CA3079553A1 - Compositions and methods for treating age-related macular degeneration - Google Patents

Compositions and methods for treating age-related macular degeneration Download PDF

Info

Publication number
CA3079553A1
CA3079553A1 CA3079553A CA3079553A CA3079553A1 CA 3079553 A1 CA3079553 A1 CA 3079553A1 CA 3079553 A CA3079553 A CA 3079553A CA 3079553 A CA3079553 A CA 3079553A CA 3079553 A1 CA3079553 A1 CA 3079553A1
Authority
CA
Canada
Prior art keywords
vector
promoter
cfh
aav
seq
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
CA3079553A
Other languages
English (en)
French (fr)
Inventor
James Mclaughlin
Adarsha KOIRALA
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Disc Medicine Inc
Original Assignee
Gemini 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 Gemini Therapeutics Inc filed Critical Gemini Therapeutics Inc
Publication of CA3079553A1 publication Critical patent/CA3079553A1/en
Abandoned legal-status Critical Current

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
    • 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
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/472Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • 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
    • C12N2750/14134Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein
    • 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
    • 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
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/60Vectors containing traps for, e.g. exons, promoters
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • 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
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/007Vectors comprising a special translation-regulating system cell or tissue specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
CA3079553A 2017-10-20 2018-10-19 Compositions and methods for treating age-related macular degeneration Abandoned CA3079553A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762574814P 2017-10-20 2017-10-20
US62/574,814 2017-10-20
PCT/US2018/056709 WO2019079718A1 (en) 2017-10-20 2018-10-19 COMPOSITIONS AND METHODS FOR TREATING AGE-RELATED MACULAR DEGENERATION

Publications (1)

Publication Number Publication Date
CA3079553A1 true CA3079553A1 (en) 2019-04-25

Family

ID=66174265

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3079553A Abandoned CA3079553A1 (en) 2017-10-20 2018-10-19 Compositions and methods for treating age-related macular degeneration

Country Status (7)

Country Link
US (1) US20210188927A1 (enExample)
EP (1) EP3697920A4 (enExample)
JP (1) JP2021500922A (enExample)
CN (1) CN111788311A (enExample)
AU (1) AU2018351491A1 (enExample)
CA (1) CA3079553A1 (enExample)
WO (1) WO2019079718A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3824095A4 (en) * 2018-07-20 2022-04-20 University of Utah Research Foundation GENE THERAPY FOR MACULATE GENERATION
US20210371480A1 (en) * 2018-10-23 2021-12-02 Gemini Therapeutics Inc. Compositions and methods for treating age-related macular degeneration and other diseases
GB201821089D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
KR20230019062A (ko) * 2019-10-22 2023-02-07 어플라이드 제네틱스 테크놀로지스 코퍼레이션 연령-관련 황반 변성 및 다른 안구 질환 및 장애의 치료를 위한 아데노-연관 바이러스 (aav) 벡터
US20220395557A1 (en) * 2019-10-23 2022-12-15 Gemini Therapeutics Sub, Inc. Methods for treating patients having cfh mutations with recombinant cfh proteins
IL299048A (en) 2020-06-14 2023-02-01 Vertex Pharma Variants of complement factor - 1 fusion structures and preparations containing them and their uses
AU2021362770A1 (en) * 2020-10-16 2022-12-08 Gyroscope Therapeutics Limited Nucleic acid encoding an anti-VEGF entity and a negative complement regulator and uses thereof for the treatment of age-related macular degeneration
WO2022094340A1 (en) * 2020-10-30 2022-05-05 Gemini Therapeutics Sub, Inc. Methods for treating inflammatory ocular diseases with complement factor h
CN115786308B (zh) * 2022-11-24 2024-05-31 江苏海洋大学 一种提高右旋糖酐酶热稳定性的方法及突变体

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005333845A (ja) * 2004-05-25 2005-12-08 Tokyoto Igaku Kenkyu Kiko ミトコンドリア局在型DsRed2及びECFP発現ベクター
EP2357257B1 (en) * 2005-02-14 2019-07-31 University of Iowa Research Foundation Methods and reagents for treatment and diagnosis of age-related macular degeneration
RU2727411C2 (ru) * 2015-09-24 2020-07-21 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Композиция и способ для лечения заболевания, опосредованного комплементом
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy

Also Published As

Publication number Publication date
WO2019079718A1 (en) 2019-04-25
JP2021500922A (ja) 2021-01-14
EP3697920A1 (en) 2020-08-26
AU2018351491A1 (en) 2020-05-07
EP3697920A4 (en) 2022-03-02
CN111788311A (zh) 2020-10-16
US20210188927A1 (en) 2021-06-24

Similar Documents

Publication Publication Date Title
US20210188927A1 (en) Compositions and methods for treating age-related macular degeneration
US10550404B2 (en) Viral vectors for the treatment of retinal dystrophy
US20210371480A1 (en) Compositions and methods for treating age-related macular degeneration and other diseases
EP3256169B1 (en) Optimized rpe65 promoter and coding sequences
KR20200086292A (ko) 바이러스 벡터를 제조하기 위한 수단 및 방법 및 그의 용도
CN113474461A (zh) 治疗bietti晶体营养不良的组合物和方法
EP3795180A1 (en) Gene therapy for ocular disorders
KR102799528B1 (ko) Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
CN115715327A (zh) 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统
US20220396807A1 (en) Methods for treating patients having cfh mutations with cfh-encoding vectors
WO2017062750A1 (en) Compositions and methods useful in treating stargardt's disease and other ocular disorders
HK40036018A (en) Compositions and methods for treating age-related macular degeneration
TW202542310A (zh) 靶向tau的人工微rna
WO2025128817A1 (en) Artificial micrornas targeting tau
HK40049894A (en) Gene therapy for ocular disorders
HK40045081A (en) Optimized rpe65 promoter and coding sequences
HK1248525B (en) Optimized rpe65 promoter and coding sequences

Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20240419