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PL3459965T3 - Sposoby przewidywania sekwencji wirusa-przodka i ich zastosowania - Google Patents

Sposoby przewidywania sekwencji wirusa-przodka i ich zastosowania

Info

Publication number
PL3459965T3
PL3459965T3 PL18190809T PL18190809T PL3459965T3 PL 3459965 T3 PL3459965 T3 PL 3459965T3 PL 18190809 T PL18190809 T PL 18190809T PL 18190809 T PL18190809 T PL 18190809T PL 3459965 T3 PL3459965 T3 PL 3459965T3
Authority
PL
Poland
Prior art keywords
methods
virus sequences
ancestral virus
predicting
predicting ancestral
Prior art date
Application number
PL18190809T
Other languages
English (en)
Inventor
Luk H. VANDENBERGHE
Eric ZINN
Original Assignee
Massachusetts Eye & Ear Infirmary
Schepens Eye Research Institute
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Massachusetts Eye & Ear Infirmary, Schepens Eye Research Institute filed Critical Massachusetts Eye & Ear Infirmary
Publication of PL3459965T3 publication Critical patent/PL3459965T3/pl

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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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PL18190809T 2013-10-11 2014-10-10 Sposoby przewidywania sekwencji wirusa-przodka i ich zastosowania PL3459965T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361889827P 2013-10-11 2013-10-11
EP14789476.0A EP3060575B1 (en) 2013-10-11 2014-10-10 Methods of predicting ancestral virus sequences and uses thereof
EP18190809.6A EP3459965B1 (en) 2013-10-11 2014-10-10 Methods of predicting ancestral virus sequences and uses thereof
PCT/US2014/060163 WO2015054653A2 (en) 2013-10-11 2014-10-10 Methods of predicting ancestral virus sequences and uses thereof

Publications (1)

Publication Number Publication Date
PL3459965T3 true PL3459965T3 (pl) 2021-07-26

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PL18190809T PL3459965T3 (pl) 2013-10-11 2014-10-10 Sposoby przewidywania sekwencji wirusa-przodka i ich zastosowania

Country Status (14)

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US (8) US9695220B2 (pl)
EP (3) EP3459965B1 (pl)
JP (6) JP6552511B2 (pl)
CN (6) CN115141258B (pl)
AU (7) AU2014331708B2 (pl)
CA (2) CA2927077C (pl)
DK (1) DK3459965T3 (pl)
ES (2) ES2857751T3 (pl)
HU (1) HUE052676T2 (pl)
LT (1) LT3459965T (pl)
NZ (5) NZ758024A (pl)
PL (1) PL3459965T3 (pl)
PT (1) PT3459965T (pl)
WO (1) WO2015054653A2 (pl)

Families Citing this family (184)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2359865B1 (en) * 2005-04-07 2013-10-02 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
DK2699270T3 (en) 2011-04-22 2017-10-02 Univ California ADENO-ASSOCIATED VIRUS VIRUSES WITH VARIANT CAPSID AND METHODS FOR USING IT
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
CN115141258B (zh) 2013-10-11 2025-05-20 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
JP6689249B2 (ja) 2014-03-17 2020-05-20 アドヴェラム バイオテクノロジーズ, インコーポレイテッド 錐体細胞における増強された遺伝子発現のための組成物および方法
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
RU2716422C2 (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
CN112410339A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
AU2016226289B2 (en) 2015-03-02 2021-04-29 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
HUE051491T2 (hu) 2015-03-06 2021-03-01 Massachusetts Eye & Ear Infirmary Gén-augmentációs terápiák a PRPF31 gén mutációi által okozott öröklött retina degenerációra
US10883117B2 (en) * 2015-03-24 2021-01-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
CA2985223A1 (en) 2015-05-07 2016-11-10 Massachusetts Eye And Ear Infirmary Methods of delivering an agent to the eye
GB201508026D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Capsid
ES2799515T3 (es) 2015-05-22 2020-12-18 Dmitry Dmitrievich Genkin ADN extracelular como una diana terapéutica en la neurodegeneración
JP7166168B2 (ja) * 2015-07-30 2022-11-07 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列およびその使用
CN108137655B (zh) 2015-09-28 2022-04-22 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
BR112018007453A2 (pt) 2015-11-05 2018-10-23 Bamboo Therapeutics Inc genes modificados de ataxia de friedreich e vetores para a terapia gênica
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
KR102794870B1 (ko) 2015-12-11 2025-04-10 매사추세츠 아이 앤드 이어 인퍼머리 핵산을 와우 및 전정 세포에 전달하기 위한 물질 및 방법
IL262207B1 (en) 2016-04-15 2025-09-01 Univ Pennsylvania Compositions for treatment of wet age-related macular degeneration
US20190142969A1 (en) * 2016-04-26 2019-05-16 Massachusetts Eye And Ear Infirmary ISL1-Based Gene Therapy to Treat Hearing Loss
CN109831916B (zh) 2016-05-18 2023-07-21 沃雅戈治疗公司 治疗亨廷顿氏舞蹈病的组合物和方法
CA3252099A1 (en) 2016-05-18 2025-06-05 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
MX2019000962A (es) * 2016-07-26 2019-08-01 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado.
KR20230039779A (ko) 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
CA3034527A1 (en) 2016-08-23 2018-03-01 Emmanuel John Simons Compositions and methods for treating non-age-associated hearing impairment in a human subject
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
AU2018215785B2 (en) 2017-02-06 2023-08-31 Children's Medical Center Corporation Materials and methods for delivering nucleic acids to cochlear and vestibular cells
CA3054941A1 (en) 2017-03-17 2018-09-20 Rescue Hearing Inc Gene therapy constructs and methods for treatment of hearing loss
US12391936B2 (en) 2017-03-17 2025-08-19 Rescue Hearing, Inc. Gene therapy systems and related methods for treatment of hearing loss
TW202532642A (zh) 2017-04-14 2025-08-16 美商銳進科斯生物股份有限公司 使用由人類神經或膠細胞產生的重組人類艾杜糖醛酸鹽 (iduronate)-2-硫酸酯酶 (ids) 之黏多醣病ii之治療
JP2020518258A (ja) 2017-05-05 2020-06-25 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. 筋萎縮性側索硬化症(als)治療組成物および方法
JP2020518259A (ja) 2017-05-05 2020-06-25 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. ハンチントン病治療組成物および方法
WO2018209154A1 (en) * 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (app)-dependence of viruses
CN110831611A (zh) * 2017-05-10 2020-02-21 马萨诸塞眼科耳科诊所 用于修饰病毒的组装活化蛋白(aap)依赖性的方法和组合物
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US11168322B2 (en) 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
WO2019006182A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF
CA3059995A1 (en) 2017-08-28 2019-03-07 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
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.
WO2019079494A1 (en) 2017-10-18 2019-04-25 Regenxbio, Inc. TREATMENT OF OCULAR DISEASES AND METASTATIC COLON CANCER WITH A VEGF TRAP WITH HUMAN POST-TRANSLATIONAL MODIFICATION
BR112020007405A2 (pt) 2017-10-18 2020-12-08 Regenxbio Inc. Produtos terapêuticos de anticorpo pós-translacionalmente modificados totalmente humanos
MY207644A (en) 2017-12-19 2025-03-07 Akouos Inc Aav-mediated delivery of therapeutic antibodies to the inner ear
AU2019209770B2 (en) * 2018-01-16 2025-07-31 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity
CN112423791A (zh) 2018-03-05 2021-02-26 儿童医疗中心有限公司 将核酸递送至耳蜗和前庭细胞的组合物和方法
DK3765616T3 (da) 2018-03-14 2023-08-21 Arbor Biotechnologies Inc Nye enzymer og systemer målrettet crispr dna og rna
CN112041444A (zh) 2018-03-14 2020-12-04 阿伯生物技术公司 新型crispr dna靶向酶及系统
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス 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
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
EP3775266A4 (en) 2018-04-05 2021-06-30 Massachusetts Eye and Ear Infirmary METHOD OF MANUFACTURING AND USING COMBINATIONAL BAR-CODED NUCLEIC ACID LIBRARIES WITH DEFINED VARIATION
JP7575273B2 (ja) 2018-04-16 2024-10-29 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 デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法
US20210231560A1 (en) 2018-04-29 2021-07-29 Regenxbio Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
CA3098565A1 (en) 2018-04-29 2019-11-07 Claire G. ZHANG Scalable clarification process for recombinant aav production
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
EP3790567B1 (en) 2018-05-11 2025-08-06 Massachusetts Eye and Ear Infirmary Liver-specific tropism of adeno-associated viruses
US20210180084A1 (en) 2018-05-14 2021-06-17 Vivet Therapeutics Gene therapy vectors comprising s/mar sequences
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
BR112020023082A2 (pt) 2018-05-15 2021-02-09 Voyager Therapeutics, Inc. composições e métodos para o tratamento de doença de parkinson
US12070702B2 (en) 2018-06-14 2024-08-27 Regenxbio Inc. Anion exchange chromatography for recombinant AAV production
WO2020005341A1 (en) 2018-06-29 2020-01-02 Icahn School Of Medicine At Mount Sinai Anc80 encoding sphingolipid-metabolizing proteins
WO2020018142A1 (en) 2018-07-16 2020-01-23 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
JP2021533757A (ja) 2018-08-10 2021-12-09 リジェネクスバイオ インコーポレイテッド 組換えaav生成のためのスケーラブルな方法
EP3837349A4 (en) 2018-08-16 2022-06-08 The Regents of the University of California CHEMICALLY AND PHOTOCHEMICALLY INITIATED CELL MEMBRANE BLISTER FOR INDUCING CELL VESICLES PRODUCTION, MODIFICATIONS THEREOF, AND USES THEREOF
CA3114621A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
EP3867412A1 (en) 2018-10-15 2021-08-25 REGENXBIO Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
EP3924364A1 (en) * 2019-02-15 2021-12-22 Sangamo Therapeutics, Inc. Compositions and methods for producing recombinant aav
WO2020168111A1 (en) 2019-02-15 2020-08-20 Exhaura, Ltd. Dual leucine zipper kinase inhibitors for gene therapy
CA3133453A1 (en) 2019-03-21 2020-09-24 Daniel Mccoy Recombinant adeno-associated virus vectors
CN113966236A (zh) 2019-04-03 2022-01-21 再生生物股份有限公司 眼睛病状的基因疗法
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
PL3953483T3 (pl) 2019-04-11 2024-04-22 Regenxbio Inc. Sposoby chromatografii wykluczania do charakteryzowania kompozycji rekombinowanych wirusów towarzyszących adenowirusom
MX2021012564A (es) 2019-04-19 2022-01-18 Regenxbio Inc Formulaciones y métodos de vectores de virus adenoasociados.
SG11202111414RA (en) 2019-04-24 2021-11-29 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
BR112021021502A2 (pt) * 2019-04-29 2022-07-19 Univ Pennsylvania Capsídeos de aav e composições contendo os mesmos
CN114174330B (zh) 2019-05-28 2025-01-17 总医院公司 Apoe抗体、融合蛋白及其用途
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
US20220241394A1 (en) * 2019-07-12 2022-08-04 Cls Therapeutics Limited TREATMENT OF DISEASES ASSOCIATED WITH PROTEIN MISFOLDING BY NERVOUS SYSTEM EXPRESSION OF AN ENZYME WHICH HAS A DEOXYRIBONUCLEASE (DNase) ACTIVITY
US20220265853A1 (en) * 2019-07-12 2022-08-25 Gene Therapy Research Institution Co., Ltd. Adeno-associated virus virion for gene transfer to human liver
EP4004214A1 (en) 2019-07-26 2022-06-01 RegenxBio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
JP7785352B2 (ja) * 2019-08-14 2025-12-15 ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. 遺伝子治療のためのaavカプシドバリアント
AU2020336314A1 (en) 2019-08-26 2022-04-07 Regenxbio Inc. Treatment of diabetic retinopathy with fully-human post-translationally modified anti-VEGF Fab
WO2021041953A1 (en) 2019-08-30 2021-03-04 The Regents Of The University Of California Gene fragment overexpression screening methodologies, and uses thereof
KR20220054435A (ko) 2019-09-09 2022-05-02 매사추세츠 아이 앤드 이어 인퍼머리 아데노-연관 바이러스 (aav)와 aav 수용체 (aavr) 사이의 상호작용을 조정하여 aav의 생체-분포를 변경시키는 방법 및 조성물
JP2022552262A (ja) 2019-10-07 2022-12-15 リジェネックスバイオ インコーポレイテッド アデノ随伴ウイルスベクター医薬組成物および方法
WO2021071976A1 (en) 2019-10-08 2021-04-15 Exhaura, Ltd. Compositions and methods for ocular therapy
CN113518824B (zh) * 2019-10-16 2024-02-23 上海药明康德新药开发有限公司 新的aav变体
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
WO2021099394A1 (en) 2019-11-19 2021-05-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides and their use for the treatment of cancer
BR112022009895A2 (pt) 2019-11-28 2022-08-09 Regenxbio Inc Construtos de terapia de gene de microdistrofina e usos dos mesmos
BR112022014563A2 (pt) 2020-01-22 2022-09-13 Regenxbio Inc Método para tratamento de um sujeito humano diagnosticado com mucopolissacaridose i (mps i)
CA3167741A1 (en) 2020-01-29 2021-08-05 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
AR121201A1 (es) 2020-01-29 2022-04-27 Regenxbio Inc Tratamiento de mucopolisacaridosis iva
EP4103238A4 (en) 2020-02-12 2024-03-20 Massachusetts Eye and Ear Infirmary HAPLOTYPE-BASED TREATMENT OF RP1-ASSOCIATED RETINAL DEGENERATION
BR112022016596A2 (pt) 2020-02-21 2022-11-16 Akouos Inc Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano
EP4179091A1 (en) 2020-07-10 2023-05-17 Institut National De La Sante Et De La Recherche Medicale - Inserm Methods and compositions for treating epilepsy
CN116745409A (zh) 2020-08-19 2023-09-12 萨雷普塔治疗公司 用于治疗雷特综合征的腺相关病毒载体
US20250326861A1 (en) 2020-09-15 2025-10-23 Regenxbio Inc. Vectorized lanadelumab and administration thereof
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
US20240024508A1 (en) 2020-10-07 2024-01-25 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
CA3194861A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as gel formulations
CA3193697A1 (en) 2020-10-07 2022-04-14 Joseph Bruder Adeno-associated viruses for ocular delivery of gene therapy
EP4225381A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
AR123948A1 (es) 2020-10-28 2023-01-25 Regenxbio Inc ANTICUERPOS ANTI-TNF-a VECTORIZADOS PARA INDICACIONES OCULARES
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
EP4237453A1 (en) 2020-10-29 2023-09-06 RegenxBio Inc. Vectorized tnf-alpha antagonists for ocular indications
MX2023005218A (es) 2020-11-03 2023-05-16 Pfizer Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico.
KR102862676B1 (ko) 2020-11-11 2025-09-23 유럽피안 몰레큘러 바이올로지 래보러토리 유전자 치료를 위한 변형된 바이러스 입자
WO2022119839A1 (en) * 2020-12-01 2022-06-09 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
WO2022130172A1 (en) 2020-12-15 2022-06-23 Pfizer Inc. Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins
KR20230120128A (ko) 2020-12-16 2023-08-16 리젠엑스바이오 인크. 재조합 바이러스 입자의 생산 방법
AU2021404944A1 (en) 2020-12-23 2023-07-06 Pfizer Inc. Methods for purification of aav vectors by affinity chromatography
WO2022147087A1 (en) 2020-12-29 2022-07-07 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
WO2022150634A2 (en) 2021-01-08 2022-07-14 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
CA3205209A1 (en) 2021-01-21 2022-07-28 Regenxbio Inc. Improved production of recombinant polypeptides and viruses
EP4291249A2 (en) 2021-02-10 2023-12-20 RegenxBio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
US20240307553A1 (en) 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
WO2023113806A1 (en) 2021-12-16 2023-06-22 Affinia Therapeutics, Inc. Recombinant aav for treatment of neural disease
EP4323531A1 (en) 2021-04-12 2024-02-21 Affinia Therapeutics Inc. Recombinant aav for treatment of neural disease
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
WO2022241030A1 (en) 2021-05-11 2022-11-17 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
IL308356A (en) 2021-05-12 2024-01-01 Fond Telethon Vector system
US20240425830A1 (en) 2021-05-27 2024-12-26 Institute Of Zoology, Chinese Academy Of Sciences Engineered cas12i nuclease, effector protein and use thereof
WO2022269466A1 (en) 2021-06-22 2022-12-29 Pfizer Inc. Production of adeno-associated virus vector in insect cells
EP4373947A1 (en) 2021-07-19 2024-05-29 New York University Auf1 combination therapies for treatment of muscle degenerative disease
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
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2023060272A2 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
EP4413018A1 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for targeted delivery
WO2023077092A1 (en) 2021-10-28 2023-05-04 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
WO2023178053A1 (en) 2022-03-13 2023-09-21 Regenxbio Inc. Modified muscle-specific promoters
EP4499154A1 (en) 2022-03-25 2025-02-05 REGENXBIO Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
TW202345913A (zh) 2022-04-06 2023-12-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如凝膠調配物
EP4504149A1 (en) 2022-04-06 2025-02-12 RegenxBio Inc. Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene for suprachoidal administration
TW202404651A (zh) 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
EP4507741A1 (en) 2022-04-14 2025-02-19 RegenxBio Inc. Gene therapy for treating an ocular disease
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
US20250288697A1 (en) 2022-05-03 2025-09-18 Regenxbio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
AR129215A1 (es) 2022-05-03 2024-07-31 Regenxbio Inc Anticuerpos anti-complemento y agentes del complemento vectorizados y administración de estos
JP2025517628A (ja) 2022-05-06 2025-06-10 ノバルティス アーゲー 新規の組換えaav vp2融合ポリペプチド
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
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
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
IL319873A (en) 2022-09-30 2025-05-01 Regenxbio Inc Treatment of eye diseases with recombinant viral vectors containing anti-VEGF FAB
EP4601710A2 (en) 2022-10-11 2025-08-20 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2024086747A1 (en) 2022-10-19 2024-04-25 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
EP4646478A1 (en) 2023-01-06 2025-11-12 Institut National de la Santé et de la Recherche Médicale Intravenous administration of antisense oligonucleotides for the treatment of pain
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
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
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
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
TW202511280A (zh) 2023-07-21 2025-03-16 義大利商博雷亞醫療有限責任公司 製備表面修飾之病毒外殼之方法
WO2025075963A1 (en) 2023-10-02 2025-04-10 Regenxbio Inc. Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025106374A1 (en) 2023-11-13 2025-05-22 Juno Therapeutics, Inc. Aav production method
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
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025186392A1 (en) 2024-03-06 2025-09-12 Stichting Radboud Universitair Medisch Centrum Therapeutics abca4 genome editing for treatment of stargardt disease
WO2025194042A1 (en) 2024-03-14 2025-09-18 Affinia Therapeutics Inc. Plasmid system for production of recombinant adenoassociated virus
WO2025196087A1 (en) 2024-03-19 2025-09-25 Borea Therapeutics S.R.L. Targeting tissues in the cns
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
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025227063A1 (en) 2024-04-26 2025-10-30 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
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

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5037384B2 (en) 1988-01-12 1995-12-26 Cornell Res Foundation Inc Method and apparatus for the treatment of complicated retinal detachments
US5817075A (en) 1989-08-14 1998-10-06 Photogenesis, Inc. Method for preparation and transplantation of planar implants and surgical instrument therefor
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6024719A (en) 1998-07-06 2000-02-15 Morris; Robert E Method and apparatus for performing surgery inside the human retina using fluidic internal limiting membrane (ILM) seperation (FILMS)
US20040115621A1 (en) * 2000-02-18 2004-06-17 Allen Rodrigo Ancestral viruses and vaccines
US6692526B1 (en) 2000-03-15 2004-02-17 Michael E. Snyder Ophthalmological surgery colorant and delivery system
AU7025201A (en) * 2000-07-03 2002-01-14 Gala Design Inc Expression vectors
US20060292117A1 (en) * 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
US9441244B2 (en) * 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP2292780B1 (en) 2003-09-30 2017-08-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
CA2567411A1 (en) 2004-05-31 2005-12-08 Senju Pharmaceutical Co., Ltd. Transparent tissue-visualizing preparation
CN101132814B (zh) 2004-12-06 2010-08-04 国立大学法人九州大学 用于染色眼膜的染色组合物
EP2359865B1 (en) * 2005-04-07 2013-10-02 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
US20150182756A1 (en) 2005-08-05 2015-07-02 Gholam A. Peyman Methods to regulate polarization and enhance function of cells
US10022457B2 (en) 2005-08-05 2018-07-17 Gholam A. Peyman Methods to regulate polarization and enhance function of cells
US8388668B2 (en) 2005-08-05 2013-03-05 Gholam A. Peyman Methods to regulate polarization of excitable cells
US20090226531A1 (en) 2008-03-07 2009-09-10 Allergan, Inc. Methods and composition for intraocular delivery of therapeutic sirna
US20120093772A1 (en) 2008-05-20 2012-04-19 Alan Horsager Vectors for delivery of light sensitive proteins and methods of use
US20110098536A1 (en) 2008-11-05 2011-04-28 Corcosteugi Borja F ILM Spatula with Malleable Blade
US11890226B2 (en) 2009-02-25 2024-02-06 University Of Maryland, College Park Device and methods for directing agents into an eye
US8476418B2 (en) * 2009-05-28 2013-07-02 Deutsches Krebsforschungszentrum Modified AAV capsid polypeptides
WO2011075838A1 (en) * 2009-12-21 2011-06-30 Audigen Inc. Method of treating or preventing hearing loss
AU2013259166B2 (en) 2012-05-11 2016-03-10 Theragenics Corporation Biocompatible hydrogel treatments for retinal detachment
AU2014244167A1 (en) 2013-03-13 2015-10-08 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
WO2014210586A1 (en) 2013-06-29 2014-12-31 Robert Edward Morris Safety cannula
CN115141258B (zh) 2013-10-11 2025-05-20 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
JP7166168B2 (ja) 2015-07-30 2022-11-07 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列およびその使用
US9782232B1 (en) 2016-04-25 2017-10-10 Novartis Ag Automated intraocular pressure tamponade
EP3558393A4 (en) * 2016-12-22 2020-11-11 Oregon Health & Science University ADENO-ASSOCIATED VIRAL VECTORS
WO2018209154A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (app)-dependence of viruses
WO2019162791A1 (en) 2018-02-22 2019-08-29 Novartis Ag Systems and methods for gas mixing in ocular surgical equipment
SG11202009451VA (en) * 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
CA3114621A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2021154923A2 (en) 2020-01-29 2021-08-05 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
WO2022150634A2 (en) 2021-01-08 2022-07-14 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav

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