CA3105381A1 - Methods and compositions for delivery of agents across the blood-brain barrier - Google Patents

Methods and compositions for delivery of agents across the blood-brain barrier Download PDF

Info

Publication number
CA3105381A1
CA3105381A1 CA3105381A CA3105381A CA3105381A1 CA 3105381 A1 CA3105381 A1 CA 3105381A1 CA 3105381 A CA3105381 A CA 3105381A CA 3105381 A CA3105381 A CA 3105381A CA 3105381 A1 CA3105381 A1 CA 3105381A1
Authority
CA
Canada
Prior art keywords
aav
seq
cpp
capsid protein
sequence
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3105381A
Other languages
English (en)
French (fr)
Inventor
Fengfeng BEI
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
The Brigham and Women's Hospital Inc
Original Assignee
The Brigham and Women's Hospital 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 The Brigham and Women's Hospital Inc filed Critical The Brigham and Women's Hospital Inc
Publication of CA3105381A1 publication Critical patent/CA3105381A1/en
Pending legal-status Critical Current

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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52—Purines, e.g. adenine
    • A61K31/522—Purines, e.g. adenine having oxo groups directly attached to the heterocyclic ring, e.g. hypoxanthine, guanine, acyclovir
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/46—Ingredients of undetermined constitution or reaction products thereof, e.g. skin, bone, milk, cotton fibre, eggshell, oxgall or plant extracts
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/001—Preparation for luminescence or biological staining
    • A61K49/0013—Luminescence
    • A61K49/0017—Fluorescence in vivo
    • A61K49/0019—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules
    • A61K49/0045—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules the fluorescent agent being a peptide or protein used for imaging or diagnosis in vivo
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/001—Preparation for luminescence or biological staining
    • A61K49/0063—Preparation for luminescence or biological staining characterised by a special physical or galenical form, e.g. emulsions, microspheres
    • A61K49/0069—Preparation for luminescence or biological staining characterised by a special physical or galenical form, e.g. emulsions, microspheres the agent being in a particular physical galenical form
    • A61K49/0097—Cells, viruses, ghosts, red blood cells, viral vectors, used for imaging or diagnosis in vivo
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • 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
    • 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
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurosurgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Radiology & Medical Imaging (AREA)
  • Pathology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Psychology (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Hospice & Palliative Care (AREA)
CA3105381A 2018-07-11 2019-07-11 Methods and compositions for delivery of agents across the blood-brain barrier Pending CA3105381A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862696422P 2018-07-11 2018-07-11
US62/696,422 2018-07-11
PCT/US2019/041386 WO2020014471A1 (en) 2018-07-11 2019-07-11 Methods and compositions for delivery of agents across the blood-brain barrier

Publications (1)

Publication Number Publication Date
CA3105381A1 true CA3105381A1 (en) 2020-01-16

Family

ID=69142796

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3105381A Pending CA3105381A1 (en) 2018-07-11 2019-07-11 Methods and compositions for delivery of agents across the blood-brain barrier

Country Status (6)

Country Link
US (3) US12398181B2 (cg-RX-API-DMAC10.html)
EP (1) EP3820885A4 (cg-RX-API-DMAC10.html)
JP (2) JP2021530218A (cg-RX-API-DMAC10.html)
CN (2) CN112703198B (cg-RX-API-DMAC10.html)
CA (1) CA3105381A1 (cg-RX-API-DMAC10.html)
WO (1) WO2020014471A1 (cg-RX-API-DMAC10.html)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
JP2021530218A (ja) * 2018-07-11 2021-11-11 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドThe Brigham and Women’s Hospital, Inc. 血液脳関門を超える薬剤の送達のための方法および組成物
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
AU2020214831B2 (en) * 2019-01-30 2025-09-25 The Broad Institute, Inc. Systems for evolved adeno-associated viruses (AAVs) for targeted delivery
CN115023242B (zh) * 2019-11-22 2025-03-14 费城儿童医院 腺相关病毒载体变体
CA3167290A1 (en) 2020-01-10 2021-07-15 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
FI4103214T3 (fi) 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon
AU2021265832A1 (en) 2020-04-29 2022-11-24 The Broad Institute, Inc. Machine learning accelerated protein engineering through fitness prediction
US12630842B2 (en) * 2020-05-13 2026-05-19 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
EP4199972A4 (en) * 2020-08-21 2025-01-15 Capsida, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS WITH 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
CN112194706B (zh) * 2020-09-30 2022-03-08 广州派真生物技术有限公司 腺相关病毒突变体及其应用
AU2022266615A1 (en) * 2021-04-27 2023-11-09 The General Hospital Corporation Aav capsids and uses thereof
WO2022272240A2 (en) * 2021-06-21 2022-12-29 The Brigham And Women’S Hospital, Inc. Adeno-associated viral (aav) vectors for tissue-targeted expression of therapeutic genes
KR102934033B1 (ko) * 2021-10-01 2026-03-06 서울대학교산학협력단 혈액 뇌장벽 투과용 펩타이드 및 이의 용도
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CN114606264B (zh) * 2022-03-22 2025-08-26 三峡大学 一种用于神经系统药物递送的aav病毒载体
WO2023240236A1 (en) * 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
JP2026509271A (ja) 2023-03-10 2026-03-17 ダイノ セラピューティクス，インコーポレーテッド カプシドポリペプチドおよびその使用方法
WO2024229777A1 (zh) * 2023-05-11 2024-11-14 深圳先进技术研究院 一种aav载体及其应用
WO2024249810A2 (en) * 2023-06-02 2024-12-05 The Brigham And Women's Hospital, Inc. Combinatory use of adeno-associated viruses and focused ultrasound for overcoming the blood-brain barrier
TW202515898A (zh) 2023-06-30 2025-04-16 日商武田藥品工業股份有限公司 Ｈｔｔ抑制物及其用途
CN116808231B (zh) * 2023-07-07 2024-03-26 中国药科大学 一种细胞穿膜肽偶联舒必利前药体系以及制备方法和应用
CN117964715B (zh) * 2023-07-21 2025-01-24 广州派真生物技术有限公司 腺相关病毒突变体及其应用
KR20260082361A (ko) 2023-08-31 2026-06-04 디노 테라퓨틱스, 인코포레이티드 캡시드 폴리펩타이드 및 이의 사용 방법
WO2025076091A1 (en) * 2023-10-03 2025-04-10 Brave Bio, Inc. Cd20 antibody aav vectors and methods of use thereof
TW202535908A (zh) * 2023-11-02 2025-09-16 美商渤健麻塞諸塞州股份有限公司 中樞神經系統靶向之ａａｖ衣殼之重定向
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025231400A1 (en) 2024-05-03 2025-11-06 Sangamo Therapeutics, Inc. Synthetic transcription factors and methods of making and using thereof
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026122963A1 (en) 2024-12-06 2026-06-11 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN120230754B (zh) * 2025-03-28 2026-03-17 贵州医科大学 一种基因、融合蛋白、重组载体、重组细胞、脑靶向外泌体及其应用

Family Cites Families (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US6054312A (en) 1997-08-29 2000-04-25 Selective Genetics, Inc. Receptor-mediated gene delivery using bacteriophage vectors
DE60035057T2 (de) 1999-10-04 2008-01-31 Novartis Vaccines and Diagnostics, Inc., Emeryville CD40 Antagonist zur Behandlung von Psoriasis
US20030059427A1 (en) 2000-04-28 2003-03-27 Force Walker R. Isolation and characterization of highly active anti-CD40 antibody
CA2658221C (en) 2001-04-27 2012-11-27 Kyowa Kirin Co., Ltd. Anti-cd40 monoclonal antibody
DE60334303D1 (de) 2002-07-03 2010-11-04 Tasuku Honjo Immunpotenzierende zusammensetzungen
US7186699B2 (en) 2003-06-03 2007-03-06 Cell Genesys, Inc. Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
US8277810B2 (en) 2003-11-04 2012-10-02 Novartis Vaccines & Diagnostics, Inc. Antagonist anti-CD40 antibodies
EP1707627B1 (en) 2003-12-25 2012-11-14 Kyowa Hakko Kirin Co., Ltd. Antagonistic anti-CD40 antibody mutant
EP3530736A3 (en) 2005-05-09 2019-11-06 ONO Pharmaceutical Co., Ltd. Human monoclonal antibodies to programmed death 1 (pd-1) and methods for treating cancer using anti-pd-1 antibodies alone or in combination with other immunotherapeutics
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
SMP200800064B (it) 2006-04-21 2009-11-06 Novartis Ag Composizioni farmaceutiche di anticorpi anti-cd40 antagonista
CA2693712C (en) 2007-07-14 2018-05-29 The University Of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2010036959A2 (en) 2008-09-26 2010-04-01 Dana-Farber Cancer Institute Human anti-pd-1, pd-l1, and pd-l2 antibodies and uses therefor
ES2536465T3 (es) 2008-10-01 2015-05-25 Immatics Biotechnologies Gmbh Composición de péptidos tumor-asociados y relacionados con la vacuna contra el cáncer para el tratamiento de glioblastoma (GBM) y otros cánceres
WO2010129023A2 (en) 2009-04-28 2010-11-11 President And Fellows Of Harvard College Supercharged proteins for cell penetration
EP2796467B1 (en) 2010-03-31 2018-03-28 Boehringer Ingelheim International GmbH Anti-CD40 antibodies
DK2561073T3 (en) * 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP3702460A1 (en) 2010-11-12 2020-09-02 The General Hospital Corporation Polycomb-associated non-coding rnas
AU2011349464B2 (en) 2010-12-20 2016-07-07 The General Hospital Corporation Polycomb-associated non-coding RNAS
ES2734076T3 (es) 2011-02-17 2019-12-04 Kyowa Hakko Kirin Co Ltd Preparación farmacéutica de anticuerpo anti-CD40 muy concentrada
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
KR102037432B1 (ko) 2011-04-29 2019-11-27 아펙시젠, 인코포레이티드 항-cd40 항체 및 사용 방법
BR112014012819B1 (pt) 2011-11-28 2022-08-16 Merck Patent Gmbh Anticorpo anti-pd-l1 ou fragmento de ligação ao antígeno do mesmo e composição
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
AU2013332061A1 (en) 2012-10-17 2015-05-07 Fondazione Telethon Gene therapy for glycogen storage diseases
CA2888763A1 (en) 2012-10-30 2014-05-08 Apexigen, Inc. Anti-cd40 antibodies and methods of use
US10253099B2 (en) * 2012-12-05 2019-04-09 Ruprecht-Karls-Universität Heidelberg Conjugates of proteins and multivalent cell-penetrating peptides and their uses
KR102298579B1 (ko) 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
AU2014276440A1 (en) 2013-06-03 2015-11-05 Novartis Ag Combinations of an anti-PD-L1 antibody and a MEK inhibitor and/or a BRaf inhibitor
EP3564379A1 (en) * 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
US11053291B2 (en) * 2014-02-19 2021-07-06 University Of Florida Research Foundation, Incorporated Delivery of Nrf2 as therapy for protection against reactive oxygen species
EP4050020A1 (en) 2014-03-11 2022-08-31 University of Florida Research Foundation, Inc. Aav-expressed m013 protein as an anti-inflammatroy therapeutic for use in a method of treating inflammatory ocular disease
US10577627B2 (en) * 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CN106604933B (zh) 2014-07-11 2021-08-10 文塔纳医疗系统公司 抗pd-l1抗体及其诊断用途
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
SG11201702401RA (en) 2014-10-14 2017-04-27 Novartis Ag Antibody molecules to pd-l1 and uses thereof
WO2016138525A1 (en) * 2015-02-27 2016-09-01 University Of Washington Polypeptide assemblies and methods for the production thereof
WO2016146143A1 (en) 2015-03-16 2016-09-22 Amal Therapeutics Sa Cell penetrating peptides and complexes comprising the same
CA3172682A1 (en) 2015-04-23 2016-10-27 Nantomics, Llc Cancer neoepitopes
CN104965364B (zh) 2015-07-14 2018-03-30 武汉华星光电技术有限公司 阵列基板及驱动阵列基板的方法
AR105654A1 (es) 2015-08-24 2017-10-25 Lilly Co Eli Anticuerpos pd-l1 (ligando 1 de muerte celular programada)
EP3373979A1 (en) 2015-11-12 2018-09-19 Pfizer Inc Tissue-specific genome engineering using crispr-cas9
AU2016366549B2 (en) * 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
CN106939047B (zh) 2016-01-04 2021-08-31 江苏怀瑜药业有限公司 一种pd-l1抗体及其制备方法
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
EP3583220A4 (en) 2017-02-15 2021-07-07 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR GENERAL TRANSFER IN THE VASCULAR SYSTEM
WO2019012176A1 (en) 2017-07-11 2019-01-17 Outotec (Finland) Oy WATERING APPARATUS
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
JP7403165B2 (ja) 2017-12-19 2023-12-22 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 血液脳関門を通過してウイルスベクターを送達するための方法および組成物
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20210207167A1 (en) 2018-05-16 2021-07-08 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
SG11202012203PA (en) 2018-06-27 2021-01-28 Precigen Inc In vivo controlled combination therapy for treatment of cancer
AU2019299555A1 (en) 2018-07-04 2021-02-25 Cytoimmune Therapeutics, Inc. Compositions and methods for immunotherapy targeting FLT3, PD-1, and/or PD-L1
JP2021530218A (ja) 2018-07-11 2021-11-11 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドThe Brigham and Women’s Hospital, Inc. 血液脳関門を超える薬剤の送達のための方法および組成物
EP3830107A2 (en) 2018-08-03 2021-06-09 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
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2020214831B2 (en) 2019-01-30 2025-09-25 The Broad Institute, Inc. Systems for evolved adeno-associated viruses (AAVs) for targeted delivery
SG11202111249YA (en) 2019-04-11 2021-11-29 California Inst Of Techn Virus compositions with enhanced specificity in the brain
US20230075314A1 (en) 2019-04-29 2023-03-09 Voyager Therapeutics, Inc. VECTORIZED ANTIBODIES (vAb) AND USES THEREOF
WO2021025995A1 (en) 2019-08-02 2021-02-11 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
CA3167290A1 (en) 2020-01-10 2021-07-15 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
WO2022272240A2 (en) 2021-06-21 2022-12-29 The Brigham And Women’S Hospital, Inc. Adeno-associated viral (aav) vectors for tissue-targeted expression of therapeutic genes

Also Published As

Publication number Publication date
JP2024129031A (ja) 2024-09-26
US20230077490A1 (en) 2023-03-16
US11518787B2 (en) 2022-12-06
EP3820885A1 (en) 2021-05-19
JP7897894B2 (ja) 2026-07-30
CN120843603A (zh) 2025-10-28
US20210277066A1 (en) 2021-09-09
US20220089650A1 (en) 2022-03-24
WO2020014471A1 (en) 2020-01-16
US12398181B2 (en) 2025-08-26
JP2021530218A (ja) 2021-11-11
CN112703198B (zh) 2025-05-30
CN112703198A (zh) 2021-04-23
EP3820885A4 (en) 2022-04-20

Similar Documents

Publication Publication Date Title
US11518787B2 (en) Methods and compositions for delivery of agents across the blood-brain barrier
US20240209394A1 (en) AAV Capsids and Uses Thereof
JP7659271B2 (ja) 導入遺伝子発現のための操作されたアデノ随伴（ａａｖ）ベクター
US20240158762A1 (en) Methods and compositions for delivery of viral vectors across the blood-brain barrier
CN114127089A (zh) 重组腺相关病毒及其用途
US20250074949A1 (en) Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
JP2023515795A (ja) Ａａｖカプシド－プロモーター相互作用および細胞選択的遺伝子発現
US20250188489A1 (en) Conjugation of adeno-associated viruses
US20230048492A1 (en) Adeno-Associated Viral (AAV) Vectors for Tissue-Targeted Expression of Therapeutic Genes
KR20250114282A (ko) 근육 특이적 조절 카세트
WO2024216244A9 (en) Targeting aav capsids, methods of manufacturing and using same
WO2024220545A2 (en) Aav capsids for on-target delivery to spinal cord
CA3092451A1 (en) A modular system for gene and protein delivery based on aav

Legal Events

Date Code Title Description
MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - STANDARD

Year of fee payment: 5

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED

Effective date: 20240709

D00 Search and/or examination requested or commenced

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20241115

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20241115

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250407

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT

Effective date: 20250429

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 6TH ANNIV.) - STANDARD

Year of fee payment: 6

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250707

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250707

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20251219

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20260417

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20260421

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20260421

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20260421

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 7TH ANNIV.) - STANDARD

Year of fee payment: 7

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260706

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260706