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CL2018001522A1 - Focalización de péptidos para dirigir virus adenoasociados (aavs) - Google Patents

Focalización de péptidos para dirigir virus adenoasociados (aavs)

Info

Publication number
CL2018001522A1
CL2018001522A1 CL2018001522A CL2018001522A CL2018001522A1 CL 2018001522 A1 CL2018001522 A1 CL 2018001522A1 CL 2018001522 A CL2018001522 A CL 2018001522A CL 2018001522 A CL2018001522 A CL 2018001522A CL 2018001522 A1 CL2018001522 A1 CL 2018001522A1
Authority
CL
Chile
Prior art keywords
aavs
peptides
direct
focalization
adeno
Prior art date
Application number
CL2018001522A
Other languages
English (en)
Inventor
Benjamin E Deverman
Viviana Gradinaru
Ken Y Chan
Original Assignee
California Inst Of Techn
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 California Inst Of Techn filed Critical California Inst Of Techn
Publication of CL2018001522A1 publication Critical patent/CL2018001522A1/es

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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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Abstract

AQUÍ SE DIVULGAN SECUENCIAS DE PÉPTIDOS CAPACES DE DIRIGIR VIRUS ADENOASOCIADOS (AAV) PARA FOCALIZAR AMBIENTES ESPECÍFICOS, POR EJEMPLO EL SISTEMA NERVIOSO Y EL CORAZÓN, EN UN SUJETO. TAMBIÉN SE DIVULGAN AAVS QUE TIENEN PROTEÍNAS DE LA CÁPSIDE QUE NO OCURREN NATURALMENTE COMPRENDIENDO LAS SECUENCIAS DE PÉPTIDOS DIVULGADAS, Y MÉTODOS PARA UTILIZAR LOS AAVS PARA TRATAR ENFERMEDADES.
CL2018001522A 2015-12-11 2018-06-07 Focalización de péptidos para dirigir virus adenoasociados (aavs) CL2018001522A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562266184P 2015-12-11 2015-12-11
US201662421891P 2016-11-14 2016-11-14

Publications (1)

Publication Number Publication Date
CL2018001522A1 true CL2018001522A1 (es) 2018-12-21

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CL2018001522A CL2018001522A1 (es) 2015-12-11 2018-06-07 Focalización de péptidos para dirigir virus adenoasociados (aavs)

Country Status (18)

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US (2) US11499165B2 (es)
EP (2) EP3875593A1 (es)
JP (1) JP7066619B2 (es)
KR (1) KR102423442B1 (es)
CN (1) CN108699565B (es)
AU (1) AU2016366549B2 (es)
BR (1) BR112018011711A2 (es)
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CL (1) CL2018001522A1 (es)
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Families Citing this family (155)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
SG10202001102XA (en) 2014-11-14 2020-03-30 Voyager Therapeutics Inc Modulatory polynucleotides
SG11201703281RA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP3230441A4 (en) 2014-12-12 2018-10-03 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
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
JP7066635B2 (ja) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド 調節性ポリヌクレオチド
WO2017201258A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
ES2944358T3 (es) 2017-01-30 2023-06-20 Brainvectis Vector de expresión para la colesterol 24-hidrolasa en terapia de ataxias espinocerebelosas de repetición de poliglutamina
MX2019012113A (es) 2017-04-14 2020-09-10 Univ Nat Taiwan Terapia génica para la deficiencia de la aadc.
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US11497576B2 (en) 2017-07-17 2022-11-15 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) * 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
KR20200042935A (ko) * 2017-08-25 2020-04-24 오비드 테라퓨틱스 인크. 재조합 아데노-관련된 벡터들
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
JP7303816B2 (ja) * 2017-10-16 2023-07-05 ビゲネロン ゲゼルシャフト ミット ベシュレンクテル ハフツング Aavベクター
WO2019090000A1 (en) 2017-11-02 2019-05-09 California Institute Of Technology Expression of neuropeptides
US11458129B2 (en) 2017-11-02 2022-10-04 California Institute Of Technology Neurokinin antagonists and uses thereof
US20190202866A1 (en) * 2017-12-22 2019-07-04 William Marsh Rice University Viral nanoparticles and methods of use thereof
CA3093490A1 (en) 2018-03-05 2019-09-12 Children's Medical Center Corporation Compositions and methods for delivering nucleic acids to cochlear and vestibular cells
EP3774853A1 (en) * 2018-03-30 2021-02-17 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
SG11202009450SA (en) 2018-04-03 2020-10-29 Stridebio Inc Virus vectors for targeting ophthalmic tissues
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
US12091435B2 (en) 2018-04-03 2024-09-17 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
WO2019199867A1 (en) * 2018-04-09 2019-10-17 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
EP3773677B1 (en) * 2018-04-12 2025-04-02 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Anti-aging compositions and methods of use
KR20210005889A (ko) 2018-04-27 2021-01-15 로켓 파마슈티컬스, 리미티드 Cns 퇴행에 대한 유전자 요법
EP3793616A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US12319929B2 (en) 2018-05-15 2025-06-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
EP3807404A1 (en) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
CN120843603A (zh) * 2018-07-11 2025-10-28 布里格姆妇女医院 用于跨血脑屏障递送试剂的方法和组合物
EP3826719A1 (en) 2018-07-24 2021-06-02 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
US20210277418A1 (en) 2018-08-03 2021-09-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
CN121160803A (zh) 2018-09-28 2025-12-19 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
CA3115217A1 (en) 2018-10-02 2020-04-09 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
CA3115248A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
US12467064B2 (en) 2018-10-08 2025-11-11 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
JP2022505106A (ja) 2018-10-15 2022-01-14 ボイジャー セラピューティクス インコーポレイテッド バキュロウイルス/Sf9システムにおけるrAAVの大規模産生のための発現ベクター
ES2936254T3 (es) 2018-10-29 2023-03-15 Inst Nat Sante Rech Med Vector de expresión para la colesterol 24-hidrolasa en la terapia de la esclerosis lateral amiotrófica
CN113395975A (zh) * 2018-11-20 2021-09-14 宾夕法尼亚州大学信托人 用于靶向血脑屏障的组合物和方法
US20220064671A1 (en) 2019-01-18 2022-03-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
AU2020214831B2 (en) * 2019-01-30 2025-09-25 The Broad Institute, Inc. Systems for evolved adeno-associated viruses (AAVs) for targeted delivery
CN110437317B (zh) * 2019-01-30 2023-05-02 上海科技大学 具有变异衣壳蛋白的腺相关病毒及其用途
KR20210130158A (ko) * 2019-01-31 2021-10-29 오레곤 헬스 앤드 사이언스 유니버시티 Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법
CA3142948A1 (en) * 2019-02-15 2020-08-20 Allen Institute Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations
TW202102525A (zh) 2019-03-21 2021-01-16 美商史崔德生物公司 重組腺相關病毒載體
KR20210143869A (ko) * 2019-03-28 2021-11-29 더 제너럴 하스피탈 코포레이션 트랜스진 발현을 위한 조작된 아데노-연관 (aav) 벡터
SG11202111249YA (en) * 2019-04-11 2021-11-29 California Inst Of Techn Virus compositions with enhanced specificity in the brain
GB201905301D0 (en) 2019-04-15 2019-05-29 Ospedale San Raffaele Srl Gene therapy
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
WO2020223274A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
WO2020223356A1 (en) * 2019-04-30 2020-11-05 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of pompe disease
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP3972631B1 (en) 2019-05-21 2024-07-10 Institut National de la Santé et de la Recherche Médicale (INSERM) Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome
US20220281922A1 (en) * 2019-08-02 2022-09-08 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP4010465A1 (en) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
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
US20220380413A1 (en) * 2019-08-29 2022-12-01 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
GB201912863D0 (en) * 2019-09-06 2019-10-23 Babraham Inst Novel method
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
JP2022551744A (ja) * 2019-10-17 2022-12-13 ストライドバイオ,インコーポレイテッド Aav導入カセット
US20210162002A1 (en) * 2019-10-17 2021-06-03 The Penn State Research Foundation Regenerating functional neurons for treatment of spinal cord injury and als
AU2020367532A1 (en) * 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
WO2021102107A1 (en) * 2019-11-19 2021-05-27 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders
WO2021102234A1 (en) * 2019-11-22 2021-05-27 The Children's Hospital Of Philadelphia Adeno-associated viral vector variants
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
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
MY209860A (en) 2020-02-13 2025-08-07 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease
AU2021259422A1 (en) * 2020-04-20 2022-12-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
CN112011571A (zh) * 2020-04-26 2020-12-01 辉大(上海)生物科技有限公司 一种用于治疗脊髓性肌萎缩的基因治疗药物
JP2023528580A (ja) * 2020-04-28 2023-07-05 ジェネトン 筋肉及び中枢神経系障害の遺伝子治療のための合成aavカプシドの使用
US20210403946A1 (en) 2020-04-29 2021-12-30 The Broad Institute, Inc. Machine learning accelerated protein engineering through fitness prediction
US20230193316A1 (en) * 2020-05-01 2023-06-22 The Broad Institute, Inc. Engineered central nervous system compositions
MX2022014256A (es) 2020-05-13 2023-01-11 Voyager Therapeutics Inc Redirección del tropismo de las cápsides de aav.
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
GB202010981D0 (en) 2020-07-16 2020-09-02 Ucl Business Ltd Gene therapy for neuromuscular and neuromotor disorders
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
US20230227802A1 (en) 2020-07-27 2023-07-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
US20230285596A1 (en) * 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
GB202011871D0 (en) * 2020-07-30 2020-09-16 Cambridge Entpr Ltd Composition and method
CN116234916A (zh) 2020-08-05 2023-06-06 太空飞船七有限责任公司 Csrp3(富含半胱氨酸和甘氨酸的蛋白质3)基因疗法
US20230295656A1 (en) 2020-08-06 2023-09-21 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
BR112023001852A2 (pt) 2020-08-07 2023-02-23 Spacecraft Seven Llc Terapia gênica com placofilina-2 (pkp2) usando vetor de aav
MX2023001998A (es) 2020-08-17 2023-05-04 Massachusetts Inst Technology Enfoques de terapia con el gen shank3.
BR112023003145A2 (pt) 2020-08-19 2023-05-09 Sarepta Therapeutics Inc Vetores de vírus adenoassociado para tratamento da síndrome de rett
AU2021329527A1 (en) * 2020-08-21 2023-03-23 Capsida, Inc. Adeno-associated virus compositions having preferred expression levels
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
AU2021344607A1 (en) * 2020-09-21 2023-06-01 AskBio Inc. Methods for treating neurological disease
CN113754728B (zh) * 2020-09-30 2022-07-12 广州派真生物技术有限公司 腺相关病毒突变体及其应用
AR123776A1 (es) 2020-10-15 2023-01-11 Hoffmann La Roche Construcciones de ácido nucleico para la activación simultánea de genes
KR102862676B1 (ko) 2020-11-11 2025-09-23 유럽피안 몰레큘러 바이올로지 래보러토리 유전자 치료를 위한 변형된 바이러스 입자
US20240024507A1 (en) 2020-12-01 2024-01-25 The Trustees Of The University Of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
JP2024511808A (ja) * 2021-03-29 2024-03-15 南京大学 ウイルスベクターに基づくrna送達システム及びその使用
WO2022221421A2 (en) * 2021-04-13 2022-10-20 Capsida, Inc. Aav compositions with high brain expression for treating mucopolysaccharidosis ii
JP2024515626A (ja) 2021-04-16 2024-04-10 アスクレピオス バイオファーマシューティカル, インコーポレイテッド 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドaavビリオン
US20240207452A1 (en) 2021-04-23 2024-06-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
CA3224488A1 (en) 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
WO2023049710A1 (en) * 2021-09-21 2023-03-30 California Institute Of Technology Viral compositions for targeting the nervous system and the lung
AU2022358779A1 (en) 2021-10-08 2024-04-18 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
CN118043039A (zh) 2021-10-25 2024-05-14 诺华股份有限公司 用于改善腺相关病毒(aav)递送的方法
US20250001012A1 (en) * 2021-11-02 2025-01-02 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4442830A4 (en) 2021-11-29 2025-04-30 Shanghai Regenelead Therapies Co., Ltd. AADC/GDNF polynucleotide and use thereof in the treatment of Parkinson's disease
CN114107231B (zh) * 2021-12-13 2023-08-18 重庆大学 实现全脑突触后神经元胞体标记的重组腺相关病毒及其应用
EP4469585A2 (en) 2022-01-25 2024-12-04 Voyager Therapeutics, Inc. Baculovirus expression system
CN116554278A (zh) * 2022-01-30 2023-08-08 上海玮美基因科技有限责任公司 变异型腺相关病毒及其在疾病治疗中的应用
CN118660913A (zh) 2022-02-21 2024-09-17 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
EP4482970A1 (en) 2022-02-23 2025-01-01 Massachusetts Institute of Technology Methods for upregulating shank3 expression
AU2023257298A1 (en) * 2022-04-22 2024-11-14 The Children's Hospital Of Philadelphia Human ependyma-specific promoter and uses thereof
WO2023240158A2 (en) * 2022-06-07 2023-12-14 Oregon Health & Science University Mutant adeno-associated virus (aav) capsids
WO2023240236A1 (en) * 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
TW202421787A (zh) 2022-09-06 2024-06-01 美商特納亞治療股份有限公司 保護心臟之心臟病療法
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
GB2622629A (en) 2022-09-23 2024-03-27 Sania Rx Ltd Method
WO2024100145A1 (en) 2022-11-08 2024-05-16 Ospedale San Raffaele S.R.L. Polynucleotide and vector
WO2024175604A1 (en) 2023-02-21 2024-08-29 Institut National de la Santé et de la Recherche Médicale Methods and pharmaceutical composition for the treatment of sanfilippo syndrome type iiib
CN120936709A (zh) * 2023-03-09 2025-11-11 新加坡科技研究局 经工程化以增强组织转导和特异性的腺相关病毒的新组合物
AU2024234833A1 (en) 2023-03-10 2025-10-23 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN121219020A (zh) 2023-03-22 2025-12-26 圣拉斐尔医院有限责任公司 基因疗法
AU2024242380A1 (en) * 2023-03-31 2025-11-06 Exegenesis Bio Singapore Pte. Ltd. Variant aav9 capsid proteins and uses thereof
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024229389A1 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2024229425A1 (en) * 2023-05-04 2024-11-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024233422A1 (en) 2023-05-05 2024-11-14 Massachusetts Institute Of Technology Shank3 gene therapy approaches
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
WO2025021839A1 (en) 2023-07-25 2025-01-30 Institut National de la Santé et de la Recherche Médicale Method to treat metabolic disorders
WO2025032158A1 (en) 2023-08-08 2025-02-13 Institut National de la Santé et de la Recherche Médicale Method to treat tauopathies
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)
CN117264912A (zh) * 2023-09-25 2023-12-22 中国科学院深圳先进技术研究院 一种重组腺相关病毒颗粒、重组腺相关病毒载体系统及其应用
WO2025073890A1 (en) 2023-10-06 2025-04-10 Institut National de la Santé et de la Recherche Médicale Method to capture circulating tumor extracellular vesicles
GB202316264D0 (en) 2023-10-24 2023-12-06 Ospendale San Raffaele S R L Gene therapy
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
WO2025106874A1 (en) * 2023-11-15 2025-05-22 Genzyme Corporation Methods of treating neurodegenerative disorders
WO2025157848A1 (en) 2024-01-23 2025-07-31 Institut National de la Santé et de la Recherche Médicale Methods for the intravenous treatment of sanfilippo syndrome type iiib
WO2025171227A1 (en) 2024-02-08 2025-08-14 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025186293A1 (en) 2024-03-06 2025-09-12 Institut National de la Santé et de la Recherche Médicale Chmp2b mutants for use for treating infection
GB202406331D0 (en) 2024-05-07 2024-06-19 Sania Rx Ltd Capsid

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5919676A (en) 1993-06-24 1999-07-06 Advec, Inc. Adenoviral vector system comprising Cre-loxP recombination
US6379943B1 (en) 1999-03-05 2002-04-30 Merck & Co., Inc. High-efficiency Cre/loxp based system for construction of adenovirus vectors
US7279313B2 (en) 1995-09-15 2007-10-09 Centelion Circular DNA molecule having a conditional origin of replication, process for their preparation and their use in gene therapy
WO1997032481A1 (en) 1996-03-07 1997-09-12 The Regents Of The University Of California Helper-free, totally defective adenovirus for gene therapy
EP0950111A1 (en) 1996-09-06 1999-10-20 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
DE19933719A1 (de) 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
AU3459701A (en) 2000-01-26 2001-08-07 Chiron Corporation Recombinant aav packaging systems
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
US6410271B1 (en) 2000-06-23 2002-06-25 Genetastix Corporation Generation of highly diverse library of expression vectors via homologous recombination in yeast
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
US9233131B2 (en) * 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP3211085B1 (en) 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
WO2006099615A2 (en) 2005-03-16 2006-09-21 The Johns Hopkins University Adenoviral fiber exchange shuttle system
JP5364903B2 (ja) 2006-06-21 2013-12-11 ユニキュアー アイピー ビー.ブイ. 昆虫細胞におけるaavの生成に有用なaav−rep78の翻訳の改変型開始コドンを有するベクター
WO2008103993A2 (en) 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions and methods for treating glycogen storage diseases
CA3002933A1 (en) 2007-07-14 2009-01-22 Beverly L. Davidson Methods and compositions for treating brain diseases
US9018138B2 (en) 2007-08-16 2015-04-28 The Johns Hopkins University Compositions and methods for generating and screening adenoviral libraries
WO2010019619A1 (en) 2008-08-11 2010-02-18 The Board Of Trustees Of The Leland Stanford Junior Univeristy Method and composition for controlling gene expression
ES2605305T3 (es) 2010-04-23 2017-03-13 University Of Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
JP6091435B2 (ja) 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達
SMT202000386T1 (it) 2011-04-22 2020-09-10 Univ California Virioni di virus adeno-associato con capside variante e relativi metodi di utilizzo
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
CN105530917B (zh) 2013-09-19 2020-06-05 扬森疫苗与预防公司 改进的腺病毒配制品
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
SG10201912977RA (en) * 2014-05-02 2020-02-27 Genzyme Corp Aav vectors for retinal and cns gene therapy
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
TWI897844B (zh) 2017-05-05 2025-09-21 美商航海家醫療公司 調節多核苷酸
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV

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