AU2017228413B2 - Closed-ended linear duplex DNA for non-viral gene transfer - Google Patents

Closed-ended linear duplex DNA for non-viral gene transfer Download PDF

Info

Publication number
AU2017228413B2
AU2017228413B2 AU2017228413A AU2017228413A AU2017228413B2 AU 2017228413 B2 AU2017228413 B2 AU 2017228413B2 AU 2017228413 A AU2017228413 A AU 2017228413A AU 2017228413 A AU2017228413 A AU 2017228413A AU 2017228413 B2 AU2017228413 B2 AU 2017228413B2
Authority
AU
Australia
Prior art keywords
hsa
mir
nucleic acid
sequence
cedna
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.)
Active
Application number
AU2017228413A
Other languages
English (en)
Other versions
AU2017228413A1 (en
Inventor
Sylvain CECCHINI
Robert M. Kotin
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.)
Voyager Therapeutics Inc
University of Massachusetts Amherst
Original Assignee
Voyager Therapeutics Inc
University of Massachusetts Amherst
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 Voyager Therapeutics Inc, University of Massachusetts Amherst filed Critical Voyager Therapeutics Inc
Publication of AU2017228413A1 publication Critical patent/AU2017228413A1/en
Assigned to UNIVERSITY OF MASSACHUSETTS, VOYAGER THERAPEUTICS, INC. reassignment UNIVERSITY OF MASSACHUSETTS Request for Assignment Assignors: UNIVERSITY OF MASSACHUSETTS
Application granted granted Critical
Publication of AU2017228413B2 publication Critical patent/AU2017228413B2/en
Priority to AU2025202724A priority Critical patent/AU2025202724A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • 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
    • A61K48/0075—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
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/12—Mucolytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • 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
    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • 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
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Pulmonology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
AU2017228413A 2016-03-03 2017-03-03 Closed-ended linear duplex DNA for non-viral gene transfer Active AU2017228413B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025202724A AU2025202724A1 (en) 2016-03-03 2025-04-17 Closed-ended linear duplex dna for non-viral gene transfer

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662303047P 2016-03-03 2016-03-03
US62/303,047 2016-03-03
US201662394720P 2016-09-14 2016-09-14
US62/394,720 2016-09-14
US201662406913P 2016-10-11 2016-10-11
US62/406,913 2016-10-11
PCT/US2017/020828 WO2017152149A1 (en) 2016-03-03 2017-03-03 Closed-ended linear duplex dna for non-viral gene transfer

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2025202724A Division AU2025202724A1 (en) 2016-03-03 2025-04-17 Closed-ended linear duplex dna for non-viral gene transfer

Publications (2)

Publication Number Publication Date
AU2017228413A1 AU2017228413A1 (en) 2018-08-23
AU2017228413B2 true AU2017228413B2 (en) 2025-01-23

Family

ID=59743259

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2017228413A Active AU2017228413B2 (en) 2016-03-03 2017-03-03 Closed-ended linear duplex DNA for non-viral gene transfer
AU2025202724A Pending AU2025202724A1 (en) 2016-03-03 2025-04-17 Closed-ended linear duplex dna for non-viral gene transfer

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2025202724A Pending AU2025202724A1 (en) 2016-03-03 2025-04-17 Closed-ended linear duplex dna for non-viral gene transfer

Country Status (18)

Country Link
US (3) US11066679B2 (enExample)
EP (2) EP3957331A1 (enExample)
JP (3) JP6994140B2 (enExample)
KR (1) KR102336362B1 (enExample)
CN (2) CN115287301B (enExample)
AU (2) AU2017228413B2 (enExample)
CA (1) CA3014683A1 (enExample)
DK (1) DK3423110T3 (enExample)
ES (1) ES2898337T3 (enExample)
HR (1) HRP20211697T1 (enExample)
IL (2) IL261524B2 (enExample)
LT (1) LT3423110T (enExample)
MX (1) MX2018010633A (enExample)
PT (1) PT3423110T (enExample)
RU (1) RU2752882C2 (enExample)
SG (2) SG11201806663TA (enExample)
WO (1) WO2017152149A1 (enExample)
ZA (1) ZA201806544B (enExample)

Families Citing this family (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
IL261524B2 (en) 2016-03-03 2023-10-01 Univ Massachusetts End-capped linear double-stranded DNA for non-viral gene transfer
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
MX2020001593A (es) 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.
EP3679148A4 (en) * 2017-09-08 2021-06-09 Generation Bio Co. LIPID NANOPARTICLE FORMULATIONS FROM NONVIRAL, CAPSID-FREE DNA VECTORS
CA3075168A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
US12098385B2 (en) 2017-09-20 2024-09-24 The Regents Of The University Of California Gene therapy strategy to restore cardiac electrical and structural function in arrhythmogenic right ventricular cardiomyopathy
WO2019060619A1 (en) 2017-09-20 2019-03-28 The Regents Of The University Of California STRATEGY OF GENE THERAPY FOR RESTORING ELECTRICAL AND CARDIAC FUNCTION AND CARDIAC STRUCTURE IN ARYTHMOGENIC RIGHT VENTRICULAR CARDIOMYOPATHY
WO2019079362A1 (en) * 2017-10-16 2019-04-25 Massachusetts Institute Of Technology HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS
JP2021505159A (ja) * 2017-12-06 2021-02-18 ジェネレーション バイオ カンパニー 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集
WO2019126222A1 (en) * 2017-12-18 2019-06-27 Spark Therapeutics, Inc. Adeno-associated virus (aav) vector lipid nanoparticle compositions and methods of use
JP2021511047A (ja) 2018-01-19 2021-05-06 ジェネレーション バイオ カンパニー 無細胞合成から得ることができる閉端ＤＮＡベクターおよびｃｅＤＮＡベクターを得るためのプロセス
AU2019221642A1 (en) * 2018-02-14 2020-07-09 Generation Bio Co. Non-viral DNA vectors and uses thereof for antibody and fusion protein production
EP3755803A4 (en) * 2018-02-22 2022-01-19 Generation Bio Co. CONTROLLED EXPRESSION OF TRANSGENES USING DNA (CEDNA) VECTORS
CA3092459A1 (en) * 2018-03-02 2019-09-06 Generation Bio Co. Closed-ended dna (cedna) vectors for insertion of transgenes at genomic safe harbors (gsh) in humans and murine genomes
MX2020009579A (es) * 2018-03-15 2020-11-09 Intergalactic Therapeutics Inc Vectores de adn sintetico y metodos de uso.
WO2019191109A1 (en) * 2018-03-28 2019-10-03 The Board Of Trustees Of The Leland Stanford Junior University Microrna inhibitors and their use in treating epiretinal membranes
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
MA52709A (fr) 2018-05-23 2021-03-31 Modernatx Inc Administration d'adn
WO2019237391A1 (zh) * 2018-06-16 2019-12-19 深圳市博奥康生物科技有限公司 CRISPR/Cas9 靶向敲除人 TXGP1 基因及其特异性 gRNA
WO2019237383A1 (zh) * 2018-06-16 2019-12-19 深圳市博奥康生物科技有限公司 用于人 tnfsf18 基因编辑的修饰载体、其制备方法及应用
CN112639110A (zh) * 2018-06-22 2021-04-09 阿斯克肋匹奥生物制药公司 用于基因递送以在细胞内持续存在的载体
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
WO2020037254A1 (en) * 2018-08-17 2020-02-20 City Of Hope Compounds of chemically modified oligonucleotides and methods of use thereof
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020097417A1 (en) * 2018-11-09 2020-05-14 Generation Bio Co. Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats
KR20210119416A (ko) * 2019-01-24 2021-10-05 제너레이션 바이오 컴퍼니 폐쇄-말단 dna (cedna), 및 유전자 또는 핵산 치료 관련 면역 반응을 감소시키는 방법에서의 이의 용도
CA3129321A1 (en) * 2019-02-15 2020-08-20 Generation Bio Co. Modulation of rep protein activity in closed-ended dna (cedna) production
AU2020232790A1 (en) * 2019-03-06 2021-09-16 Generation Bio Co. Non-active lipid nanoparticles with non-viral, capsid free DNA
SG11202108864UA (en) * 2019-03-06 2021-09-29 Generation Bio Co Closed-ended dna (cedna) and immune modulating compounds
KR20210151785A (ko) * 2019-03-13 2021-12-14 제너레이션 바이오 컴퍼니 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
US12415002B2 (en) * 2019-03-13 2025-09-16 Generation Bio Co. Non-viral DNA vectors and uses thereof for expressing phenylalanine hydroxylase (PAH) therapeutics
US12582726B1 (en) 2019-04-05 2026-03-24 Earli Inc. Synthetic cancer-specific promoters
EP3946470A4 (en) 2019-04-05 2023-05-31 Earli Inc. IMPROVED METHODS AND COMPOSITIONS FOR SYNTHETIC BIOMARKERS
WO2021011842A1 (en) 2019-07-17 2021-01-21 Generation Bio Co. Synthetic production of single-stranded adeno associated viral dna vectors
WO2021055760A1 (en) 2019-09-18 2021-03-25 Intergalactic Therapeutics, Inc. Synthetic dna vectors and methods of use
US20230089312A1 (en) 2020-02-25 2023-03-23 University Of Massachusetts Inducible single aav system and uses thereof
IL296660A (en) * 2020-03-24 2022-11-01 Generation Bio Co Non-viral DNA vectors and their use for therapeutic expression of factor ix
US12534435B2 (en) 2020-03-27 2026-01-27 Generation Bio Co. Lipids and nanoparticle compositions thereof
US20210330759A1 (en) * 2020-04-22 2021-10-28 The Cleveland Clinic Foundation Methods and compositions for treating cytokine storm, ards, and acute lung injury using beta-glucocerebrosidase
AU2021285812A1 (en) 2020-06-01 2023-01-05 Modernatx, Inc. Phenylalanine hydroxylase variants and uses thereof
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
AU2021330936A1 (en) 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
MX2023003019A (es) * 2020-09-16 2023-05-08 Generation Bio Co Vectores de adn de extremo cerrado y usos de estos para expresar fenilalanina hidroxilasa (pah).
CA3191743A1 (en) * 2020-09-16 2022-03-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing fviii therapeutics
EP4333886A4 (en) * 2021-05-07 2025-11-12 Generation Bio Co NON-VIRAL DNA VECTORS FOR VACCINE ADMINISTRATION
WO2022236016A1 (en) * 2021-05-07 2022-11-10 Generation Bio Co. Lyophilized non-viral dna vector compositions and uses thereof
WO2022240806A1 (en) 2021-05-11 2022-11-17 Modernatx, Inc. Non-viral delivery of dna for prolonged polypeptide expression in vivo
JP2024534159A (ja) * 2021-08-23 2024-09-18 アビラマックス・バイオファーマ・インコーポレイテッド 導入遺伝子発現のための組成物および方法
WO2023028440A2 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
CN114525282A (zh) * 2022-03-04 2022-05-24 西南大学 一种二价核酸适配体及其设计方法
KR20250151437A (ko) * 2023-02-17 2025-10-21 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 변형된 우라실을 포함하는 dna 조성물
US12383615B2 (en) 2023-03-23 2025-08-12 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods
WO2024249438A1 (en) * 2023-05-26 2024-12-05 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Copiparvovirus genetic vector platform
WO2025212926A1 (en) * 2024-04-05 2025-10-09 University Of Massachusetts Aav-mediated rhoa knockdown
US20260000702A1 (en) 2024-06-26 2026-01-01 Flagship Pioneering Innovations Vii, Llc Therapeutic circular dna forms
WO2026085495A1 (en) 2024-10-18 2026-04-23 Generation Bio Co. Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
US5316908A (en) * 1990-07-13 1994-05-31 Life Technologies, Inc. Size markers for electrophoretic analysis of DNA
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
JP3696238B2 (ja) * 1993-08-30 2005-09-14 プロメガ・コーポレイシヨン 核酸精製用組成物及び方法
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US7098320B1 (en) 1996-07-29 2006-08-29 Nanosphere, Inc. Nanoparticles having oligonucleotides attached thereto and uses therefor
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
CA2353596A1 (en) 1998-12-04 2000-06-15 Mosaic Technologies, Inc. Method for the immobilization of oligonucleotides
DE10066104A1 (de) * 2000-09-08 2003-01-09 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
US8241622B2 (en) * 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
CA3144493A1 (en) 2006-10-03 2008-04-10 Arbutus Biopharma Corporation Lipid containing formulations
EP2229186A2 (en) 2007-12-04 2010-09-22 Alnylam Pharmaceuticals Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
US8507455B2 (en) 2007-12-04 2013-08-13 Alnylam Pharmaceuticals, Inc. Folate conjugates
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
WO2012068187A1 (en) 2010-11-19 2012-05-24 Merck Sharp & Dohme Corp. Poly(amide) polymers for the delivery of oligonucleotides
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
IL261524B2 (en) 2016-03-03 2023-10-01 Univ Massachusetts End-capped linear double-stranded DNA for non-viral gene transfer

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
HICKMAN, AB ET AL.: "The Nuclease Domain of Adeno-Associated Virus Rep Coordinates Replication Initiation Using Two Distinct DNA Recognition Interfaces", MOLECULAR CELL, vol. 13, 13 February 2004 (2004-02-13), pages 403 - 414, XP055412372 *

Also Published As

Publication number Publication date
SG11201806663TA (en) 2018-09-27
LT3423110T (lt) 2021-12-27
IL261524B2 (en) 2023-10-01
JP2022025123A (ja) 2022-02-09
EP3423110B1 (en) 2021-08-11
CN115287301A (zh) 2022-11-04
JP2023024489A (ja) 2023-02-16
JP7193096B2 (ja) 2022-12-20
EP3957331A1 (en) 2022-02-23
IL302398B1 (en) 2025-02-01
JP2019506889A (ja) 2019-03-14
CN115287301B (zh) 2026-06-16
US20220195456A9 (en) 2022-06-23
ZA201806544B (en) 2022-07-27
US20190032083A1 (en) 2019-01-31
US12252703B2 (en) 2025-03-18
CN109195636B (zh) 2022-07-05
EP3423110A4 (en) 2019-09-11
PT3423110T (pt) 2021-11-09
DK3423110T3 (da) 2021-11-15
IL302398A (en) 2023-06-01
RU2752882C2 (ru) 2021-08-11
IL261524B1 (en) 2023-06-01
MX2018010633A (es) 2019-06-13
RU2018134295A (ru) 2020-04-06
AU2017228413A1 (en) 2018-08-23
ES2898337T3 (es) 2022-03-07
WO2017152149A1 (en) 2017-09-08
HRP20211697T1 (hr) 2022-02-04
US20250290094A1 (en) 2025-09-18
KR102336362B1 (ko) 2021-12-08
NZ744961A (en) 2025-06-27
US11066679B2 (en) 2021-07-20
KR20180117171A (ko) 2018-10-26
CA3014683A1 (en) 2017-09-08
JP6994140B2 (ja) 2022-02-03
AU2025202724A1 (en) 2025-05-08
SG10201913688TA (en) 2020-03-30
RU2018134295A3 (enExample) 2020-06-29
US20210355507A1 (en) 2021-11-18
IL261524A (en) 2018-10-31
CN109195636A (zh) 2019-01-11
IL302398B2 (en) 2025-06-01
EP3423110A1 (en) 2019-01-09

Similar Documents

Publication Publication Date Title
AU2025202724A1 (en) Closed-ended linear duplex dna for non-viral gene transfer
US20250034555A1 (en) High efficiency library-identified aav vectors
AU2017341849B2 (en) AAV capsid designs
EP3209311B1 (en) Recombinant aav variants and uses thereof
US20190389934A1 (en) Heterologous targeting peptide grafted aavs
US11472848B2 (en) AAV capsids identified by in vivo library selection
HK40069282A (en) Closed-ended linear duplex dna for non-viral gene transfer
HK40002860B (en) Closed-ended linear duplex dna for non-viral gene transfer

Legal Events

Date Code Title Description
PC1 Assignment before grant (sect. 113)

Owner name: VOYAGER THERAPEUTICS, INC.

Free format text: FORMER APPLICANT(S): UNIVERSITY OF MASSACHUSETTS

Owner name: UNIVERSITY OF MASSACHUSETTS

Free format text: FORMER APPLICANT(S): UNIVERSITY OF MASSACHUSETTS

FGA Letters patent sealed or granted (standard patent)