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

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 (17)

* 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
CN113041451B (zh) * 2015-05-11 2023-06-09 亚克安娜治疗学有限公司 药物输送系统和方法
EP3448987A4 (en) * 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF DISEASES
EP3506817A4 (en) 2016-08-30 2020-07-22 The Regents of The University of California METHOD FOR BIOMEDICAL TARGETING AND RELEASE, AND DEVICES AND SYSTEMS FOR IMPLEMENTING THEM
CN117821509A (zh) 2017-04-03 2024-04-05 编码治疗公司 组织选择性转基因表达
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
WO2019070894A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMIAL DISORDERS
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
MX2020004005A (es) 2017-10-03 2020-10-05 Prevail Therapeutics Inc Terapias genicas para los trastornos lisosomales.
KR20210084459A (ko) 2018-09-26 2021-07-07 캘리포니아 인스티튜트 오브 테크놀로지 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물
JP2022500066A (ja) * 2018-09-28 2022-01-04 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
KR20220006527A (ko) 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
SG11202111373YA (en) * 2019-04-29 2021-11-29 Univ Pennsylvania Novel aav capsids and compositions containing same
EP4028027A4 (en) * 2019-09-13 2023-11-01 Lacerta Therapeutics, Inc COMPOSITIONS AND METHODS FOR TREATING FRIEDREICH AAXIA
AU2021357520A1 (en) 2020-03-05 2022-09-29 Neotx Therapeutics Ltd. Methods and compositions for treating cancer with immune cells
WO2022165004A1 (en) * 2021-02-01 2022-08-04 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
WO2016115503A1 (en) * 2015-01-16 2016-07-21 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
WO2016172659A1 (en) * 2015-04-24 2016-10-27 University Of Florida Research Foundation, Inc. Aav vector for treatment of friedreich's ataxia

Also Published As

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

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