IN2014DN08812A - - Google Patents

Info

Publication number
IN2014DN08812A
IN2014DN08812A IN8812DEN2014A IN2014DN08812A IN 2014DN08812 A IN2014DN08812 A IN 2014DN08812A IN 8812DEN2014 A IN8812DEN2014 A IN 8812DEN2014A IN 2014DN08812 A IN2014DN08812 A IN 2014DN08812A
Authority
IN
India
Prior art keywords
disclosed
methods
gene therapy
mediated gene
improved compositions
Prior art date
Application number
Other languages
English (en)
Inventor
Mustafa N Yazicioglu
Federico Mingozzi
Xavier Anguela
Katherine A High
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
Application filed by Philadelphia Children Hospital filed Critical Philadelphia Children Hospital
Publication of IN2014DN08812A publication Critical patent/IN2014DN08812A/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use 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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
IN8812DEN2014 2012-04-18 2013-04-18 IN2014DN08812A (enExample)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261635273P 2012-04-18 2012-04-18
US201361794995P 2013-03-15 2013-03-15
PCT/US2013/037170 WO2013158879A1 (en) 2012-04-18 2013-04-18 Composition and methods for highly efficient gene transfer using aav capsid variants

Publications (1)

Publication Number Publication Date
IN2014DN08812A true IN2014DN08812A (enExample) 2015-05-22

Family

ID=49384070

Family Applications (1)

Application Number Title Priority Date Filing Date
IN8812DEN2014 IN2014DN08812A (enExample) 2012-04-18 2013-04-18

Country Status (26)

Country Link
US (2) US9909142B2 (enExample)
EP (1) EP2839014B1 (enExample)
JP (1) JP6342886B2 (enExample)
KR (1) KR102063483B1 (enExample)
CN (1) CN104487579B (enExample)
AU (1) AU2013249202B2 (enExample)
BR (2) BR122021020419B1 (enExample)
CA (1) CA2870736C (enExample)
CO (1) CO7200251A2 (enExample)
DK (1) DK2839014T3 (enExample)
ES (1) ES2862912T3 (enExample)
HU (1) HUE054087T2 (enExample)
IL (1) IL235102B (enExample)
IN (1) IN2014DN08812A (enExample)
MX (1) MX359518B (enExample)
MY (1) MY172457A (enExample)
NZ (1) NZ701693A (enExample)
PE (1) PE20150163A1 (enExample)
PH (1) PH12014502347B1 (enExample)
PL (1) PL2839014T3 (enExample)
PT (1) PT2839014T (enExample)
RU (1) RU2683497C2 (enExample)
SG (1) SG11201406776TA (enExample)
SI (1) SI2839014T1 (enExample)
WO (1) WO2013158879A1 (enExample)
ZA (1) ZA201407582B (enExample)

Families Citing this family (131)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102994549B (zh) * 2005-04-07 2015-02-11 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
WO2008124724A1 (en) 2007-04-09 2008-10-16 University Of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2012149268A1 (en) 2011-04-29 2012-11-01 Selecta Biociences, Inc. Tolerogenic synthetic nanocarriers for allergy therapy
IN2014DN08812A (enExample) 2012-04-18 2015-05-22 Philadelphia Children Hospital
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US10357483B2 (en) 2013-05-03 2019-07-23 Selecta Biosciences, Inc. Methods comprising dosing combinations for reducing undesired humoral immune responses
CN105247044B (zh) * 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
PL3024498T3 (pl) * 2013-07-22 2020-05-18 The Children's Hospital Of Philadelphia Wariant aav oraz kompozycje, sposoby i zastosowania transferu genów do komórek, narządów i tkanek
CA2930549A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
EP3113787B1 (en) * 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
AU2015311707B2 (en) 2014-09-07 2021-12-09 Selecta Biosciences, Inc. Methods and compositions for attenuating gene expression modulating anti-viral transfer vector immune responses
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
CA2966620A1 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CA2975583A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
CA2967367C (en) 2014-11-14 2023-05-16 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
US20180044698A1 (en) * 2015-02-19 2018-02-15 University Of Florida Research Foundation, Incorporated Site-specific integrating recombinant aav vectors for gene therapy and improved production methods
JP6519080B2 (ja) * 2015-06-22 2019-05-29 ライオン株式会社 繊維製品用抗ウイルス組成物
US10799566B2 (en) 2015-06-23 2020-10-13 The Children's Hospital Of Philadelphia Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
CN114606267A (zh) 2015-09-28 2022-06-10 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
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
CN116196410A (zh) 2015-10-28 2023-06-02 宾夕法尼亚州大学信托人 鞘内施用腺伴随病毒载体用于基因治疗
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
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
KR20250065734A (ko) * 2016-04-16 2025-05-13 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 바큘로바이러스 시스템-생산된 재조합 아데노-연관 바이러스의 생물학적 효력을 증진시키는 방법
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
KR20220108216A (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
TN2019000047A1 (en) * 2016-08-15 2020-07-15 Genzyme Corp Methods for detecting aav
AU2017321488B2 (en) 2016-08-30 2022-10-20 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US11578340B2 (en) 2016-10-13 2023-02-14 University Of Massachusetts AAV capsid designs
KR20190104194A (ko) 2017-01-07 2019-09-06 셀렉타 바이오사이언시즈, 인크. 합성 나노담체에 커플링된 면역억제제의 패턴화된 투여
EP3575398A4 (en) * 2017-01-30 2020-11-11 Nippon Medical School Foundation ADENO-ASSOCIATED VIRUS (AAV) CAPSIDE PROTEIN MUTANT
JP7237843B2 (ja) 2017-02-21 2023-03-13 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． 修飾されたａａｖキャプシドタンパク質およびその使用
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CA3055936A1 (en) 2017-03-11 2018-09-20 Selecta Biosciences, Inc. Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
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
CN117801075A (zh) 2017-03-15 2024-04-02 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
EP4512820A3 (en) 2017-03-30 2025-05-21 The University of Queensland Chimeric molecules and uses thereof
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US12404526B2 (en) 2017-06-07 2025-09-02 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
CN110461368A (zh) 2017-06-30 2019-11-15 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
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
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
US12076369B2 (en) 2017-09-01 2024-09-03 The Frances Crick Institute Limited Immunoregulatory molecules and uses therefor
IL273261B2 (en) 2017-09-20 2025-04-01 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of using them
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
BR112020007157A2 (pt) 2017-10-13 2020-09-24 Selecta Biosciences, Inc. métodos e composições para a atenuação de respostas de igm antivetor de transferência viral
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
MX2020005451A (es) 2017-11-27 2020-08-27 4D Molecular Therapeutics Inc Capsides variantes de virus adenoasociados y uso para inhibir la angiogenesis.
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
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3746101A4 (en) 2018-02-01 2022-03-23 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
CA3092372A1 (en) 2018-02-26 2019-08-29 AnTolRx, Inc. Tolerogenic liposomes and methods of use thereof
IL276859B1 (en) * 2018-02-27 2025-08-01 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
KR20250111395A (ko) * 2018-03-16 2025-07-22 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달
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
SG11202009450SA (en) 2018-04-03 2020-10-29 Stridebio Inc Virus vectors for targeting ophthalmic tissues
BR112020020195A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
KR20210015770A (ko) 2018-04-05 2021-02-10 제네똥 감소된 간 향성을 갖는 AAV9과 AAVrh74의 하이브리드 재조합 아데노-관련 바이러스 세로타입
CN119410636A (zh) 2018-04-27 2025-02-11 分贝治疗公司 肌球蛋白15启动子及其用途
US20210363192A1 (en) * 2018-04-27 2021-11-25 Spark Therapeutics, Inc. Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
MX2020012077A (es) 2018-05-15 2021-03-09 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de la enfermedad de parkinson.
SG11202011191WA (en) * 2018-05-16 2020-12-30 Spark Therapeutics Inc Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same
WO2020018583A1 (en) 2018-07-16 2020-01-23 Selecta Biosciences, Inc. Methods and compositions of otc constructs and vectors
MX2021000638A (es) 2018-07-16 2021-06-23 Selecta Biosciences Inc Métodos y composiciones de construcciones y vectores de acidemia metilmalónica.
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
AR116569A1 (es) 2018-10-01 2021-05-19 Ultragenyx Pharmaceutical Inc Terapia génica para tratar la acidemia propiónica
CA3116331A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
US11702673B2 (en) 2018-10-18 2023-07-18 University Of Florida Research Foundation, Incorporated Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus
WO2020163761A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
WO2020163743A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
EP3931334A1 (en) 2019-02-25 2022-01-05 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
EA202192501A1 (ru) 2019-03-21 2021-12-16 Страйдбайо, Инк. Рекомбинантные аденоассоциированные вирусные векторы
CA3138525A1 (en) 2019-04-28 2020-11-05 Selecta Biosciences, Inc. Methods for treatment of subjects with preexisting immunity to viral transfer vectors
BR112021023594A2 (pt) 2019-05-28 2022-02-08 Selecta Biosciences Inc Métodos e composições para resposta imune de vetor de transferência antiviral atenuada
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
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
BR112022006530A2 (pt) 2019-10-17 2022-07-05 Stridebio Inc Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c
IL292620A (en) * 2019-10-30 2022-07-01 Decibel Therapeutics Inc Compounds and methods for use in dual vector autoperlin arrays for the treatment of structural hearing loss
CN114902051A (zh) * 2020-01-03 2022-08-12 萨勒普塔医疗公司 用于分析aav衣壳蛋白的方法
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
WO2021163322A1 (en) 2020-02-14 2021-08-19 Ultragenyx Pharmaceutical Inc. Gene therapy for treating cdkl5 deficiency disorder
JP2023518415A (ja) 2020-03-19 2023-05-01 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 組換えａａｖにおいてｃａｐおよびｒｅｐ配列の逆パッケージングを低減するための組成物および方法
US20230129893A1 (en) 2020-03-31 2023-04-27 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
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
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
JP2023546116A (ja) 2020-10-15 2023-11-01 エフ． ホフマン－ラ ロシュ アーゲー Ｖａ ｒｎａ転写のための核酸構築物
AR123776A1 (es) 2020-10-15 2023-01-11 Hoffmann La Roche Construcciones de ácido nucleico para la activación simultánea de genes
AU2022206197A1 (en) 2021-01-05 2023-07-13 Selecta Biosciences, Inc. Viral vector dosing protocols
MX2023012509A (es) * 2021-04-23 2024-01-03 Univ Florida Vectores aavrh74 para la terapia génica de distrofias musculares.
WO2023019168A1 (en) 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
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
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
MX2024002200A (es) 2021-08-20 2024-04-29 Biocad Joint Stock Co Metodo para obtener una capside de virus adenoasociado modificada.
WO2023044306A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
US20230140196A1 (en) 2021-10-12 2023-05-04 Selecta Biosciences, Inc. Viral vector dosing protocols
JP2024545594A (ja) 2021-11-14 2024-12-10 カーティザン セラピューティクス，インコーポレーテッド ウイルスベクターでの複数回投薬
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
JP2025509328A (ja) 2022-03-07 2025-04-11 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 改良されたバッチａａｖ産生システムおよび方法
WO2023172624A1 (en) 2022-03-09 2023-09-14 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
JP2025516117A (ja) 2022-04-13 2025-05-27 エフ． ホフマン－ラ ロシュ アーゲー Ａａｖゲノムを決定するための方法
IL316908A (en) * 2022-05-11 2025-01-01 Childrens Hospital Philadelphia Adeno-associated viral vectors for targeting deep brain structures
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
AR129500A1 (es) 2022-06-03 2024-09-04 Hoffmann La Roche Método para producir partículas de aav recombinante
JP2025524619A (ja) 2022-07-14 2025-07-30 エフ． ホフマン－ラ ロシュ アーゲー 組換えａａｖ粒子を産生するための方法
WO2024036324A1 (en) 2022-08-11 2024-02-15 Selecta Biosciences, Inc. Compositions and methods related to immunoglobulin proteases and fusions thereof
CN119730884A (zh) 2022-08-15 2025-03-28 豪夫迈·罗氏有限公司 与重组病毒载体相关的不良反应的预防或减轻
CA3264505A1 (en) 2022-09-12 2024-03-21 Hoffmann La Roche METHOD FOR SEPARING SOLID AND EMPTY VAA PARTICLES
IL322001A (en) 2023-02-07 2025-09-01 Hoffmann La Roche Method for detecting antibodies to anti-AAV particles
AU2024239386A1 (en) 2023-03-21 2025-08-14 F. Hoffmann-La Roche Ag Method for the production of recombinant aav particle preparations
WO2024213596A1 (en) 2023-04-13 2024-10-17 F. Hoffmann-La Roche Ag Improved recombinant polyadenylation signal sequences and use thereof
WO2025057182A1 (en) * 2023-09-15 2025-03-20 Indian Institute Of Technology Kanpur Modified aav vectors for enhanced gene therapy in hemophilia b
WO2025082618A1 (en) 2023-10-20 2025-04-24 Fuse Vectors Aps In vitro parvovirus capsid manufacturing
WO2025168663A1 (en) 2024-02-09 2025-08-14 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral particles

Family Cites Families (13)

* 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
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
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
US6127525A (en) * 1995-02-21 2000-10-03 Cornell Research Foundation, Inc. Chimeric adenoviral coat protein and methods of using same
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (ja) 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
CN102994549B (zh) * 2005-04-07 2015-02-11 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
KR20130040844A (ko) * 2010-03-29 2013-04-24 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 약학적으로 유발된 전이유전자 제거 시스템
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
IN2014DN08812A (enExample) 2012-04-18 2015-05-22 Philadelphia Children Hospital

Also Published As

Publication number Publication date
SG11201406776TA (en) 2015-03-30
BR122021020419B1 (pt) 2023-01-17
NZ701693A (en) 2017-02-24
IL235102B (en) 2019-08-29
CN104487579B (zh) 2022-01-18
HUE054087T2 (hu) 2021-09-28
PE20150163A1 (es) 2015-02-23
PL2839014T3 (pl) 2021-08-23
US11279950B2 (en) 2022-03-22
EP2839014B1 (en) 2020-12-16
EP2839014A1 (en) 2015-02-25
RU2014146159A (ru) 2016-06-10
MX2014012680A (es) 2015-05-11
JP2015514427A (ja) 2015-05-21
MY172457A (en) 2019-11-26
JP6342886B2 (ja) 2018-06-13
WO2013158879A1 (en) 2013-10-24
US9909142B2 (en) 2018-03-06
ES2862912T3 (es) 2021-10-08
DK2839014T3 (da) 2021-03-08
KR102063483B1 (ko) 2020-01-08
KR20150004859A (ko) 2015-01-13
ZA201407582B (en) 2021-05-26
RU2683497C2 (ru) 2019-03-28
HK1207663A1 (en) 2016-02-05
AU2013249202A1 (en) 2014-11-06
EP2839014A4 (en) 2015-12-30
PH12014502347A1 (en) 2014-12-22
PT2839014T (pt) 2021-03-19
US20150065562A1 (en) 2015-03-05
SI2839014T1 (sl) 2021-05-31
US20180245098A1 (en) 2018-08-30
MX359518B (es) 2018-10-01
PH12014502347B1 (en) 2014-12-22
CA2870736C (en) 2021-11-02
CN104487579A (zh) 2015-04-01
CA2870736A1 (en) 2013-10-24
AU2013249202B2 (en) 2018-08-09
BR112014025985A2 (pt) 2017-07-11
IL235102A0 (en) 2014-12-31
CO7200251A2 (es) 2015-02-27

Similar Documents

Publication Publication Date Title
IN2014DN08812A (enExample)
PH12019502292A1 (en) Methods and compositions for treatment of a genetic condition
IL283585B1 (en) Methods and preparations for targeted genetic modification using paired guide RNA
AU2019232913A1 (en) Methods and processes for non-invasive assessment of genetic variations
EP2850189B8 (en) Compositions and methods for modulating gene expression
EP2855680B8 (en) Compositions and methods for silencing gene expression
IN2014DN08481A (enExample)
ZA201409229B (en) Compositions and methods for modulating utrn expression
ZA201409228B (en) Compositions and methods for modulating smn gene family expression
MX356525B (es) Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia.
AU2013225816A8 (en) Compositions and methods for treating Type III Gaucher Disease
GB201222934D0 (en) Particle toughening for improving fracture toughness
MX359769B (es) Metodos para descelularizar huesos.
JO3755B1 (ar) تركيبات تستوستيرون
EP3091999A4 (en) Improved cell compositions and methods for cancer therapy
MX350868B (es) Medicamentos y metodos para tratar cancer.
IL233445A0 (en) Methods and preparations for gene transfer
HK1255578A1 (en) Compositions and methods for cell targeted hpv treatment
AU345349S (en) Swimming googles
IL235676A0 (en) Preparations and methods for regulating smn family gene expression
AU2012903646A0 (en) Therapeutic methods and compositions
AU2012901350A0 (en) Therapeutic methods and compositions
IL235878A0 (en) Preparations and methods for silencing gene expression
UA79072U (en) (4-tert-butylperoxy-4-methylpentyl)methacrylate