MA39390B2 - Thérapie génique pour le traitement de la rétinite pigmentaire - Google Patents

Thérapie génique pour le traitement de la rétinite pigmentaire

Info

Publication number
MA39390B2
MA39390B2 MA39390A MA39390A MA39390B2 MA 39390 B2 MA39390 B2 MA 39390B2 MA 39390 A MA39390 A MA 39390A MA 39390 A MA39390 A MA 39390A MA 39390 B2 MA39390 B2 MA 39390B2
Authority
MA
Morocco
Prior art keywords
treatment
retinitis pigmentosa
gene therapy
viral particles
particles
Prior art date
Application number
MA39390A
Other languages
English (en)
Other versions
MA39390A1 (fr
Inventor
Catherine O'riordan
Matthew Adamowicz
Original Assignee
Genzyme Corp
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=52829343&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=MA39390(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of MA39390A1 publication Critical patent/MA39390A1/fr
Publication of MA39390B2 publication Critical patent/MA39390B2/fr

Links

Classifications

    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • 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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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 N.A.
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses
    • 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
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • 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

Abstract

La présente invention concerne des procédés pour le traitement de la rétinite pigmentaire au moyen de particules d'aav codant mir -708. Dans un aspect de l'invention, des particules virales sont administrées dans l'œil d'un sujet humain, par exemple, par injection sous-rétinienne. Des particules virales comprenant des capsides d'aav5 ou des mutants de ceux-ci sont envisagées.
MA39390A 2014-03-21 2015-03-20 Thérapie génique pour le traitement de la rétinite pigmentaire MA39390B2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461969027P 2014-03-21 2014-03-21
PCT/US2015/021896 WO2015143418A2 (fr) 2014-03-21 2015-03-20 Thérapie génique pour le traitement de la rétinite pigmentaire

Publications (2)

Publication Number Publication Date
MA39390A1 MA39390A1 (fr) 2017-12-29
MA39390B2 true MA39390B2 (fr) 2022-04-29

Family

ID=52829343

Family Applications (1)

Application Number Title Priority Date Filing Date
MA39390A MA39390B2 (fr) 2014-03-21 2015-03-20 Thérapie génique pour le traitement de la rétinite pigmentaire

Country Status (36)

Country Link
US (3) US10383953B2 (fr)
EP (3) EP3119437B1 (fr)
JP (3) JP6669664B2 (fr)
KR (2) KR20160127832A (fr)
CN (2) CN115252823A (fr)
AR (1) AR099837A1 (fr)
AU (2) AU2015230942B2 (fr)
CA (1) CA2943185A1 (fr)
CL (1) CL2016002333A1 (fr)
CR (1) CR20160480A (fr)
DK (2) DK3628334T3 (fr)
DO (1) DOP2016000237A (fr)
EA (1) EA201691891A1 (fr)
EC (1) ECSP16083000A (fr)
ES (2) ES2760263T3 (fr)
FI (1) FI3628334T3 (fr)
HR (2) HRP20231077T1 (fr)
HU (2) HUE046454T2 (fr)
IL (3) IL247543B (fr)
LT (2) LT3119437T (fr)
MA (1) MA39390B2 (fr)
MX (2) MX2016012201A (fr)
MY (1) MY190726A (fr)
NZ (1) NZ724622A (fr)
PE (1) PE20161252A1 (fr)
PH (1) PH12016501684A1 (fr)
PL (2) PL3119437T3 (fr)
PT (2) PT3628334T (fr)
RS (2) RS59634B1 (fr)
SG (3) SG10201912968WA (fr)
SI (2) SI3119437T1 (fr)
TW (2) TWI706789B (fr)
UA (1) UA120050C2 (fr)
UY (1) UY36044A (fr)
WO (1) WO2015143418A2 (fr)
ZA (1) ZA201605924B (fr)

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WO2016130589A2 (fr) 2015-02-10 2016-08-18 Genzyme Corporation Variant d'arni
US10994026B2 (en) 2015-12-03 2021-05-04 Friedrich Miescher Institute For Biomedical Research SynP160, a promoter for the specific expression of genes in rod photoreceptors
JP7075341B2 (ja) 2015-12-03 2022-05-25 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ SynP162、桿体光受容器における遺伝子の特異的発現のためのプロモーター
WO2017093566A1 (fr) 2015-12-04 2017-06-08 Universite Pierre Et Marie Curie (Paris 6) Promoteurs et leurs utilisations
US11118185B2 (en) * 2016-03-01 2021-09-14 University Of Florida Research Foundation, Incorporated AAV vectors for treatment of dominant retinitis pigmentosa
BR112019000057A2 (pt) * 2016-07-05 2019-04-02 The Johns Hopkins University composições e métodos com base em crispr/cas9 para o tratamento de degeneração de retina
BR112020005569A2 (pt) * 2017-09-22 2020-10-06 Genzyme Corporation rna de interferência (rnai) variante
KR20200057051A (ko) * 2017-09-27 2020-05-25 시질론 테라퓨틱스, 인크. 활성 세포를 포함하는 방법, 조성물 및 이식 가능한 요소
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EP4186921A1 (fr) * 2018-03-23 2023-05-31 The Trustees of Columbia University in the City of New York Édition de gènes pour maladies autosomiques dominantes
CA3102095A1 (fr) * 2018-06-01 2019-12-05 University Of Florida Research Foundation, Incorporated Compositions et methodes pour le traitement de la retinite pigmentaire dominante
CN111518813B (zh) * 2019-02-03 2023-04-28 武汉纽福斯生物科技有限公司 视紫红质的编码序列、其表达载体构建及其应用
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Publication number Publication date
EA201691891A1 (ru) 2017-01-30
AU2015230942B2 (en) 2020-11-19
IL247543B (en) 2020-07-30
IL284741B2 (en) 2023-02-01
PT3628334T (pt) 2023-09-26
MY190726A (en) 2022-05-12
UA120050C2 (uk) 2019-09-25
CN106456660B (zh) 2022-05-31
AR099837A1 (es) 2016-08-24
SG10201912968WA (en) 2020-02-27
NZ724622A (en) 2022-05-27
US20220054657A1 (en) 2022-02-24
PH12016501684A1 (en) 2016-10-03
PL3119437T3 (pl) 2020-04-30
PE20161252A1 (es) 2016-11-30
ECSP16083000A (es) 2017-02-24
RS64611B1 (sr) 2023-10-31
CL2016002333A1 (es) 2017-05-26
IL247543A0 (en) 2016-11-30
HRP20192141T1 (hr) 2020-02-21
FI3628334T3 (fi) 2023-09-15
HUE046454T2 (hu) 2020-03-30
IL275918A (en) 2020-08-31
SG11201607005UA (en) 2016-09-29
SI3119437T1 (sl) 2020-01-31
DK3119437T3 (da) 2019-12-09
PT3119437T (pt) 2019-12-12
SI3628334T1 (sl) 2023-11-30
IL284741B (en) 2022-10-01
US10383953B2 (en) 2019-08-20
CN115252823A (zh) 2022-11-01
ES2760263T3 (es) 2020-05-13
EP4345165A2 (fr) 2024-04-03
AU2021200988A1 (en) 2021-03-11
TWI780401B (zh) 2022-10-11
KR20160127832A (ko) 2016-11-04
BR112016021017A2 (pt) 2017-10-03
LT3119437T (lt) 2019-12-27
TW202021627A (zh) 2020-06-16
UY36044A (es) 2015-10-30
TWI706789B (zh) 2020-10-11
MX2016012201A (es) 2017-01-19
CN106456660A (zh) 2017-02-22
WO2015143418A3 (fr) 2015-11-19
PL3628334T3 (pl) 2023-12-18
IL275918B (en) 2021-07-29
AU2015230942A1 (en) 2016-10-13
JP2020059737A (ja) 2020-04-16
ES2957840T3 (es) 2024-01-26
HUE063460T2 (hu) 2024-01-28
EP3119437A2 (fr) 2017-01-25
US20200046851A1 (en) 2020-02-13
MA39390A1 (fr) 2017-12-29
US11103598B2 (en) 2021-08-31
US20170173183A1 (en) 2017-06-22
TW201622752A (zh) 2016-07-01
EP3628334B1 (fr) 2023-06-28
KR20230006039A (ko) 2023-01-10
RS59634B1 (sr) 2020-01-31
WO2015143418A2 (fr) 2015-09-24
JP2017509632A (ja) 2017-04-06
CA2943185A1 (fr) 2015-09-24
EP3119437B1 (fr) 2019-09-04
HRP20231077T1 (hr) 2023-12-22
ZA201605924B (en) 2017-09-27
JP7048563B2 (ja) 2022-04-05
MX2020010694A (es) 2020-11-06
DK3628334T3 (da) 2023-10-02
DOP2016000237A (es) 2016-10-16
LT3628334T (lt) 2023-09-25
EP3628334A1 (fr) 2020-04-01
IL284741A (en) 2021-08-31
CR20160480A (es) 2016-12-14
NZ762841A (en) 2023-11-24
SG10201808218YA (en) 2018-10-30
JP6669664B2 (ja) 2020-03-18
JP2022084810A (ja) 2022-06-07

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