PE20211581A1 - Composiciones para la reduccion de la expresion transgenica especifica de drg - Google Patents

Composiciones para la reduccion de la expresion transgenica especifica de drg

Info

Publication number
PE20211581A1
PE20211581A1 PE2021000908A PE2021000908A PE20211581A1 PE 20211581 A1 PE20211581 A1 PE 20211581A1 PE 2021000908 A PE2021000908 A PE 2021000908A PE 2021000908 A PE2021000908 A PE 2021000908A PE 20211581 A1 PE20211581 A1 PE 20211581A1
Authority
PE
Peru
Prior art keywords
drg
expression
aav
itr
compositions
Prior art date
Application number
PE2021000908A
Other languages
English (en)
Spanish (es)
Inventor
Juliette Hordeaux
James M Wilson
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of PE20211581A1 publication Critical patent/PE20211581A1/es

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Mycology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PE2021000908A 2018-12-21 2019-12-20 Composiciones para la reduccion de la expresion transgenica especifica de drg PE20211581A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862783956P 2018-12-21 2018-12-21
US201962924970P 2019-10-23 2019-10-23
US201962934915P 2019-11-13 2019-11-13
PCT/US2019/067872 WO2020132455A1 (en) 2018-12-21 2019-12-20 Compositions for drg-specific reduction of transgene expression

Publications (1)

Publication Number Publication Date
PE20211581A1 true PE20211581A1 (es) 2021-08-17

Family

ID=71100588

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2021000908A PE20211581A1 (es) 2018-12-21 2019-12-20 Composiciones para la reduccion de la expresion transgenica especifica de drg

Country Status (17)

Country Link
US (1) US20210077553A1 (enExample)
EP (1) EP3908326A4 (enExample)
JP (1) JP7660062B2 (enExample)
KR (1) KR20210107037A (enExample)
CN (1) CN113646005A (enExample)
AU (1) AU2019401314A1 (enExample)
BR (1) BR112021011143A2 (enExample)
CA (1) CA3123600A1 (enExample)
CL (1) CL2021001624A1 (enExample)
CO (1) CO2021008538A2 (enExample)
IL (1) IL284185B2 (enExample)
JO (1) JOP20210160A1 (enExample)
MX (1) MX2021007600A (enExample)
PE (1) PE20211581A1 (enExample)
PH (1) PH12021551341A1 (enExample)
SG (1) SG11202105907QA (enExample)
WO (1) WO2020132455A1 (enExample)

Families Citing this family (62)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12338451B2 (en) 2019-05-03 2025-06-24 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
CN116249712A (zh) 2020-04-15 2023-06-09 沃雅戈治疗公司 Tau结合化合物
BR112022022212A2 (pt) * 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
AU2021273447A1 (en) 2020-05-13 2022-12-08 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
JP2023534037A (ja) 2020-07-13 2023-08-07 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 シャルコー・マリー・トゥース病の治療に有用な組成物
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
KR20230093241A (ko) 2020-07-27 2023-06-27 보이저 테라퓨틱스, 인크. 글루코실세라미데이스 베타 결핍증과 관련된 신경 장애의 치료를 위한 조성물 및 방법
KR20230118075A (ko) * 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
AU2021392642A1 (en) 2020-12-01 2023-06-22 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022198079A1 (en) * 2021-03-19 2022-09-22 Mayo Foundation For Medical Education And Research Methods and materials for treating propionic acidemia
WO2022221193A1 (en) 2021-04-12 2022-10-20 Affinia Therapeutics Inc. Recombinant aav for treatment of neural disease
WO2023113806A1 (en) 2021-12-16 2023-06-22 Affinia Therapeutics, Inc. Recombinant aav for treatment of neural disease
MX2023012513A (es) 2021-04-23 2023-12-15 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen.
TW202309066A (zh) 2021-04-27 2023-03-01 賓州大學委員會 衍生自豬的腺相關病毒衣殼及其用途
US20250223376A1 (en) 2021-09-20 2025-07-10 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
US20240384298A1 (en) 2021-10-02 2024-11-21 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
EP4419690A2 (en) * 2021-10-18 2024-08-28 The Trustees of The University of Pennsylvania Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
WO2023087019A2 (en) * 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20250011372A1 (en) 2021-11-17 2025-01-09 Voyager Thrapeutics, Inc. Aav capsid variants and uses thereof
US20250034559A1 (en) 2021-11-17 2025-01-30 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
US20250346920A1 (en) 2022-01-25 2025-11-13 Voyager Therapeutics, Inc. Baculovirus expression system
IL314156A (en) 2022-02-08 2024-09-01 Voyager Therapeutics Inc Adeno-associated virus capsid variants and their uses
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
CN119317645A (zh) 2022-04-06 2025-01-14 宾夕法尼亚州大学信托人 用于治疗her2阳性转移性乳腺癌及其他癌症的组合物和方法
US20250295809A1 (en) 2022-05-13 2025-09-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
IL316719A (en) 2022-06-02 2024-12-01 Voyager Therapeutics Inc Adeno-associated virus capsid variants and their uses
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
EP4543473A1 (en) 2022-06-22 2025-04-30 Voyager Therapeutics, Inc. Tau binding compounds
TW202417467A (zh) 2022-06-28 2024-05-01 美商航海家醫療公司 Aav蛋白殼變異體及其用途
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
JP2025520967A (ja) * 2022-07-08 2025-07-03 オスペダーレ サン ラファエレ エス.アール.エル 導入遺伝子カセット
EP4565597A2 (en) 2022-08-03 2025-06-11 Voyager Therapeutics, Inc. Compositions and methods for crossing the blood brain barrier
AU2023342086A1 (en) 2022-09-15 2025-03-13 Voyager Therapeutics, Inc. Tau binding compounds
AU2023351041A1 (en) * 2022-09-26 2025-03-13 Encoded Therapeutics, Inc. Elements for de-targeting gene expression in dorsal root ganglion and/or liver
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
AU2023417630A1 (en) 2022-12-29 2025-05-15 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
AU2024266140A1 (en) 2023-05-02 2025-09-11 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2024229389A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
CN121127597A (zh) 2023-05-03 2025-12-12 沃雅戈治疗公司 用于治疗与葡糖苷酰鞘氨醇酶β1缺陷相关的病症的组合物及方法
TW202444917A (zh) 2023-05-04 2024-11-16 美商航海家醫療公司 Aav衣殼變異體及其用途
CN121175424A (zh) * 2023-05-12 2025-12-19 上海金珂博生物技术有限公司 一种携带smn基因表达框的病毒载体及其用途
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025122530A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2025122634A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025217023A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Gene therapy for gangliosidosis type 2 (gm2)
WO2025235734A2 (en) 2024-05-09 2025-11-13 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1838870A2 (en) * 2004-12-29 2007-10-03 Exiqon A/S NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF MICRORNAS AND THEIR TARGET MRNAs
HUE027013T2 (en) * 2005-05-27 2016-10-28 Ospedale San Raffaele Srl A gene vector containing MI-RNA
US20100286044A1 (en) * 2005-12-29 2010-11-11 Exiqon A/S Detection of tissue origin of cancer
US20100240058A1 (en) * 2007-11-23 2010-09-23 Panagene Inc. MicroRNA Antisense PNAs, Compositions Comprising the Same, and Methods for Using and Evaluating the Same
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
DK2826860T3 (en) * 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
FR3004463A1 (fr) * 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
KR20180121899A (ko) * 2016-02-03 2018-11-09 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 I형 점액다당류증을 치료하기 위한 유전자 요법
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي

Also Published As

Publication number Publication date
PH12021551341A1 (en) 2021-12-13
BR112021011143A2 (pt) 2022-01-25
EP3908326A1 (en) 2021-11-17
CL2021001624A1 (es) 2021-11-26
CO2021008538A2 (es) 2021-07-19
WO2020132455A1 (en) 2020-06-25
IL284185B1 (en) 2024-12-01
CN113646005A (zh) 2021-11-12
MX2021007600A (es) 2021-08-11
IL284185A (en) 2021-08-31
JP2022517174A (ja) 2022-03-07
JOP20210160A1 (ar) 2023-01-30
SG11202105907QA (en) 2021-07-29
JP7660062B2 (ja) 2025-04-10
CA3123600A1 (en) 2020-06-25
US20210077553A1 (en) 2021-03-18
AU2019401314A1 (en) 2021-06-24
EP3908326A4 (en) 2022-10-26
KR20210107037A (ko) 2021-08-31
IL284185B2 (en) 2025-04-01

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