Genethon Tools and method for preventing a a¥ neutralization by antibodies
EP3913060A1 (en) 2020-05-22 2021-11-24 Genethon Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
JP2023532923A (ja) * 2020-06-30 2023-08-01 チルドレンズ ナショナル メディカル センター 骨格筋線維の修復を改善するためのリコンビナントヒト酸性スフィンゴミエリナーゼの使用
KR20230050336A (ko) 2020-07-10 2023-04-14 인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸) 뇌전증을 치료하기 위한 방법과 조성물
AR123288A1 (es) 2020-08-19 2022-11-16 Stridebio Inc Vectores de virus adenoasociados para el tratamiento del síndrome de rett
EP4200410A1 (en) 2020-08-24 2023-06-28 Genethon C-terminal truncated gde for the treatment of glycogen storage disease iii
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
JP2023545722A (ja) 2020-10-07 2023-10-31 リジェネックスバイオ インコーポレイテッド 遺伝子治療剤の眼送達のためのアデノ随伴ウイルス
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
JP2023548746A (ja) 2020-10-13 2023-11-21 クリヤ セラピューティクス インコーポレイテッド サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用
IL302045A (en) 2020-10-15 2023-06-01 Hoffmann La Roche Nucleic acid constructs for simultaneous gene activation
ES3035361T3 (en) 2020-10-15 2025-09-02 Hoffmann La Roche Nucleic acid constructs for va rna transcription
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
AU2021371307A1 (en) 2020-10-28 2023-06-01 Regenxbio, Inc. VECTORIZED ANTI-TNF-α ANTIBODIES FOR OCULAR INDICATIONS
CN116457373A (zh) 2020-10-29 2023-07-18 再生生物股份有限公司 用于眼部适应症的载体化TNF-α拮抗剂
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
CA3200401A1 (en) 2020-11-03 2022-05-12 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
CA3199661A1 (en) 2020-12-09 2022-06-16 Mario Amendola Lysosomal acid lipase variants and uses thereof
WO2022130172A1 (en) 2020-12-15 2022-06-23 Pfizer Inc. Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins
IL303614A (en) 2020-12-16 2023-08-01 Regenxbio Inc Method of producing a recombinant adeno-associated virus particle
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of aav vectors by affinity chromatography
TW202241943A (zh) 2020-12-29 2022-11-01 美商銳進科斯生物股份有限公司 Tau特異性抗體基因療法組合物、方法及其用途
EP4281568A1 (en) 2021-01-21 2023-11-29 RegenxBio Inc. Improved production of recombinant polypeptides and viruses
US20240191213A1 (en) 2021-03-19 2024-06-13 Adrenas Therapeutics, Inc. Gene therapies for 21-hydroxylase deficiency
WO2022208342A1 (en) 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
CA3216491A1 (en) * 2021-04-16 2022-10-20 Asklepios Biopharmaceutical, Inc. Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response
JP2024515902A (ja) 2021-04-26 2024-04-10 アレクシオン ファーマ インターナショナル オペレーションズ リミテッド 組織向性が改善されたアデノ随伴ウイルスベクターカプシド
TW202309293A (zh) 2021-04-26 2023-03-01 美商銳進科斯生物股份有限公司 用於治療肌縮蛋白症(dystrophinopathy)之微小肌縮蛋白基因療法給藥
US20240218397A1 (en) 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
EP4337267A1 (en) 2021-05-11 2024-03-20 RegenxBio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
MX2023015329A (es) 2021-06-22 2024-01-23 Pfizer Produccion de vector de virus adeno-asociado en celulas de insecto.
EP4373947A1 (en) 2021-07-19 2024-05-29 New York University Auf1 combination therapies for treatment of muscle degenerative disease
AR126840A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
US20250001006A1 (en) 2021-10-07 2025-01-02 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
EP4413019A2 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2023060264A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2023069987A1 (en) 2021-10-20 2023-04-27 University Of Rochester Rejuvenation treatment of age-related white matter loss cross reference to related application
EP4423285A1 (en) 2021-10-28 2024-09-04 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2023081633A1 (en) 2021-11-02 2023-05-11 University Of Rochester Tcf7l2 mediated remyelination in the brain
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
US20230227779A1 (en) 2022-01-19 2023-07-20 Innovative Cellular Therapeutics Holdings, Ltd. Enhanced Chimeric Antigen Receptor Cells in Hypoxic Tumor Microenvironment
JP2025504478A (ja) 2022-01-21 2025-02-12 アストラゼネカ アイルランド リミテッド ゴーシェ病の遺伝子療法
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
EP4493226A1 (en) 2022-03-13 2025-01-22 RegenxBio Inc. Modified muscle-specific promoters
US20250213727A1 (en) 2022-03-25 2025-07-03 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
EP4508241A1 (en) 2022-04-13 2025-02-19 F. Hoffmann-La Roche AG Method for determining aav genomes
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
US20250249127A1 (en) 2022-04-14 2025-08-07 Regenxbio Inc. Gene therapy for treating an ocular disease
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
EP4518973A1 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
WO2023215806A2 (en) 2022-05-03 2023-11-09 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
JP2025517987A (ja) 2022-05-23 2025-06-12 エフ. ホフマン-ラ ロシュ アーゲー Aav粒子血清型およびaav粒子負荷状態の識別のためのラマンベースの方法
CN119301260A (zh) 2022-06-03 2025-01-10 豪夫迈·罗氏有限公司 用于生产重组aav颗粒的方法
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
CA3252764A1 (en) 2022-06-09 2023-12-14 Institut National de la Santé et de la Recherche Médicale GDE truncated at the N-terminal level for the treatment of type III glycogen storage disease
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
CA3261054A1 (en) 2022-07-14 2024-01-18 F. Hoffmann-La Roche Ag PROCESS FOR THE PRODUCTION OF RECOMBINANT ADENO-ASSOCIATED VIRUS PARTICLES
EP4558149A1 (en) 2022-07-21 2025-05-28 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
IL317385A (en) 2022-08-15 2025-02-01 Hoffmann La Roche Preventing or reducing side effects associated with recombinant viral vectors
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
JP2025527658A (ja) 2022-08-24 2025-08-22 リジェネックスバイオ インコーポレイテッド 組換えアデノ随伴ウイルス及びその使用
KR20250067819A (ko) 2022-09-12 2025-05-15 에프. 호프만-라 로슈 아게 핵산 포함 및 핵산 비포함 aav 입자를 분리하는 방법
JP2025534666A (ja) 2022-10-11 2025-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
CN120752339A (zh) 2023-01-06 2025-10-03 国家医疗保健研究所 静脉内施用用于治疗疼痛的反义寡核苷酸
WO2024163747A2 (en) 2023-02-02 2024-08-08 University Of Rochester Competitive replacement of glial cells
AU2024216670A1 (en) 2023-02-07 2025-07-31 F. Hoffmann-La Roche Ag Method for the detection of anti-aav particle antibodies
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
KR20250162543A (ko) 2023-03-21 2025-11-18 에프. 호프만-라 로슈 아게 재조합 aav 입자 제제 생산 방법
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
CN120936723A (zh) 2023-04-13 2025-11-11 豪夫迈·罗氏有限公司 改进的重组多腺苷酸化信号序列及其用途
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024233099A1 (en) * 2023-05-05 2024-11-14 Regeneron Pharmaceuticals, Inc. Labeling and anti-drug antibody assays for aav vectors
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2025003477A1 (en) 2023-06-29 2025-01-02 Genethon N-terminal truncated glycogen debranching enzymes for the treatment of glycogen storage disease iii
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
WO2025082618A1 (en) 2023-10-20 2025-04-24 Fuse Vectors Aps In vitro parvovirus capsid manufacturing
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025108407A2 (en) 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
WO2025168663A1 (en) 2024-02-09 2025-08-14 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral particles
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025237990A1 (en) 2024-05-14 2025-11-20 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2025252480A1 (en) 2024-06-07 2025-12-11 F. Hoffmann-La Roche Ag Method for purifying plasmid dna

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA1202962A (en) 1982-10-19 1986-04-08 David P. Clifford Substituted n-phenyl-n'-benzoyl ureas and their use as insecticides and acaricides
US4965188A (en) 1986-08-22 1990-10-23 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
JP2001500015A (ja) 1996-09-06 2001-01-09 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 T7ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO1999003496A1 (en) 1997-07-21 1999-01-28 The University Of North Carolina At Chapel Hill Factor ix antihemophilic factor with increased clotting activity
WO2001036603A2 (en) * 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US20080220015A1 (en) 2001-08-03 2008-09-11 Nokad, S.A. Method of modulating neutralizing antibodies formation in mammals, and uses thereof in gene therapy, animal trangenesis and in functional inactivation of endogenous proteins
SG168422A1 (en) 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
CA2479759A1 (en) 2002-03-22 2003-10-09 Board Of Regents, The University Of Texas System Protamine-adenoviral vector complexes and methods of use
AU2003295312B2 (en) 2002-05-01 2008-08-14 University Of Florida Research Foundation, Inc. Improved rAAV expression systems for genetic modification of specific capsid proteins
PL2357189T3 (pl) * 2003-06-19 2017-08-31 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
WO2006039253A2 (en) 2004-09-29 2006-04-13 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
EP3409296A1 (en) * 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
CN101495624A (zh) 2006-04-28 2009-07-29 宾夕法尼亚州立大学托管会 衣壳免疫原性降低的经修饰aav载体及其用途
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP3540055A1 (en) 2010-04-23 2019-09-18 University of Massachusetts Cns targeting aav vectors and methods of use thereof
AU2011242527B2 (en) 2010-04-23 2016-05-19 University Of Florida Research Foundation, Inc. rAAV-guanylate cyclase compositions and methods for treating Leber's congenital amaurosis-1 (LCA1)
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
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
US10640785B2 (en) * 2011-11-22 2020-05-05 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
WO2013123457A1 (en) 2012-02-15 2013-08-22 Biogen Idec Ma Inc. Recombinant factor viii proteins
CN104520428B (zh) * 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
CA2870736C (en) * 2012-04-18 2021-11-02 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
PH12016500162B1 (en) * 2013-07-22 2024-02-21 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues
CA2990193A1 (en) 2015-06-23 2016-12-29 The Children's Hospital Of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues

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