CL2014002198A1 - Composiciones de vector de virus adenoasociado (aav) y métodos para transferencia de genes a células, órganos y tejidos - Google Patents

Composiciones de vector de virus adenoasociado (aav) y métodos para transferencia de genes a células, órganos y tejidos

Info

Publication number
CL2014002198A1
CL2014002198A1 CL2014002198A CL2014002198A CL2014002198A1 CL 2014002198 A1 CL2014002198 A1 CL 2014002198A1 CL 2014002198 A CL2014002198 A CL 2014002198A CL 2014002198 A CL2014002198 A CL 2014002198A CL 2014002198 A1 CL2014002198 A1 CL 2014002198A1
Authority
CL
Chile
Prior art keywords
aav
adeno
organs
tissues
cells
Prior art date
Application number
CL2014002198A
Other languages
English (en)
Inventor
Katherine A High
Federico Mingozzi
Junwei Sun
Philip Johnson
Original Assignee
Philadelphia Children Hospital
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=48984822&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CL2014002198(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Philadelphia Children Hospital filed Critical Philadelphia Children Hospital
Publication of CL2014002198A1 publication Critical patent/CL2014002198A1/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CL2014002198A 2012-02-17 2014-08-18 Composiciones de vector de virus adenoasociado (aav) y métodos para transferencia de genes a células, órganos y tejidos CL2014002198A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201261600415P 2012-02-17 2012-02-17

Publications (1)

Publication Number Publication Date
CL2014002198A1 true CL2014002198A1 (es) 2015-03-13

Family

ID=48984822

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2014002198A CL2014002198A1 (es) 2012-02-17 2014-08-18 Composiciones de vector de virus adenoasociado (aav) y métodos para transferencia de genes a células, órganos y tejidos

Country Status (16)

Country Link
US (2) US20150111955A1 (es)
EP (1) EP2814958B1 (es)
JP (2) JP6373763B2 (es)
KR (1) KR102057540B1 (es)
CN (1) CN104520428B (es)
AU (1) AU2013221212B2 (es)
BR (1) BR112014020325A2 (es)
CA (1) CA2864879C (es)
CL (1) CL2014002198A1 (es)
ES (1) ES2752191T3 (es)
IL (1) IL234148B (es)
MX (1) MX367100B (es)
RU (1) RU2653444C2 (es)
SG (1) SG11201404956PA (es)
WO (1) WO2013123503A1 (es)
ZA (1) ZA201406065B (es)

Families Citing this family (190)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
MX374963B (es) 2011-04-29 2025-03-06 Selecta Biosciences Inc Nanoportadores sintéticos tolerogénicos para reducir las respuestas de anticuerpos.
HK1202434A1 (en) 2011-11-22 2015-10-02 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
US9539307B2 (en) 2012-09-17 2017-01-10 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
HK1220488A1 (zh) 2013-03-15 2017-05-05 The Children's Hospital Of Philadelphia 含有填充者/填充物多核苷酸序列的载体及其使用方法
BR122020023215B1 (pt) 2013-05-03 2022-11-22 Selecta Biosciences, Inc Composição e kit de nanocarreadores sintéticos tolerogênicos para reduzir ou prevenir anafilaxia em resposta a um antígeno não alergênico
IL297919A (en) * 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
US9725719B2 (en) 2013-11-05 2017-08-08 The Research Institute At Nationwide Children's Hospital Compositions and methods for inhibiting NF-κB and SOD-1 to treat amyotrophic lateral sclerosis
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
CA2942515C (en) 2014-03-18 2025-12-09 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
PL3628334T3 (pl) * 2014-03-21 2023-12-18 Genzyme Corporation Terapia genowa w retinopatii barwnikowej
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
CN105396143B (zh) * 2014-09-01 2020-06-26 江苏命码生物科技有限公司 埃博拉病毒特异性的miRNA以及通过miRNA抑制埃博拉病毒的方法
JP7242180B2 (ja) * 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
ES2799515T3 (es) 2015-05-22 2020-12-18 Dmitry Dmitrievich Genkin ADN extracelular como una diana terapéutica en la neurodegeneración
MX390848B (es) 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
CN107921067A (zh) * 2015-07-10 2018-04-17 杜克大学 用于治疗疼痛的方法
IL258005B2 (en) 2015-09-17 2025-01-01 Res Inst Nationwide Childrens Hospital Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2
TW202446416A (zh) 2015-09-24 2024-12-01 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
ES2879400T3 (es) * 2015-10-21 2021-11-22 Us Health ADNc de ATP7A de tamaño reducido con codones optimizados y usos para el tratamiento de trastornos del transporte de cobre
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EA202190827A1 (ru) 2015-11-13 2021-11-30 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
CA3005565A1 (en) 2015-11-13 2017-05-18 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
KR102877920B1 (ko) 2015-11-16 2025-10-30 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
EP3384015A4 (en) * 2015-12-01 2019-05-29 Spark Therapeutics, Inc. SCALABLE PROCESS FOR THE PREPARATION OF A RECOMBINANT ADENO ASSOCIATED VIRAL VECTOR IN A SERUM-FREE SUSPENSION CELL CULTURE SYSTEM SUITABLE FOR THE CLINICAL APPLICATION
EP3384035A4 (en) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES
AU2016366560A1 (en) * 2015-12-11 2018-06-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
WO2017173043A1 (en) * 2016-03-30 2017-10-05 Spark Therapeutics, Inc. Cell line for recombinant protein and/or viral vector production
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
LT3442600T (lt) * 2016-04-15 2024-05-27 Research Institute At Nationwide Children's Hospital Su adeno virusu susijusio viruso vektoriaus iš β-sarkoglikano ir mikro- rnr-29 pristatymas ir raumenų distrofijos gydymas
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11364308B2 (en) 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
IL305149A (en) 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
CN106344932B (zh) * 2016-08-26 2019-12-27 首都医科大学附属北京同仁医院 一种用于提高视网膜退行性疾病基因治疗效果的组合物及其应用
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
WO2018082093A1 (en) * 2016-11-07 2018-05-11 Wuhan Institute Of Virology, Chinese Academy Of Sciences Anterograde monosynaptic transneuronal tracer system
BR112019013245A2 (pt) 2016-12-30 2020-02-11 The Trustees Of The University Of Pennsylvania Terapia gênica para tratamento da doença de wilson
JP7215716B2 (ja) 2017-01-13 2023-01-31 学校法人自治医科大学 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのaavベクター
JP2020510648A (ja) 2017-02-20 2020-04-09 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 家族性高コレステロール血症を処置するための遺伝子治療
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
EP3589307B1 (en) * 2017-02-28 2022-02-16 The Penn State Research Foundation Regenerating functional neurons for treatment of neural injury caused by disruption of blood flow
RU2019130004A (ru) * 2017-03-01 2021-04-01 Зе Трастис Оф Зе Юниверсити Оф Пеннсильвания Генная терапия при глазных заболеваниях
ES3036482T3 (en) 2017-03-11 2025-09-19 Cartesian Therapeutics Inc Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
EP3596112A2 (en) 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
JP7676111B2 (ja) * 2017-03-17 2025-05-14 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達
MX2019011772A (es) 2017-04-03 2020-01-09 Encoded Therapeutics Inc Expresion transgenica selectiva de tejidos.
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
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
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
AU2018269050A1 (en) * 2017-05-19 2020-01-16 Encoded Therapeutics, Inc. High activity regulatory elements
CN110945127B (zh) 2017-05-22 2024-07-12 武田药品工业株式会社 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US20200164008A1 (en) * 2017-07-17 2020-05-28 Spark Therapeutics, Inc. Apheresis methods and uses
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
AU2018329741B2 (en) 2017-09-08 2025-02-20 Poseida Therapeutics, Inc. Compositions and methods for chimeric ligand receptor (CLR)-mediated conditional gene expression
RU2770922C2 (ru) 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
CN111448321A (zh) 2017-09-22 2020-07-24 马萨诸塞大学 Sod1双表达载体及其用途
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
EP3697908A1 (en) 2017-10-16 2020-08-26 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
CN107760719B (zh) * 2017-10-24 2020-09-22 北京领柯生物科技有限公司 柯萨奇病毒在过继免疫基因递送系统中的应用
CN111770999A (zh) 2017-11-27 2020-10-13 4D分子治疗有限公司 腺相关病毒变体衣壳和用于抑制血管生成的应用
EP3717505A4 (en) 2017-12-01 2021-12-01 Encoded Therapeutics, Inc. GENENICALLY MODIFIED DNA-BINDING PROTEINS
EP3728568A4 (en) 2017-12-19 2021-10-06 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR DELIVERING VIRAL VECTORS BY THE BLOOD-BRAIN BARRIER
CA3086046C (en) 2017-12-29 2023-02-21 Helixmith Co., Ltd. Adeno-associated virus (aav) vector having hybrid hgf gene introduced thereto
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
AR114350A1 (es) 2018-01-31 2020-08-26 Res Institute At Nationwide Children’S Hospital Terapia genética para la distrofia muscular de cinturas del tipo 2c
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド F8遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
MA51353B1 (fr) 2018-04-05 2022-09-30 Univ Sorbonne Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
TW202016314A (zh) 2018-05-09 2020-05-01 美商拜奧馬林製藥公司 治療苯丙酮尿症之方法
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
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
WO2019236995A1 (en) 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
MY208145A (en) 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
US12091675B2 (en) 2018-07-16 2024-09-17 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
US20220010333A1 (en) * 2018-09-06 2022-01-13 The Regents Of The University Of California Rna and dna base editing via engineered adar recruitment
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US20220089670A1 (en) * 2018-12-28 2022-03-24 University Of Rochester Gene Therapy for BEST1 Dominant Mutations
DK3906066T5 (da) * 2019-01-04 2024-08-05 Ultragenyx Pharmaceutical Inc Geneterapikonstruktioner til behandling af wilsons sygdom
TWI851647B (zh) 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
BR112021015751A2 (pt) * 2019-02-12 2022-02-08 Spacecraft Seven Llc Vetores de terapia genética para tratamento da doença de danon
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
AU2020229340A1 (en) 2019-02-26 2021-09-16 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy
SG11202109362XA (en) 2019-02-28 2021-09-29 Encoded Therapeutics Inc Compositions and methods for treating laminopathies
CN109999182B (zh) * 2019-03-29 2023-03-07 四川大学华西医院 凝血因子在制备抗肿瘤药物中的应用
MX2021014226A (es) * 2019-05-20 2022-01-06 BioNTech SE Arn terapeutico para cancer de ovario.
PL3976798T3 (pl) 2019-05-29 2026-04-07 Encoded Therapeutics, Inc. Kompozycje i sposoby selektywnej regulacji genów
AU2020287627A1 (en) 2019-06-04 2021-11-18 Selecta Biosciences, Inc. Formulations and doses of PEGylated uricase
CA3140685A1 (en) * 2019-06-10 2020-12-17 Vijay G. Sankaran Compositions and methods for the treatment of dba using gata1 gene therapy
RS65421B1 (sr) 2019-08-21 2024-05-31 Res Inst Nationwide Childrens Hospital Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije
EP4022057A1 (en) 2019-08-27 2022-07-06 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of disorders associated with repetitive dna
US20220347318A1 (en) * 2019-09-25 2022-11-03 University Of Utah Research Foundation Methods and compositions for the expression of constitutively active rap1a from a vmd2 promoter
AU2020356962A1 (en) * 2019-10-02 2022-04-14 Sangamo Therapeutics, Inc. Zinc finger protein transcription factors for repressing alpha-synuclein expression
KR20220130093A (ko) * 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
TWI902721B (zh) * 2019-11-04 2025-11-01 美商迪賽博治療公司 前庭支持細胞啟動子及其用途
WO2021096927A1 (en) * 2019-11-13 2021-05-20 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Compositions and uses thereof for treating, prognosing and diagnosing pulmonary hypertension
JP2023503850A (ja) 2019-11-14 2023-02-01 バイオマリン ファーマシューティカル インコーポレイテッド 肝臓特異的遺伝子療法ベクターによる遺伝性血管浮腫の治療
US20220409745A1 (en) * 2019-11-19 2022-12-29 The Regents Of The University Of California Compositions and methods of using engineered fusion proteins that bind g4c2 human repeats
IL293012A (en) * 2019-11-20 2022-07-01 Bavarian Nordic As Medicinal uses of 4-1bbl modified ankara virus vaccinia ((recombinant mva
AU2020388637A1 (en) * 2019-11-21 2022-07-07 Remedium Bio, Inc. Growth factor restoration
AU2020395327A1 (en) * 2019-12-05 2022-07-21 Cornell University Stabilization of retromer for the treatment of Alzheimer's disease and other neurodegenerative disorders
WO2021119538A1 (en) * 2019-12-11 2021-06-17 Myeloid Therapeutics, Inc. Therapeutic cell compositions and methods for manufacture and uses thereof
JP7846007B2 (ja) * 2019-12-18 2026-04-14 ノバルティス アーゲー 異常ヘモグロビン症の治療用組成物及び方法
GB201918879D0 (en) * 2019-12-19 2020-02-05 Ucl Business Ltd Treatment of chronic pain
CN113025618B (zh) * 2019-12-24 2024-02-06 朗信启昇(苏州)生物制药有限公司 一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用
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
JP7703543B2 (ja) * 2020-01-17 2025-07-07 ザ ユナイテッド ステイツ オブ アメリカ アズ リプリゼンテッド バイ ザ セクレタリー、デパートメント オブ ヘルス アンド ヒューマン サービシーズ Crx常染色体優性網膜症の処置のための遺伝子治療
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
EP4114952A4 (en) * 2020-03-05 2024-05-08 Board of Regents of the University of Nebraska Crispr/cas9 system for multistrain hiv-1 treatment
WO2021183755A1 (en) * 2020-03-11 2021-09-16 City Of Hope Dual-targeting chimeric antigen receptor modified t cells comprising il-13 and chlorotoxin for cancer treatment
TW202144575A (zh) 2020-04-03 2021-12-01 美商拜奧馬林製藥公司 使用aav及治療調配物之苯酮尿症治療
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
US12391745B2 (en) 2020-06-15 2025-08-19 Sarepta Therapeutics, Inc. Adeno-associated virus antibodies and fragments thereof
US20230256117A1 (en) * 2020-06-19 2023-08-17 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
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
CN111500635B (zh) * 2020-07-02 2020-10-09 北京大学第三医院(北京大学第三临床医学院) 一种包含携带核酸分子的载体的试剂盒
CN116333984A (zh) * 2020-07-14 2023-06-27 广州泛恩生物科技有限公司 基于galnt2阳性t细胞的用于杀伤肿瘤的tcr-t细胞及其制备方法和应用
CN111876432B (zh) * 2020-07-29 2022-10-28 舒泰神(北京)生物制药股份有限公司 一组肝靶向新型腺相关病毒的获得及其应用
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
US20220096606A1 (en) 2020-09-09 2022-03-31 Vertex Pharmaceuticals Incorporated Compositions and Methods for Treatment of Duchenne Muscular Dystrophy
US12071633B2 (en) 2020-10-13 2024-08-27 Kriya Therapeutics, Inc. Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
US20220110970A1 (en) * 2020-10-14 2022-04-14 The Texas A&M University System Engineered Extracellular Vesicle Delivery Systems and Uses Thereof
WO2022098933A1 (en) 2020-11-06 2022-05-12 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of dm1 with slucas9 and sacas9
KR102803305B1 (ko) * 2020-11-25 2025-05-07 연세대학교 산학협력단 트랜스-스플라이싱 아데노-연관 바이러스 벡터를 포함하는 프라임에디팅용 조성물
US20220238230A1 (en) * 2021-01-15 2022-07-28 Trustees Of Tufts College Methods for modulating the function of biological regulatory networks in health and disease by exploiting their memory properties
TW202302848A (zh) 2021-02-26 2023-01-16 美商維泰克斯製藥公司 以crispr/sacas9治療第1型肌強直性營養不良之組合物及方法
EP4298221A1 (en) 2021-02-26 2024-01-03 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/slucas9
US20240165271A1 (en) 2021-03-26 2024-05-23 The Board Of Regents Of The University Of Texas System Nucleotide editing to reframe dmd transcripts by base editing and prime editing
WO2022229851A1 (en) 2021-04-26 2022-11-03 Crispr Therapeutics Ag Compositions and methods for using slucas9 scaffold sequences
WO2022234519A1 (en) 2021-05-05 2022-11-10 Crispr Therapeutics Ag Compositions and methods for using sacas9 scaffold sequences
US20240252679A1 (en) 2021-05-28 2024-08-01 Shanghai Regenelead Therapies Co., Ltd. Recombinant adeno-associated virus having variant capsid, and application thereof
WO2023280175A1 (en) * 2021-07-06 2023-01-12 National Institute Of Biological Sciences, Beijing Methods for treating complex i deficiencies or cancers by modulating gro3p biosynthesis
WO2023018637A1 (en) 2021-08-09 2023-02-16 Vertex Pharmaceuticals Incorporated Gene editing of regulatory elements
WO2023039444A2 (en) 2021-09-08 2023-03-16 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exon 51 for treatment of duchenne muscular dystrophy
US20240408240A1 (en) 2021-10-01 2024-12-12 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
JP2024540987A (ja) 2021-10-21 2024-11-06 バーテックス ファーマシューティカルズ インコーポレイテッド 低免疫細胞
TW202325850A (zh) 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc、gdnf多核苷酸及其用於治療帕金森病
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
GB202202628D0 (en) * 2022-02-25 2022-04-13 Babraham Inst Novel use and method comprising IL-2 and a tissue- or organic-specific targeting moiety
WO2023172927A1 (en) 2022-03-08 2023-09-14 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exon 44, 50, and 53 for treatment of duchenne muscular dystrophy
WO2023172926A1 (en) 2022-03-08 2023-09-14 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exons for treatment of duchenne muscular dystrophy
AU2023255614A1 (en) 2022-04-18 2024-10-24 Vertex Pharmaceuticals Incorporated Compositions and methods for enhancing aav therapy and decreasing tropism of aav to the liver.
CN118460618A (zh) * 2022-04-19 2024-08-09 康霖生物科技(杭州)有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体
EP4558634A1 (en) 2022-07-18 2025-05-28 Vertex Pharmaceuticals Incorporated Tandem guide rnas (tg-rnas) and their use in genome editing
CN120380156A (zh) 2022-09-22 2025-07-25 迪纳柯公司 用aav基因治疗载体治疗心肌病
KR20260019521A (ko) 2023-05-25 2026-02-10 주노 쎄러퓨티크스 인코퍼레이티드 Aav 정제 방법
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
WO2025082618A1 (en) 2023-10-20 2025-04-24 Fuse Vectors Aps In vitro parvovirus capsid manufacturing
WO2025106374A1 (en) 2023-11-13 2025-05-22 Juno Therapeutics, Inc. Aav production method
WO2025214477A1 (en) 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
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
MX359371B (es) * 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US7588772B2 (en) * 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EP2561073B1 (en) * 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US9175280B2 (en) * 2010-10-12 2015-11-03 Sangamo Biosciences, Inc. Methods and compositions for treating hemophilia B
US9434928B2 (en) * 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
MX390848B (es) * 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.

Also Published As

Publication number Publication date
BR112014020325A2 (pt) 2017-08-08
SG11201404956PA (en) 2014-09-26
CA2864879A1 (en) 2013-08-22
JP2018121655A (ja) 2018-08-09
ES2752191T3 (es) 2020-04-03
RU2653444C2 (ru) 2018-05-08
KR102057540B1 (ko) 2019-12-19
MX367100B (es) 2019-08-05
MX2014009975A (es) 2015-02-20
US20150111955A1 (en) 2015-04-23
AU2013221212A1 (en) 2014-09-04
CN104520428B (zh) 2018-09-21
EP2814958A1 (en) 2014-12-24
CA2864879C (en) 2021-07-20
AU2013221212B2 (en) 2018-08-09
ZA201406065B (en) 2015-12-23
JP6373763B2 (ja) 2018-08-22
JP2015509711A (ja) 2015-04-02
EP2814958A4 (en) 2015-11-11
WO2013123503A1 (en) 2013-08-22
KR20150005521A (ko) 2015-01-14
US11939590B1 (en) 2024-03-26
IL234148B (en) 2020-01-30
CN104520428A (zh) 2015-04-15
RU2014137450A (ru) 2016-04-10
EP2814958B1 (en) 2019-08-07
HK1205191A1 (en) 2015-12-11

Similar Documents

Publication Publication Date Title
CL2014002198A1 (es) Composiciones de vector de virus adenoasociado (aav) y métodos para transferencia de genes a células, órganos y tejidos
IL289249A (en) Modified Aav and preparations, methods and uses for gene transfer to cells, organs and tissues
FIC20250001I1 (en) Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
EP2870237A4 (en) MODIFIED ADENOASSOZED VIRUS VECTOR COMPOSITIONS
DK2857420T3 (da) Målvævsspecifikt antigenbindende molekyle
EP2536345A4 (en) Prosthetic heart valve packaging system
IL232106B (en) A multi- cell battery assembly
BR112013033350A2 (pt) imunocitocina, ácido nucleico, vetor, célula hospedeira e composição farmacêutica
BR112014028631A2 (pt) composições e métodos para modulação da expressão da família de genes da hemoglobina
DK2895102T3 (da) Forbedret virtuel skinne
CO6791570A2 (es) Nueva cepa del prrsv europea
DE112011103303B8 (de) Ladeanschluss
EP2834647A4 (en) VIRTUAL PATTERN QUESTIONS
LT2678918T (lt) Virtuali jėgainė
GB2504023B (en) Virtual BIOS
ZA201305940B (en) Hair styling composition
DK3520828T3 (da) Decellulariseret muskelmatrix
DK2709656T3 (da) Proteinmatrix-vaccinesammensætninger omfattende polykationer
ZA201402011B (en) Hair styling composition
EP2686335A4 (en) DECORINAL COMPOSITIONS AND THEIR USE
ES1075488Y (es) Cantonera para apilar placas
GB2497654B (en) Sunless tanning compositions with adjuvants comprising sulfur comprising moieties
TH1501000544A (th) องค์ประกอบยางธรรมชาติที่มีสมบัติดีขึ้น (Improved natural rubber composition)
FR2984745B3 (fr) Compositions comprenant du cetilistat
EM19403880003S (es) Secadores de pelo (parte de -)