CL2020001060A1 - Métodos y materiales para terapia génica con nt-3. - Google Patents

Métodos y materiales para terapia génica con nt-3.

Info

Publication number
CL2020001060A1
CL2020001060A1 CL2020001060A CL2020001060A CL2020001060A1 CL 2020001060 A1 CL2020001060 A1 CL 2020001060A1 CL 2020001060 A CL2020001060 A CL 2020001060A CL 2020001060 A CL2020001060 A CL 2020001060A CL 2020001060 A1 CL2020001060 A1 CL 2020001060A1
Authority
CL
Chile
Prior art keywords
methods
gene therapy
raav
materials
muscle
Prior art date
Application number
CL2020001060A
Other languages
English (en)
Inventor
Zarife Sahenk
Original Assignee
Res Inst Nationwide Childrens Hospital
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 Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of CL2020001060A1 publication Critical patent/CL2020001060A1/es

Links

Classifications

    • 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
    • C07K14/475—Growth factors; Growth regulators
    • 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
    • A61K39/12—Viral antigens
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • 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
    • 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
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente descripción se refiere a la administración del virus adenoasociado recombinante (rAAV) de un polinucleótido de neurotrofina 3 (NT-3). La descripción proporciona rAAV y métodos de uso del rAAV para terapia génica con NT-3 para mejorar la fuerza muscular, estimular el crecimiento muscular y para tratar trastornos de desgaste muscular, tales como distrofia muscular y neuropatía de Charcot-Marie-Tooth.
CL2020001060A 2017-10-20 2020-04-20 Métodos y materiales para terapia génica con nt-3. CL2020001060A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762574828P 2017-10-20 2017-10-20
US201862676687P 2018-05-25 2018-05-25
US201862741335P 2018-10-04 2018-10-04

Publications (1)

Publication Number Publication Date
CL2020001060A1 true CL2020001060A1 (es) 2020-11-13

Family

ID=66173875

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2020001060A CL2020001060A1 (es) 2017-10-20 2020-04-20 Métodos y materiales para terapia génica con nt-3.

Country Status (16)

Country Link
US (1) US11926653B2 (es)
EP (1) EP3697916A4 (es)
JP (2) JP2021500029A (es)
KR (1) KR20200083495A (es)
CN (1) CN111433367A (es)
AU (1) AU2018351528A1 (es)
BR (1) BR112020007790A2 (es)
CA (1) CA3079416A1 (es)
CL (1) CL2020001060A1 (es)
CO (1) CO2020006148A2 (es)
IL (1) IL273976A (es)
MA (1) MA50412A (es)
MX (1) MX2020004035A (es)
SG (1) SG11202003285PA (es)
TW (1) TW201923078A (es)
WO (1) WO2019079755A1 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201907882D0 (en) * 2019-06-03 2019-07-17 Cyprus Foundation For Muscular Dystrophy Res Methods
US20220324921A1 (en) * 2019-09-03 2022-10-13 University Of Cincinnati Methods and compositions for the treatment of als
WO2021211713A1 (en) * 2020-04-14 2021-10-21 Research Institute At Nationwide Children's Hospital Treatment of charcot-marie-tooth axonal type 2d using nt-3 gene therapy
WO2021211705A1 (en) * 2020-04-14 2021-10-21 Research Institute At Nationwide Children's Hospital Treatment of sarcopenia using nt-3 gene therapy
CN111514279B (zh) * 2020-06-16 2023-07-18 温州医科大学附属第二医院（温州医科大学附属育英儿童医院） 神经营养因子3在制备治疗男性性腺功能减退症的药物中的应用
EP4108263A3 (en) * 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
CN115838725B (zh) * 2022-12-30 2023-09-08 广州派真生物技术有限公司 在哺乳动物心脏中特异性启动基因的启动子序列及其应用

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
CA2176117C (en) 1993-11-09 2006-01-03 Terence R. Flotte Generation of high titers of recombinant aav vectors
US5837484A (en) 1993-11-09 1998-11-17 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
US6143548A (en) 1995-08-30 2000-11-07 Genzyme Corporation Chromatographic purification of adeno-associated virus (AAV)
EP0850313B8 (en) 1995-09-08 2009-07-29 Genzyme Corporation Improved aav vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
AU723497C (en) 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The Method for recombinant adeno-associated virus-directed gene therapy
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
WO2001083692A2 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
AU2002360291A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
WO2010089706A1 (en) * 2009-02-04 2010-08-12 Ecole Polytechnique Federale De Lausanne (Epfl) Use of igf-1 and nt-3 in the treatment of x-linked adrenoleukodystrophy
MX340102B (es) 2010-01-28 2016-06-24 The Children's Hospital Of Philadelphia * Una plataforma de fabricacion ajustable en escala, para la purificacion de vector viral y vectores virales asi purificados para el uso en terapia de genes.
EP3290055A3 (en) 2011-07-25 2018-05-30 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
MX2016010781A (es) 2014-02-18 2017-02-15 Univ Duke Composiciones para la inactivacion de la replicacion de virus y metodos de fabricacion y uso de las mismas.
KR20160068558A (ko) * 2014-12-05 2016-06-15 삼성바이오에피스 주식회사 마우스 cmv 프로모터를 포함하는 융합 폴리뉴클레오타이드 및 이를 이용한 목적 폴리펩타이드의 생산 방법
WO2016100963A1 (en) 2014-12-19 2016-06-23 Research Institute At Nationwide Children's Hospital Pyruvate compounds for treatment of peripheral neuropathy
MX2018003234A (es) 2015-09-17 2018-09-07 Res Institute At Nationwide Children´S Hospital Metodos y materiales para la terapia genica galgt2.
GB2547179A (en) 2015-10-26 2017-08-16 Quethera Ltd Genetic construct
KR20180081566A (ko) 2015-11-12 2018-07-16 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 근이영양증을 치료하는 방법
CN116064423A (zh) 2016-03-25 2023-05-05 普瑞菲根公司 用于递送nt3和治疗cipn的hsv载体
CN109563496B (zh) 2016-03-30 2023-08-29 星火治疗有限公司 用于重组蛋白和/或病毒载体生产的细胞系
JP7079733B2 (ja) * 2016-04-15 2022-06-02 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
WO2018170408A1 (en) 2017-03-17 2018-09-20 Research Institute At Nationwide Children's Hospital, Inc. Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

Also Published As

Publication number Publication date
IL273976A (en) 2020-05-31
MX2020004035A (es) 2020-11-09
KR20200083495A (ko) 2020-07-08
EP3697916A1 (en) 2020-08-26
US20200339960A1 (en) 2020-10-29
SG11202003285PA (en) 2020-05-28
WO2019079755A1 (en) 2019-04-25
JP2023166580A (ja) 2023-11-21
JP2021500029A (ja) 2021-01-07
TW201923078A (zh) 2019-06-16
CN111433367A (zh) 2020-07-17
US11926653B2 (en) 2024-03-12
CO2020006148A2 (es) 2020-08-10
MA50412A (fr) 2020-08-26
BR112020007790A2 (pt) 2020-10-20
AU2018351528A1 (en) 2020-05-14
EP3697916A4 (en) 2021-08-11
CA3079416A1 (en) 2019-04-25

Similar Documents

Publication Publication Date Title
CO2020006148A2 (es) Métodos y materiales para terapia génica con nt-3
DOP2016000237A (es) Terapia genica para la retinitis pigmentaria
BR112018008358A2 (pt) ?composições e métodos para terapia relacionada à microbiota fecal?
CO2019011250A2 (es) Administración de vectores del virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscular
MX2018006840A (es) Focalizacion de peptidos para dirigir virus adenoasociados.
CR20190376A (es) Formulaciones de anticuerpos de her2 subcutáneas
MX2019007873A (es) Terapia génica para tratar la enfermedad de wilson.
CO2018012082A2 (es) Administración de vectores del virus adenoasociado de β-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular
CR20170407A (es) Mejora del suministro de partículas virales al cuerpo estriado y al córtex
WO2016057975A3 (en) Guided injections for aav gene transfer to muscle
MX2018012537A (es) Terapia de genes para tratar hemofilia a.
ECSP21090478A (es) Amidas de pirazolo-piridina sustituidas y su uso como moduladores del receptor glun2b
BR112019022908A2 (pt) Micropartícula de agregação sólida, sólido liofilizado ou reconstituível de micropartículas de agregação, método para tratamento de um distúrbio ocular, e, uso das micropartículas de agregação sólidas
MX2020004716A (es) Método seguro y eficaz para tratar la artritis psoriásica con el anticuerpo específico anti-il23.
CL2021002049A1 (es) Administración de virus adenoasociado de polinucleótido de cln3.
CL2017001595A1 (es) Composiciones de vacuna para el virus del dengue y métodos de uso de las mismas
CL2021002051A1 (es) Administración del virus adenoasociado de polinucleótido de cln6.
EA201591902A1 (ru) Способы и композиции, использующие 4-амино-2-(2,6-диоксо-пиперидин-3-ил)изоиндолин-1,3-дион для лечения и контроля злокачественных опухолей центральной нервной системы
MX2018007307A (es) Conjugados de peptidos y oligonucleotidos.
AR113395A1 (es) Métodos y materiales para la terapia génica nt-3
EA202090989A1 (ru) Способы и материалы для направленной на nt-3 генной терапии
BR112017022621A2 (pt) liberação do gene de smad7 como uma substância terapêutica
EA202090480A1 (ru) Композиция для контролируемой стимуляции яичников
AR118192A1 (es) ADMINISTRACIÓN DE b-SARCOGLICANO CON UN VECTOR DE VIRUS ADENOASOCIADO Y TRATAMIENTO DE LA DISTROFIA MUSCULAR
RU2014120624A (ru) Способ лечения инсульта