MA50914A - Vecteurs viraux comprenant des séquences codantes de rdh12 et méthodes de traitement de dystrophies rétiniennes - Google Patents

Vecteurs viraux comprenant des séquences codantes de rdh12 et méthodes de traitement de dystrophies rétiniennes

Info

Publication number: MA50914A
Authority: MA; Morocco
Prior art keywords: drh12; coding sequences; viral vectors; treatment methods; vectors including
Prior art date: 2017-11-15

Application number

MA050914A

Other languages

English (en)

Inventor

Robin R Ali

Alexander J Smith

Debra A Thompson

Original Assignee

Univ Michigan Regents

Ucl Business Ltd

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.)

2017-11-15

Filing date

2018-11-15

Publication date

2020-09-23

2018-11-15 Application filed by Univ Michigan Regents, Ucl Business Ltd filed Critical Univ Michigan Regents

2020-09-23 Publication of MA50914A publication Critical patent/MA50914A/fr

Links

Classifications

- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/44—Oxidoreductases (1)
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/44—Oxidoreductases (1)
- A61K38/443—Oxidoreductases (1) acting on CH-OH groups as donors, e.g. glucose oxidase, lactate dehydrogenase (1.1)
- 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
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
- C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
- C12Y101/01105—Retinol dehydrogenase (1.1.1.105)
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- 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
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10041—Use of virus, viral particle or viral elements as a vector
- C12N2710/10043—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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

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

MA050914A 2017-11-15 2018-11-15 Vecteurs viraux comprenant des séquences codantes de rdh12 et méthodes de traitement de dystrophies rétiniennes MA50914A (fr)

Applications Claiming Priority (1)

Application Number	Priority Date	Filing Date	Title
US201762586624P	2017-11-15	2017-11-15

Publications (1)

Publication Number	Publication Date
MA50914A true MA50914A (fr)	2020-09-23

Family

ID=66431192

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
MA050914A MA50914A (fr)	2017-11-15	2018-11-15	Vecteurs viraux comprenant des séquences codantes de rdh12 et méthodes de traitement de dystrophies rétiniennes

Country Status (27)

Country	Link
US (2)	US20190142909A1 (fr)
EP (2)	EP3710590B1 (fr)
JP (2)	JP2021502978A (fr)
KR (1)	KR102704296B1 (fr)
CN (2)	CN117752822A (fr)
AU (1)	AU2018367606B2 (fr)
BR (1)	BR112020009828A2 (fr)
DK (1)	DK3710590T3 (fr)
EA (1)	EA202091194A1 (fr)
ES (1)	ES2983990T3 (fr)
FI (1)	FI3710590T3 (fr)
HR (1)	HRP20241073T1 (fr)
HU (1)	HUE068393T2 (fr)
IL (2)	IL274609B2 (fr)
LT (1)	LT3710590T (fr)
MA (1)	MA50914A (fr)
MX (1)	MX2020005008A (fr)
MY (1)	MY204271A (fr)
PH (1)	PH12020551319A1 (fr)
PL (1)	PL3710590T3 (fr)
PT (1)	PT3710590T (fr)
RS (1)	RS65881B1 (fr)
SG (1)	SG11202004527YA (fr)
SI (1)	SI3710590T1 (fr)
SM (1)	SMT202400321T1 (fr)
WO (1)	WO2019099696A1 (fr)
ZA (1)	ZA202003416B (fr)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
KR102526506B1 (ko)	2016-07-08	2023-05-03	더 트러스티스 오브 더 유니버시티 오브 펜실베니아	Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
EP4329882A4 (fr) *	2021-04-27	2025-02-26	The Trustees of Columbia University in the City of New York	Procédés et compositions pour réguler l'efflux de cholestérol pour prévenir, traiter ou guérir une dégénérescence maculaire
CN115725659A (zh) *	2022-04-01	2023-03-03	首都医科大学附属北京同仁医院	一种应用视网膜原位基因编辑制作非人灵长类动物先天性黑矇模型的方法
CN118453915A (zh) *	2024-04-29	2024-08-09	中国医学科学院北京协和医院	一种针对rdh12突变的基因治疗药物
CN119432856B (zh) *	2024-12-30	2025-08-12	云舟生物科技(广州)股份有限公司	核酸分子、病毒颗粒及其制备方法和应用

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
US20120172419A1 (en) *	2009-09-15	2012-07-05	Medical College Of Wisconsin Research Foundation Inc.	Reagents and methods for modulating cone photoreceptor activity
KR20190060881A (ko) *	2010-04-23	2019-06-03	유니버시티 오브 플로리다 리서치 파운데이션, 인크.	레베르 선천성 흑내장-1(lca1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법
WO2012167109A2 (fr) *	2011-06-03	2012-12-06	Massachusetts Eye & Ear Infirmary	Thérapie génique par rpgrip1 pour l'amaurose congénitale de leber
JP6199965B2 (ja) *	2012-07-11	2017-09-20	ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア	Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療
WO2015168666A2 (fr) *	2014-05-02	2015-11-05	Genzyme Corporation	Vecteurs aav pour thérapie génique de la rétine et du snc
GB201412011D0 (en) *	2014-07-04	2014-08-20	Ucl Business Plc	Treatments
PT3191139T (pt) *	2014-07-24	2020-11-05	Massachusetts Eye & Ear Infirmary	Terapia do gene rpgr para retinite pigmentosa
US10646588B2 (en) *	2015-03-11	2020-05-12	The United States Of America, As Represented By The Secretary, Department Of Health And Human Services	RP2 and RPGR vectors for treating X-linked retinitis pigmentosa
EP3289080B1 (fr) *	2015-04-30	2021-08-25	The Trustees of Columbia University in the City of New York	Thérapie génique pour maladies autosomiques dominantes
KR102526506B1 (ko)	2016-07-08	2023-05-03	더 트러스티스 오브 더 유니버시티 오브 펜실베니아	Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물

2018
- 2018-11-15 WO PCT/US2018/061319 patent/WO2019099696A1/fr not_active Ceased
- 2018-11-15 EP EP18878835.0A patent/EP3710590B1/fr active Active
- 2018-11-15 JP JP2020526473A patent/JP2021502978A/ja active Pending
- 2018-11-15 DK DK18878835.0T patent/DK3710590T3/da active
- 2018-11-15 KR KR1020207016694A patent/KR102704296B1/ko active Active
- 2018-11-15 MX MX2020005008A patent/MX2020005008A/es unknown
- 2018-11-15 CN CN202311261068.0A patent/CN117752822A/zh active Pending
- 2018-11-15 SG SG11202004527YA patent/SG11202004527YA/en unknown
- 2018-11-15 HU HUE18878835A patent/HUE068393T2/hu unknown
- 2018-11-15 IL IL274609A patent/IL274609B2/en unknown
- 2018-11-15 EA EA202091194A patent/EA202091194A1/ru unknown
- 2018-11-15 ES ES18878835T patent/ES2983990T3/es active Active
- 2018-11-15 EP EP24163209.0A patent/EP4364803A3/fr active Pending
- 2018-11-15 RS RS20240857A patent/RS65881B1/sr unknown
- 2018-11-15 LT LTEPPCT/US2018/061319T patent/LT3710590T/lt unknown
- 2018-11-15 SM SM20240321T patent/SMT202400321T1/it unknown
- 2018-11-15 MY MYPI2020002341A patent/MY204271A/en unknown
- 2018-11-15 PT PT188788350T patent/PT3710590T/pt unknown
- 2018-11-15 FI FIEP18878835.0T patent/FI3710590T3/fi active
- 2018-11-15 AU AU2018367606A patent/AU2018367606B2/en active Active
- 2018-11-15 BR BR112020009828-0A patent/BR112020009828A2/pt unknown
- 2018-11-15 IL IL308645A patent/IL308645B2/en unknown
- 2018-11-15 MA MA050914A patent/MA50914A/fr unknown
- 2018-11-15 CN CN201880086649.6A patent/CN111886340A/zh active Pending
- 2018-11-15 HR HRP20241073TT patent/HRP20241073T1/hr unknown
- 2018-11-15 US US16/192,382 patent/US20190142909A1/en not_active Abandoned
- 2018-11-15 PL PL18878835.0T patent/PL3710590T3/pl unknown
- 2018-11-15 SI SI201831135T patent/SI3710590T1/sl unknown
2020
- 2020-05-15 PH PH12020551319A patent/PH12020551319A1/en unknown
- 2020-06-08 ZA ZA2020/03416A patent/ZA202003416B/en unknown
2022
- 2022-08-09 US US17/818,623 patent/US20230190884A1/en active Pending
2023
- 2023-07-26 JP JP2023121924A patent/JP7615238B2/ja active Active

Also Published As

Publication number	Publication date
HUE068393T2 (hu)	2024-12-28
EP4364803A2 (fr)	2024-05-08
PL3710590T3 (pl)	2024-10-21
JP2023133434A (ja)	2023-09-22
AU2018367606A1 (en)	2020-06-25
MX2020005008A (es)	2020-11-06
JP2021502978A (ja)	2021-02-04
US20230190884A1 (en)	2023-06-22
EP3710590B1 (fr)	2024-07-17
SI3710590T1 (sl)	2024-10-30
PH12020551319A1 (en)	2021-09-01
ZA202003416B (en)	2024-10-30
SMT202400321T1 (it)	2024-11-15
EP3710590A4 (fr)	2021-06-23
RS65881B1 (sr)	2024-09-30
PT3710590T (pt)	2024-08-09
IL274609B2 (en)	2024-05-01
JP7615238B2 (ja)	2025-01-16
LT3710590T (lt)	2024-09-25
DK3710590T3 (da)	2024-08-12
IL308645B1 (en)	2025-09-01
IL274609B1 (en)	2024-01-01
CN117752822A (zh)	2024-03-26
AU2018367606B2 (en)	2024-08-29
KR20200088385A (ko)	2020-07-22
US20190142909A1 (en)	2019-05-16
FI3710590T3 (fi)	2024-08-06
MY204271A (en)	2024-08-20
SG11202004527YA (en)	2020-06-29
CA3082586A1 (fr)	2019-05-23
KR102704296B1 (ko)	2024-09-05
WO2019099696A1 (fr)	2019-05-23
EP4364803A3 (fr)	2024-06-05
EP3710590A1 (fr)	2020-09-23
EA202091194A1 (ru)	2020-08-14
CN111886340A (zh)	2020-11-03
IL308645B2 (en)	2026-01-01
IL274609A (en)	2020-06-30
ES2983990T3 (es)	2024-10-28
BR112020009828A2 (pt)	2020-11-17
HRP20241073T1 (hr)	2024-11-08
IL308645A (en)	2024-01-01

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EP3490582A4 (fr)	2020-04-01	Méthodes et compositions de traitement de maladie.
IL259877A (en)	2018-07-31	Adeno-type viral vectors used to treat spinal muscular atrophy
MA50914A (fr)	2020-09-23	Vecteurs viraux comprenant des séquences codantes de rdh12 et méthodes de traitement de dystrophies rétiniennes
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