BR112022015979A2 - Vetores de terapia genética para tratamento de doenças cardíacas - Google Patents

Vetores de terapia genética para tratamento de doenças cardíacas

Info

Publication number
BR112022015979A2
BR112022015979A2 BR112022015979A BR112022015979A BR112022015979A2 BR 112022015979 A2 BR112022015979 A2 BR 112022015979A2 BR 112022015979 A BR112022015979 A BR 112022015979A BR 112022015979 A BR112022015979 A BR 112022015979A BR 112022015979 A2 BR112022015979 A2 BR 112022015979A2
Authority
BR
Brazil
Prior art keywords
treatment
gene therapy
therapy vectors
heart disease
heart diseases
Prior art date
Application number
BR112022015979A
Other languages
English (en)
Inventor
Lombardi Laura
Original Assignee
Tenaya Therapeutics Inc
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 Tenaya Therapeutics Inc filed Critical Tenaya Therapeutics Inc
Priority claimed from PCT/US2021/017699 external-priority patent/WO2021163357A2/en
Publication of BR112022015979A2 publication Critical patent/BR112022015979A2/pt

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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657—Cardiomyocytes; Heart cells
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0375—Animal model for cardiovascular diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Cardiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cell Biology (AREA)
  • Environmental Sciences (AREA)
  • Rheumatology (AREA)
  • Transplantation (AREA)

Abstract

VETORES DE TERAPIA GENÉTICA PARA TRATAMENTO DE DOENÇAS CARDÍACAS. A presente divulgação fornece métodos e composições úteis para o tratamento ou prevenção de doenças cardíacas. Em particular, a presente divulgação fornece um vetor compreendendo um promotor de troponina T modificado operativamente ligado a um produto gênico terapêutico para o tratamento ou prevenção de doenças cardíacas, por exemplo, cardiomiopatia. O produto do gene pode ser MYBPC3. A divulgação também fornece viriãos de vírus adeno-associados recombinantes (rAAV), genomas virais de rAAV e cassetes de expressão e suas composições farmacêuticas. A divulgação fornece ainda métodos para tratar uma doença ou distúrbio, como doença cardíaca.
BR112022015979A 2020-02-13 2021-02-11 Vetores de terapia genética para tratamento de doenças cardíacas BR112022015979A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062976160P 2020-02-13 2020-02-13
US202063047633P 2020-07-02 2020-07-02
PCT/US2021/017699 WO2021163357A2 (en) 2020-02-13 2021-02-11 Gene therapy vectors for treating heart disease

Publications (1)

Publication Number Publication Date
BR112022015979A2 true BR112022015979A2 (pt) 2022-10-11

Family

ID=77273691

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022015979A BR112022015979A2 (pt) 2020-02-13 2021-02-11 Vetores de terapia genética para tratamento de doenças cardíacas

Country Status (11)

Country Link
US (5) US11129908B2 (pt)
EP (1) EP4103214A2 (pt)
JP (1) JP2023513749A (pt)
KR (1) KR20220139956A (pt)
CN (1) CN116096394A (pt)
AU (1) AU2021218411A1 (pt)
BR (1) BR112022015979A2 (pt)
CA (1) CA3171166A1 (pt)
CO (1) CO2022012917A2 (pt)
IL (1) IL295529A (pt)
MX (1) MX2022009883A (pt)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2021218411A1 (en) 2020-02-13 2022-10-06 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
KR20240122827A (ko) * 2021-12-10 2024-08-13 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 바이러스 벡터로 mybpc3 관련 비대형 심근병증을 치료하는 방법 및 조성물
TW202404993A (zh) 2022-04-11 2024-02-01 美商特納亞治療股份有限公司 具經工程化蛋白殼之腺相關病毒
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
WO2024064863A2 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors
WO2024064856A1 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of cardiomyopathy with aav gene therapy vectors
WO2024163571A1 (en) * 2023-02-01 2024-08-08 Board Of Regents, The University Of Texas System MESSENGER RNA ENGINEERED THERAPEUTICS FOR TREATING GENETIC DISORDERS (MeET)

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5266488A (en) 1988-04-06 1993-11-30 The Regents Of The University Of California Troponin T gene promoter and derivatives thereof
DE60236364D1 (en) * 2001-10-11 2010-06-24 Angeletti P Ist Richerche Bio Hepatitis-c-virus-impfstoff
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
NZ555830A (en) 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
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
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
CA2837322C (en) 2011-05-26 2023-03-21 University Of Washington Cell and gene based methods to improve cardiac function
CN103987854A (zh) 2011-07-21 2014-08-13 小利兰·斯坦福大学托管委员会 来自患者的诱导性多能干细胞的心肌细胞及其使用方法
US8933048B2 (en) 2011-09-21 2015-01-13 Case Western Reserve University Methods of treating cardiomyopathy
US20130136729A1 (en) 2011-11-11 2013-05-30 University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors
WO2014028762A1 (en) * 2012-08-15 2014-02-20 University Of Virginia Patent Foundation Compositions and methods for treating peripheral arterial disease
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3209311B1 (en) 2014-10-21 2024-03-06 University of Massachusetts Recombinant aav variants and uses thereof
KR102684387B1 (ko) 2015-01-16 2024-07-11 유니버시티 오브 워싱톤 신규한 마이크로-디스트로핀 및 관련된 사용 방법
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
WO2016168728A2 (en) 2015-04-16 2016-10-20 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
BR112018011881A2 (pt) 2015-12-14 2018-12-04 The University Of North Carolina At Chapel Hill proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus
WO2017192750A1 (en) 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
FI3469073T3 (fi) 2016-06-09 2024-04-17 Centre Nat Rech Scient Kemiallisesti modifioidun kapsidin omaava raav
EA038695B1 (ru) 2017-02-15 2021-10-06 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Способы и композиции для переноса генов по сосудистой сети
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
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
RU2770922C2 (ru) 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
AU2019209002A1 (en) * 2018-01-19 2020-09-03 Agency For Science, Technology And Research Disrupting the LINC complex for treating laminopathy
WO2019183630A2 (en) * 2018-03-23 2019-09-26 The Trustees Of Columbia University In The City Of New York Gene editing for autosomal dominant diseases
CN112703198A (zh) 2018-07-11 2021-04-23 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
US11242368B2 (en) 2018-09-21 2022-02-08 Arizona Board Of Regents On Behalf Of The University Of Arizona Methods and compositions for rapidly replacing cardiac myosin binding protein-C in sarcomeres
WO2020117898A1 (en) 2018-12-05 2020-06-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
KR20210143869A (ko) 2019-03-28 2021-11-29 더 제너럴 하스피탈 코포레이션 트랜스진 발현을 위한 조작된 아데노-연관 (aav) 벡터
US20200405824A1 (en) 2019-06-26 2020-12-31 University Of Washington Use of ribonucleotide reductase alone or in combination with micro-dystrophin to treat duchenne muscular dystrophy striated muscle disease
WO2021038291A1 (en) 2019-08-27 2021-03-04 Dinaqor Ag Drug perfusion into an unarrested beating heart
AU2021218411A1 (en) 2020-02-13 2022-10-06 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
MX2023000453A (es) 2020-07-08 2023-02-09 Childrens Medical Center Polipeptidos de mybpc3 y usos de los mismos.
JP2024501821A (ja) 2020-12-23 2024-01-16 ベーリンガー インゲルハイム インターナショナル ゲゼルシャフト ミット ベシュレンクテル ハフツング 心臓組織細胞に対する特異性を有するウイルスカプシドタンパク質
TW202302173A (zh) 2021-02-22 2023-01-16 瑞士商迪納柯公司 肝之局部區域(loco-regional)灌流
IL305232A (en) 2021-02-22 2023-10-01 Dinaqor Ag Local-regional perfusion of a kidney
EP4294496A1 (en) 2021-02-22 2023-12-27 Dinaqor AG Loco-regional perfusion of an unarrested beating heart

Also Published As

Publication number Publication date
JP2023513749A (ja) 2023-04-03
US11446397B2 (en) 2022-09-20
CO2022012917A2 (es) 2022-09-20
EP4103214A2 (en) 2022-12-21
US11129908B2 (en) 2021-09-28
MX2022009883A (es) 2022-10-03
US20210252165A1 (en) 2021-08-19
US20210346521A1 (en) 2021-11-11
AU2021218411A1 (en) 2022-10-06
US20220160896A1 (en) 2022-05-26
CA3171166A1 (en) 2021-08-19
IL295529A (en) 2022-10-01
KR20220139956A (ko) 2022-10-17
US20220031866A1 (en) 2022-02-03
US11759531B2 (en) 2023-09-19
CN116096394A (zh) 2023-05-09
US20230372541A1 (en) 2023-11-23

Similar Documents

Publication Publication Date Title
BR112022015979A2 (pt) Vetores de terapia genética para tratamento de doenças cardíacas
BR112021021156A2 (pt) Terapêuticos anticorpos totalmente humanos pós-tradução modificados
WO2021163357A3 (en) Gene therapy vectors for treating heart disease
BR112023002904A2 (pt) Vírus adenoassociado recombinante para tratar neurodegeneração com início na idade adulta associada a grn
CL2022000939A1 (es) Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c
BR112021023692A2 (pt) Partícula viral de vírus adeno-associado recombinante, proteína de capsídeo de vírus adeno-associado, molécula de ácido nucleico, célula empacotadora, métodos de produção de uma partícula viral e de distribuição de um nucleotídeo, e, composição farmacêutica
CO2020002283A2 (es) Métodos de terapia génica del factor viii (fviii)
BR112021021908A2 (pt) Composições úteis no tratamento da leucodistrofia metacromática
BR112021015751A2 (pt) Vetores de terapia genética para tratamento da doença de danon
BR112022015921A2 (pt) Terapia gênica para tratar o transtorno de deficiência de cdkl5
CO2024001148A2 (es) Casetes de expresión optimizada para genoterapia
BR112022000898A2 (pt) Terapia de gene cardíaco aav para cardiomiopatia em seres humanos
BR112022004027A2 (pt) Métodos para tratar doenças neovasculares oculares usando variantes de aav2 que codificam aflibercepte
WO2022076556A3 (en) Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21
BR112022019729A2 (pt) Repetição terminal invertida (itr), vetor de ácido nucleico, partícula de vírus adenoassociado recombinante, composição farmacêutica e métodos de entrega de um ácido nucleico e de prevenção ou tratamento de uma doença
EA202290001A1 (ru) Векторы aav с промотором миелинового белка ноль и их применение для лечения заболеваний, связанных со шванновскими клетками, таких как болезнь шарко-мари-тута
BR112023021999A2 (pt) Administração de terapia gênica de microdistrofina para tratamento de distrofinopatias
BR112021016570A2 (pt) Entrega de vetor de vírus adenoassociado de beta-sarcoglicano e tratamento de distrofia muscular
CL2024000062A1 (es) Composición farmacéutica de virus no encapsulado
BR112022021762A2 (pt) Composições úteis em tratamento de distúrbio de deficiência de cdkl5 (cdd)
BR112022011332A2 (pt) Vetor de vírus adeno-associado recombinante, vírus adeno-associado recombinante, e, método para tratar um indivíduo com síndrome de hunter
CL2023001650A1 (es) Tratamiento de la enfermedad de danon
BR112022013914A2 (pt) Composições úteis para tratar gangliosidose gm1
BR112022003206A2 (pt) Métodos para tratar doenças neovasculares oculares usando variantes de aav2 que codificam aflibercepte
UY38881A (es) Métodos de tratamiento de enfermedades neovasculares oculares utilizando variantes de aav2 que codifican aflibercept