PE20170261A1 - Composiciones que comprenden virus adenoasociado (aav) que expresa constructos de anticuerpos duales y sus usos - Google Patents
Composiciones que comprenden virus adenoasociado (aav) que expresa constructos de anticuerpos duales y sus usosInfo
- Publication number
- PE20170261A1 PE20170261A1 PE2016002227A PE2016002227A PE20170261A1 PE 20170261 A1 PE20170261 A1 PE 20170261A1 PE 2016002227 A PE2016002227 A PE 2016002227A PE 2016002227 A PE2016002227 A PE 2016002227A PE 20170261 A1 PE20170261 A1 PE 20170261A1
- Authority
- PE
- Peru
- Prior art keywords
- aav
- orf
- expression
- antibody constructs
- expresses
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title abstract 3
- 230000009977 dual effect Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 108700026244 Open Reading Frames Proteins 0.000 abstract 5
- 230000001105 regulatory effect Effects 0.000 abstract 3
- 108060003951 Immunoglobulin Proteins 0.000 abstract 2
- 102000018358 immunoglobulin Human genes 0.000 abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/08—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses
- C07K16/10—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses from RNA viruses
- C07K16/1018—Orthomyxoviridae, e.g. influenza virus
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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- 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
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- 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
- A61P31/04—Antibacterial agents
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- 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
- A61P31/10—Antimycotics
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- 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
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- 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
- A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P33/00—Antiparasitic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
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- 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
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- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
- A61K2039/507—Comprising a combination of two or more separate antibodies
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/10—Immunoglobulins specific features characterized by their source of isolation or production
- C07K2317/14—Specific host cells or culture conditions, e.g. components, pH or temperature
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
- C07K2317/31—Immunoglobulins specific features characterized by aspects of specificity or valency multispecific
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/51—Complete heavy chain or Fd fragment, i.e. VH + CH1
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/515—Complete light chain, i.e. VL + CL
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/52—Constant or Fc region; Isotype
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
- C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
- C07K2317/622—Single chain antibody (scFv)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron
- C12N2830/205—Vector systems having a special element relevant for transcription transcription of more than one cistron bidirectional
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
- C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES
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- Life Sciences & Earth Sciences (AREA)
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- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
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- General Chemical & Material Sciences (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Bioinformatics & Cheminformatics (AREA)
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Abstract
Se describe un virus adenoasociado (AAV) recombinante que tiene una capside de AAV y empaquetado en ella un acido nucleico heterologo que expresa dos constructos de anticuerpos funcionales en una celula. Tambien se describen anticuerpos que comprenden una cadena pesada y una cadena ligera procedentes de un anticuerpo heterologo. En una realizacion, los anticuerpos se coexpresan a partir de un vector que contiene: un primer casete de expresion que codifica al menos un primer marco de lectura abierto (ORF) para una primera inmunoglobulina controlado por las secuencias de control reguladoras que dirigen su expresion; un segundo casete de expresion que comprende un segundo ORF, un conector y un tercer ORF controlados por secuencias de control reguladoras que dirigen su expresion, donde el segundo y el tercer ORF codifican para un segundo y un tercer constructo de inmunoglobulina; el vector que coexpresa estos dos constructos de anticuerpos es en una realizacion un AAV, en el cual las ITR de 5' y 3' flanquean los casetes de expresion y las secuencias reguladoras
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201461992649P | 2014-05-13 | 2014-05-13 |
Publications (1)
Publication Number | Publication Date |
---|---|
PE20170261A1 true PE20170261A1 (es) | 2017-04-12 |
Family
ID=54480597
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2016002227A PE20170261A1 (es) | 2014-05-13 | 2015-05-13 | Composiciones que comprenden virus adenoasociado (aav) que expresa constructos de anticuerpos duales y sus usos |
Country Status (27)
Country | Link |
---|---|
US (4) | US10138295B2 (es) |
EP (1) | EP3142750B1 (es) |
JP (3) | JP6974943B2 (es) |
KR (1) | KR102550926B1 (es) |
CN (1) | CN106470736B (es) |
AU (2) | AU2015259188B2 (es) |
CA (1) | CA2947614C (es) |
CL (1) | CL2016002840A1 (es) |
CR (1) | CR20160519A (es) |
DK (1) | DK3142750T3 (es) |
EA (1) | EA037980B1 (es) |
ES (1) | ES2833230T3 (es) |
HR (1) | HRP20201530T1 (es) |
HU (1) | HUE050609T2 (es) |
IL (1) | IL248508B2 (es) |
MA (2) | MA44179B1 (es) |
MX (1) | MX365658B (es) |
MY (1) | MY186389A (es) |
PE (1) | PE20170261A1 (es) |
PH (1) | PH12016502239A1 (es) |
PL (1) | PL3142750T3 (es) |
PT (1) | PT3142750T (es) |
SA (1) | SA516380285B1 (es) |
SG (1) | SG11201609207SA (es) |
SI (1) | SI3142750T1 (es) |
UA (1) | UA123986C2 (es) |
WO (1) | WO2015175639A1 (es) |
Families Citing this family (31)
Publication number | Priority date | Publication date | Assignee | Title |
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WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
CA2947614C (en) | 2014-05-13 | 2023-10-03 | The Trustees Of The University Of Pennsylvania | Compositions comprising aav expressing dual antibody constructs and uses thereof |
EA201792500A1 (ru) | 2015-05-13 | 2018-04-30 | Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания | Aav-опосредованная экспрессия антител против гриппа и способы их использования |
WO2017096363A1 (en) * | 2015-12-04 | 2017-06-08 | Board Of Regents, The University Of Texas System | Reporter system for detecting and targeting activated cells |
SG11201808440YA (en) * | 2016-04-15 | 2018-10-30 | Regenxbio Inc | Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab |
JP7046828B2 (ja) * | 2016-04-15 | 2022-04-04 | 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 | 滲出型加齢性黄斑変性の治療のための組成物 |
US11401527B2 (en) | 2016-04-17 | 2022-08-02 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for prophylaxis of organophosphates |
WO2017189963A1 (en) * | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
US11326182B2 (en) | 2016-04-29 | 2022-05-10 | 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 |
KR102573534B1 (ko) * | 2016-05-12 | 2023-08-31 | 얀센 백신스 앤드 프리벤션 비.브이. | 강력하고 균형 잡힌 양방향성 프로모터 |
CN109312362B (zh) | 2016-06-20 | 2022-06-28 | 扬森疫苗与预防公司 | 有效和平衡的双向启动子 |
AU2017313917B2 (en) | 2016-08-18 | 2023-12-21 | The Regents Of The University Of California | CRISPR-Cas genome engineering via a modular AAV delivery system |
SI3589730T1 (sl) | 2017-02-28 | 2024-04-30 | The Trustees Of The University Of Pennsylvania | Adeno-povezan virus (AAV) clade F vektor in njihova uporaba |
SG10201913833PA (en) * | 2017-02-28 | 2020-03-30 | Univ Pennsylvania | Influenza vaccines based on aav vectors |
JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
CN111246854A (zh) * | 2017-08-17 | 2020-06-05 | 宾夕法尼亚大学理事会 | 编码单纯疱疹病毒糖蛋白的修饰mrna疫苗及其用途 |
EP3727468A4 (en) | 2017-12-19 | 2021-09-22 | Akouos, Inc. | AAV-MEDIATED DELIVERY OF THERAPEUTIC ANTIBODIES TO THE INNER EAR |
CN108424934A (zh) * | 2018-04-16 | 2018-08-21 | 和元生物技术(上海)股份有限公司 | 一种慢病毒cag-cmv双启动子改造载体构建及应用 |
JP7413629B2 (ja) | 2018-07-17 | 2024-01-16 | ヘリックスミス カンパニー, リミテッド | Igf-1異型体を発現させるdnaコンストラクトを用いた神経病症の治療 |
BR112021002017A2 (pt) * | 2018-08-09 | 2021-05-11 | Bioverativ Therapeutics Inc. | moléculas de ácido nucleico e usos das mesmas para terapia genética não viral |
US20200148762A1 (en) * | 2018-11-09 | 2020-05-14 | Avamab Pharma Inc. | Methods for regulating endogenous production of antibodies against infectious diseases |
WO2020132382A1 (en) * | 2018-12-21 | 2020-06-25 | Merck Sharp & Dohme Corp. | Expression vectors for eukaryotic expression systems |
US20230193315A1 (en) | 2019-01-31 | 2023-06-22 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of aav capsids |
TW202106708A (zh) * | 2019-04-24 | 2021-02-16 | 美商銳進科斯生物股份有限公司 | 全人類轉譯後修飾之抗體治療劑 |
AU2020403133A1 (en) * | 2019-12-11 | 2022-06-23 | Visterra, Inc. | Compositions and methods for treating and preventing influenza |
JP2023550459A (ja) * | 2020-11-23 | 2023-12-01 | ノバルティス アーゲー | 抗体構築物の発現技術 |
AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
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WO2024125530A1 (zh) * | 2022-12-12 | 2024-06-20 | 苏州荷光科汇生物科技有限公司 | 一种感染视网膜的aav载体、阿达木单抗及其应用 |
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