[go: up one dir, main page]

MX356525B - Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia. - Google Patents

Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia.

Info

Publication number
MX356525B
MX356525B MX2013009604A MX2013009604A MX356525B MX 356525 B MX356525 B MX 356525B MX 2013009604 A MX2013009604 A MX 2013009604A MX 2013009604 A MX2013009604 A MX 2013009604A MX 356525 B MX356525 B MX 356525B
Authority
MX
Mexico
Prior art keywords
choroideremia
gene therapy
aav
vectors
prevention
Prior art date
Application number
MX2013009604A
Other languages
English (en)
Other versions
MX2013009604A (es
Inventor
Maclaren Robert
Seabra Miguel
John During Matthew
Original Assignee
Isis Innovation
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=43881504&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=MX356525(B) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Isis Innovation filed Critical Isis Innovation
Publication of MX2013009604A publication Critical patent/MX2013009604A/es
Publication of MX356525B publication Critical patent/MX356525B/es

Links

Classifications

    • 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
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/12Viral antigens
    • 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
    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1085Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • 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
    • 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
    • C12N2750/14171Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La presente invención se refiere a terapia de genes para tratamiento o prevención de coroideremia.
MX2013009604A 2011-02-22 2012-02-21 Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia. MX356525B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB1103062.4A GB201103062D0 (en) 2011-02-22 2011-02-22 Method
PCT/GB2012/050376 WO2012114090A1 (en) 2011-02-22 2012-02-21 Aav -vectors for use in gene therapy of choroideremia

Publications (2)

Publication Number Publication Date
MX2013009604A MX2013009604A (es) 2013-09-16
MX356525B true MX356525B (es) 2018-06-01

Family

ID=43881504

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018006733A MX388146B (es) 2011-02-22 2012-02-21 Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia.
MX2013009604A MX356525B (es) 2011-02-22 2012-02-21 Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2018006733A MX388146B (es) 2011-02-22 2012-02-21 Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia.

Country Status (18)

Country Link
US (4) US9834788B2 (es)
EP (2) EP3428283A1 (es)
JP (4) JP2014512171A (es)
KR (3) KR102139203B1 (es)
CN (2) CN107881198A (es)
AU (2) AU2012220404B2 (es)
BR (1) BR112013021318B1 (es)
CA (1) CA2827975C (es)
DK (1) DK2678435T3 (es)
ES (1) ES2676550T5 (es)
GB (1) GB201103062D0 (es)
HK (2) HK1258917A1 (es)
IL (1) IL228031B (es)
MX (2) MX388146B (es)
PL (1) PL2678435T3 (es)
PT (1) PT2678435T (es)
SG (1) SG192911A1 (es)
WO (1) WO2012114090A1 (es)

Families Citing this family (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
EP3030665B9 (en) 2013-08-05 2023-10-04 Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp
MA39082B2 (fr) 2013-12-06 2021-11-30 Inst Nat Sante Rech Med Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
LT3265568T (lt) * 2015-03-06 2020-08-25 Massachusetts Eye & Ear Infirmary Genų augmentacijos terapijos, skirtos paveldimai tinklainės degeneracijai, sukeltai prpf31 geno mutacijos
MX2018007230A (es) * 2015-12-14 2018-11-09 Univ Pennsylvania Terapia genica para trastornos oculares.
US20190017096A1 (en) * 2016-03-10 2019-01-17 Nightstarx Limited Prenylation assay
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2018014152A (es) 2016-05-18 2019-03-28 Voyager Therapeutics Inc Composiciones y métodos para tratar la enfermedad de huntington.
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10633689B2 (en) 2016-11-07 2020-04-28 Spark Therapeutics, Inc. Rab Escort Protein potency assay
AU2018207259B2 (en) * 2017-01-10 2023-11-09 Children's Medical Research Institute Polynucleotides and vectors for the expression of transgenes
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2018232149A1 (en) 2017-06-14 2018-12-20 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Aavを送達するための組成物および方法
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
KR20200078513A (ko) * 2017-10-03 2020-07-01 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US20200341015A1 (en) 2017-10-17 2020-10-29 Nightstarx Ltd. Prenylation assay
AU2018378622A1 (en) * 2017-12-06 2020-05-28 Ovid Therapeutics Inc. Use of miR101 or miR128 in the treatment of seizure disorders
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
BR112020015798A2 (pt) 2018-02-01 2021-03-09 Homology Medicines, Inc. Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
EP3775234A1 (en) * 2018-04-05 2021-02-17 NightstaRx Limited Aav compositions, methods of making and methods of use
NZ759802A (en) * 2018-05-07 2025-08-29 Univ North Carolina Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
AU2019346655B2 (en) 2018-09-28 2025-03-13 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US20220193207A1 (en) * 2019-04-30 2022-06-23 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of pompe disease
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
MX2023001998A (es) 2020-08-17 2023-05-04 Massachusetts Inst Technology Enfoques de terapia con el gen shank3.
WO2022109247A1 (en) 2020-11-20 2022-05-27 Biogen Ma Inc. Viral vector potency assay
EP4482970A1 (en) 2022-02-23 2025-01-01 Massachusetts Institute of Technology Methods for upregulating shank3 expression
WO2024033834A1 (en) 2022-08-11 2024-02-15 Institute Of Molecular And Clinical Opthalmology Basel (Iob) Promoters for specific expression of genes in cone photoreceptors
KR20250051697A (ko) 2022-08-11 2025-04-17 인스티튜트 오브 몰레큘러 앤드 클리니컬 오프탈몰로지 바젤 (아이오비) 인간 원추형 광수용체 광유전학 작제물
WO2024233422A1 (en) 2023-05-05 2024-11-14 Massachusetts Institute Of Technology Shank3 gene therapy approaches
WO2025090942A1 (en) * 2023-10-26 2025-05-01 University Of Notre Dame Du Lac Gene therapy for treatment of neurometabolic disease

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0856585A1 (en) 1997-01-29 1998-08-05 Introgene B.V. A conditional replication and expression system
JP2003526377A (ja) * 2000-03-14 2003-09-09 ニユーロロジクス・インコーポレーテツド キメラキャプシドベクターの製造
CA2442670A1 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
US20040208847A1 (en) * 2003-03-28 2004-10-21 Fabienne Rolling Method and vectors for selectively transducing retinal pigment epithelium cells
KR20070114761A (ko) * 2005-02-23 2007-12-04 디나벡크 가부시키가이샤 Siv­pedf 벡터를 사용한 안조직 세포에 있어서의아포토시스 변성을 동반하는 질환의 치료제
JP2008539698A (ja) * 2005-04-29 2008-11-20 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 転写後レベルでの核酸発現調節のための方法および組成物
US20090022731A1 (en) * 2006-08-25 2009-01-22 Wyeth Arthritis-associated B cell gene expression
CN101854947A (zh) * 2007-08-16 2010-10-06 圣文森特医院悉尼有限公司 用于调节巨噬细胞抑制因子(mic-1)活性的药剂和方法
WO2009097129A1 (en) 2008-01-29 2009-08-06 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
AU2009212309B2 (en) * 2008-02-07 2014-04-03 Ceregene, Inc. Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein
HUE072183T2 (hu) * 2009-05-02 2025-10-28 Genzyme Corp Génterápia neurodegeneratív betegségekre
US9169494B2 (en) * 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method

Also Published As

Publication number Publication date
JP2017221192A (ja) 2017-12-21
BR112013021318B1 (pt) 2021-12-07
PL2678435T3 (pl) 2018-11-30
JP2023036618A (ja) 2023-03-14
ES2676550T5 (es) 2024-06-27
ES2676550T3 (es) 2018-07-20
US20230332176A1 (en) 2023-10-19
MX388146B (es) 2025-03-19
EP2678435B2 (en) 2024-02-07
BR112013021318A2 (pt) 2016-10-25
EP3428283A1 (en) 2019-01-16
KR20140044793A (ko) 2014-04-15
IL228031B (en) 2018-02-28
PT2678435T (pt) 2018-07-17
CA2827975C (en) 2023-06-13
DK2678435T3 (en) 2018-08-27
CN107881198A (zh) 2018-04-06
AU2012220404B2 (en) 2017-04-20
US20210032656A1 (en) 2021-02-04
KR102299430B1 (ko) 2021-09-07
SG192911A1 (en) 2013-09-30
HK1249548A1 (zh) 2018-11-02
IL228031A0 (en) 2013-09-30
WO2012114090A1 (en) 2012-08-30
EP2678435B1 (en) 2018-05-16
HK1258917A1 (en) 2019-11-22
KR20210112407A (ko) 2021-09-14
JP2014512171A (ja) 2014-05-22
KR102139203B1 (ko) 2020-07-29
US20180135078A1 (en) 2018-05-17
JP2020054345A (ja) 2020-04-09
EP2678435A1 (en) 2014-01-01
US20140107185A1 (en) 2014-04-17
AU2012220404A1 (en) 2013-09-12
MX2013009604A (es) 2013-09-16
CN103562396A (zh) 2014-02-05
KR20200092422A (ko) 2020-08-03
GB201103062D0 (en) 2011-04-06
AU2017206254A1 (en) 2017-08-10
US9834788B2 (en) 2017-12-05
CA2827975A1 (en) 2012-08-30

Similar Documents

Publication Publication Date Title
MX356525B (es) Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia.
PH12019502292A1 (en) Methods and compositions for treatment of a genetic condition
IN2014DN00288A (es)
IN2014DN00286A (es)
IN2014MN00093A (es)
PH12015500525A1 (en) Formulations of enzalutamide
IN2014MN01378A (es)
HUE041553T2 (hu) Lizoszomális tárolási betegségek (LSD) kezelése és génterápia
IN2014DN06792A (es)
PL2879672T3 (pl) Terapia skojarzona w leczeniu stwardnienia rozsianego
SI2804599T1 (sl) Kombinirana terapija za zdravljenje raka
SG10201703677VA (en) Methods of treating s. aureus-associated diseases
IN2014DN00254A (es)
IL275636A (en) Medical combination for cancer treatment
ZA201307058B (en) Use of polyaminoisoprenyl derrivatives in antobiotic or antisertic treatment
UA117567C2 (uk) Композиція, яка стимулює лактацію, на основі фосфатидилсерину
EP3449917A3 (en) Tapentadol for preventing chronification of pain
PT2686005T (pt) Composições tópicas compreendendo diamina oxidase para o tratamento ou prevenção de doenças associadas com elevados níveis de histamina que envolvem um aumento na dor
HK40043513B (en) Interval therapy for the treatment of eye diseases
IL233495A0 (en) Combination for cancer treatment
AU2012900286A0 (en) "Methods of treatment or prophylaxis"
AU2012902296A0 (en) Compositions for Therapy and Methods of Treatment

Legal Events

Date Code Title Description
FG Grant or registration