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MX2018004755A - Entrega de polinucleotidos dirigidos al sistema nervioso central. - Google Patents

Entrega de polinucleotidos dirigidos al sistema nervioso central.

Info

Publication number
MX2018004755A
MX2018004755A MX2018004755A MX2018004755A MX2018004755A MX 2018004755 A MX2018004755 A MX 2018004755A MX 2018004755 A MX2018004755 A MX 2018004755A MX 2018004755 A MX2018004755 A MX 2018004755A MX 2018004755 A MX2018004755 A MX 2018004755A
Authority
MX
Mexico
Prior art keywords
delivery
nervous system
central nervous
targeting polynucleotides
system targeting
Prior art date
Application number
MX2018004755A
Other languages
English (en)
Inventor
Wen- Yee Sah Dinah
Hou Jinzhao
Goulet Martin
Philip KELLS Adrian
Zhou Pengcheng
Robert Stewart Gregory
Original Assignee
Voyager Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of MX2018004755A publication Critical patent/MX2018004755A/es

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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M25/00—Catheters; Hollow probes
    • A61M25/0021—Catheters; Hollow probes characterised by the form of the tubing
    • A61M25/0023—Catheters; Hollow probes characterised by the form of the tubing by the form of the lumen, e.g. cross-section, variable diameter
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M5/00—Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/14—Infusion devices, e.g. infusing by gravity; Blood infusion; Accessories therefor
    • A61M5/142—Pressure infusion, e.g. using pumps
    • A61M5/14244—Pressure infusion, e.g. using pumps adapted to be carried by the patient, e.g. portable on the body
    • A61M5/14276—Pressure infusion, e.g. using pumps adapted to be carried by the patient, e.g. portable on the body specially adapted for implantation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Anesthesiology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Vascular Medicine (AREA)
  • Neurosurgery (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Infusion, Injection, And Reservoir Apparatuses (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La invención se refiere a composiciones y métodos para la preparación, administración, fabricación y uso terapéutico de partículas virales para el tratamiento de trastornos del SNC.
MX2018004755A 2015-10-29 2016-10-28 Entrega de polinucleotidos dirigidos al sistema nervioso central. MX2018004755A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562248220P 2015-10-29 2015-10-29
US201562248223P 2015-10-29 2015-10-29
US201662279420P 2016-01-15 2016-01-15
PCT/US2016/059302 WO2017075338A2 (en) 2015-10-29 2016-10-28 Delivery of central nervous system targeting polynucleotides

Publications (1)

Publication Number Publication Date
MX2018004755A true MX2018004755A (es) 2018-12-19

Family

ID=58631154

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018004755A MX2018004755A (es) 2015-10-29 2016-10-28 Entrega de polinucleotidos dirigidos al sistema nervioso central.

Country Status (7)

Country Link
US (1) US20190055578A1 (es)
EP (1) EP3368065A4 (es)
AU (1) AU2016343979A1 (es)
CA (1) CA3002406A1 (es)
HK (1) HK1258413A1 (es)
MX (1) MX2018004755A (es)
WO (1) WO2017075338A2 (es)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EP3294398A4 (en) * 2015-05-11 2019-01-09 Alcyone Lifesciences, Inc. SYSTEM AND METHOD FOR THE ADMINISTRATION OF MEDICAMENTS
EP3448987A4 (en) * 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF DISEASES
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
EA201992358A1 (ru) 2017-04-03 2020-03-24 Инкоудид Терапьютикс, Инк. Тканеселективная экспрессия трансгена
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN111542549A (zh) 2017-10-03 2020-08-14 普利维尔治疗公司 用于溶酶体障碍的基因疗法
EP3692151A4 (en) 2017-10-03 2021-07-14 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISORDERS
KR102697811B1 (ko) 2017-10-03 2024-08-22 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
WO2019109051A1 (en) 2017-12-01 2019-06-06 Encoded Therapeutics, Inc. Engineered dna binding proteins
EP3856913A4 (en) 2018-09-26 2022-10-26 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
SG11202103151RA (en) * 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
SG11202108504YA (en) * 2019-02-22 2021-09-29 Univ Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
MX2021012184A (es) 2019-04-10 2022-01-24 Prevail Therapeutics Inc Terapias genicas para trastornos lisosomales.
CN113853209B (zh) * 2019-04-29 2024-12-13 宾夕法尼亚州大学信托人 新型aav衣壳和含有其的组合物
JP2022548270A (ja) * 2019-09-13 2022-11-17 ラセルタ セラピューティクス， インコーポレイテッド フリードライヒ運動失調症の治療のための組成物及び方法
CA3154115A1 (en) 2019-10-08 2021-04-15 Yunan ZHENG Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins
CA3170369A1 (en) 2020-03-05 2022-04-14 Michal Shahar Methods and compositions for treating cancer with immune cells
EP4284441A1 (en) * 2021-02-01 2023-12-06 BioMarin Pharmaceutical Inc. Aav production systems for aav viral particles with improved infectivity

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120253261A1 (en) * 2011-03-29 2012-10-04 Medtronic, Inc. Systems and methods for optogenetic modulation of cells within a patient
US20130136729A1 (en) * 2011-11-11 2013-05-30 University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors
EP3245221A4 (en) * 2015-01-16 2018-06-13 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US11040113B2 (en) * 2015-03-23 2021-06-22 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with Friedreich ataxia
EP3286321A4 (en) * 2015-04-24 2018-10-17 University of Florida Research Foundation, Inc. Aav vector for treatment of friedreich's ataxia

Also Published As

Publication number Publication date
WO2017075338A3 (en) 2017-06-01
WO2017075338A2 (en) 2017-05-04
CA3002406A1 (en) 2017-05-04
US20190055578A1 (en) 2019-02-21
EP3368065A4 (en) 2019-03-20
EP3368065A2 (en) 2018-09-05
AU2016343979A1 (en) 2018-05-10
HK1258413A1 (zh) 2019-11-08

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