CN116271104A - 基因疗法 - Google Patents

基因疗法 Download PDF

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Publication number
CN116271104A
CN116271104A CN202310134734.8A CN202310134734A CN116271104A CN 116271104 A CN116271104 A CN 116271104A CN 202310134734 A CN202310134734 A CN 202310134734A CN 116271104 A CN116271104 A CN 116271104A
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aav
nucleotide sequence
factor
pharmaceutical composition
cfi
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CN202310134734.8A
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Chinese (zh)
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C·格伦达尔
T·芬内尔
C·霍洛伍德
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Cincona Intellectual Property Holding Co ltd
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Cincona Intellectual Property Holding Co ltd
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=55130363&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CN116271104(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Cincona Intellectual Property Holding Co ltd filed Critical Cincona Intellectual Property Holding Co ltd
Publication of CN116271104A publication Critical patent/CN116271104A/zh
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    • 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
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    • 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
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    • A61K38/1725—Complement proteins, e.g. anaphylatoxin, C3a or C5a
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    • A61K48/0008—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 '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
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    • 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
    • A61K48/0066—Manipulation 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
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
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    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Microbiology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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CN202310134734.8A 2015-10-28 2016-10-27 基因疗法 Pending CN116271104A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
GBGB1519086.1A GB201519086D0 (en) 2015-10-28 2015-10-28 Gene Therapy
GB1519086.1 2015-10-28
PCT/GB2016/053343 WO2017072515A1 (en) 2015-10-28 2016-10-27 Gene therapy
CN201680073421.4A CN108431030B (zh) 2015-10-28 2016-10-27 基因疗法

Related Parent Applications (1)

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CN201680073421.4A Division CN108431030B (zh) 2015-10-28 2016-10-27 基因疗法

Publications (1)

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CN116271104A true CN116271104A (zh) 2023-06-23

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CN202310134734.8A Pending CN116271104A (zh) 2015-10-28 2016-10-27 基因疗法
CN202310132380.3A Pending CN116370655A (zh) 2015-10-28 2016-10-27 基因疗法
CN201680073421.4A Active CN108431030B (zh) 2015-10-28 2016-10-27 基因疗法
CN202311083674.8A Pending CN117343960A (zh) 2015-10-28 2016-10-27 基因疗法

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CN202310132380.3A Pending CN116370655A (zh) 2015-10-28 2016-10-27 基因疗法
CN201680073421.4A Active CN108431030B (zh) 2015-10-28 2016-10-27 基因疗法
CN202311083674.8A Pending CN117343960A (zh) 2015-10-28 2016-10-27 基因疗法

Country Status (19)

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US (3) US20190255193A1 (https=)
EP (3) EP3360890B1 (https=)
JP (3) JP7126944B2 (https=)
KR (1) KR102922606B1 (https=)
CN (4) CN116271104A (https=)
AU (3) AU2016344523B2 (https=)
CA (1) CA3002125A1 (https=)
DK (2) DK3360890T3 (https=)
ES (2) ES2666204T5 (https=)
GB (1) GB201519086D0 (https=)
HK (1) HK1248723B (https=)
IL (3) IL304084B2 (https=)
MX (1) MX366343B (https=)
NO (1) NO3262066T3 (https=)
PL (2) PL3262066T5 (https=)
RU (1) RU2740038C2 (https=)
SG (1) SG11201802934WA (https=)
WO (1) WO2017072515A1 (https=)
ZA (1) ZA201802462B (https=)

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GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
SG11201808426XA (en) 2016-04-15 2018-10-30 Univ Pennsylvania Compositions for treatment of wet age-related macular degeneration
GB201608046D0 (en) * 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
SG11202000840YA (en) * 2017-07-31 2020-02-27 Reflection Biotechnologies Ltd Cellular models of and therapies for ocular diseases
EP3697920A4 (en) * 2017-10-20 2022-03-02 Gemini Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF AGE-RELATED MACULAR DEGENERATION
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
WO2020077091A1 (en) * 2018-10-10 2020-04-16 Wisconsin Alumni Research Foundation Kir 7.1 gene therapy vectors and methods of using the same
CA3117551A1 (en) * 2018-10-23 2020-04-30 Gemini Therapeutics Inc. Compositions and methods for treating age-related macular degeneration and other diseases
GB201821082D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Combination of complement factors i and h, and vector encoding thereof
GB201821089D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
WO2020206098A1 (en) * 2019-04-03 2020-10-08 Regenxbio Inc. Gene therapy for eye pathologies
US20220313722A1 (en) * 2019-09-09 2022-10-06 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of restoring lysosomal function of retinal pigment epithelial cells by activation of tfeb
CA3182800A1 (en) 2020-06-14 2021-12-23 Vertex Pharmaceuticals Inc. Complement factor i-related compositions and methods
GB202009741D0 (en) 2020-06-25 2020-08-12 Freeline Therapeutics Ltd Polynucleotide
US20220106611A1 (en) * 2020-09-09 2022-04-07 Homology Medicines, Inc. Vectorized antibodies and uses thereof
CN117083065A (zh) 2020-10-30 2023-11-17 庆应义塾 肌肉减少症相关疾病等的新颖治疗和预防
GB202018320D0 (en) * 2020-11-20 2021-01-06 Univ Newcastle Methods of producing recombinant complement proteins
WO2023079301A1 (en) 2021-11-04 2023-05-11 Freeline Therapeutics Limited Assays using mutant complement factor i variants with decreased activity
JPWO2023145735A1 (https=) 2022-01-25 2023-08-03

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RU2740038C2 (ru) 2020-12-30
SG11201802934WA (en) 2018-05-30
EP3360890B1 (en) 2020-10-07
CA3002125A1 (en) 2017-05-04
IL258806B (en) 2021-07-29
DK3360890T3 (da) 2020-11-02
MX366343B (es) 2019-07-05
WO2017072515A1 (en) 2017-05-04
IL258806A (en) 2018-06-28
IL304084B1 (en) 2025-11-01
CN108431030B (zh) 2023-10-20
AU2022203675B2 (en) 2025-03-06
JP2022141670A (ja) 2022-09-29
BR112018008731A8 (pt) 2020-03-31
JP2018537122A (ja) 2018-12-20
IL284577B1 (en) 2023-08-01
AU2016344523B2 (en) 2022-03-17
EP3360890A1 (en) 2018-08-15
CN117343960A (zh) 2024-01-05
GB201519086D0 (en) 2015-12-09
EP3786176A1 (en) 2021-03-03
JP2024156706A (ja) 2024-11-06
PL3360890T3 (pl) 2020-12-28
ES2828050T3 (es) 2021-05-25
AU2016344523A1 (en) 2018-06-14
DK3262066T4 (da) 2023-08-21
KR20180066247A (ko) 2018-06-18
CN108431030A (zh) 2018-08-21
US20250319210A1 (en) 2025-10-16
MX2018005145A (es) 2018-08-09
ES2666204T5 (es) 2023-11-03
EP3262066A1 (en) 2018-01-03
US20190255193A1 (en) 2019-08-22
DK3262066T3 (en) 2018-05-22
IL304084B2 (en) 2026-03-01
US20230277689A1 (en) 2023-09-07
PL3262066T3 (pl) 2018-07-31
KR102922606B1 (ko) 2026-02-03
EP3262066B2 (en) 2023-06-07
IL304084A (en) 2023-08-01
AU2025200979A1 (en) 2025-03-06
RU2018118954A3 (https=) 2020-03-13
ES2666204T3 (es) 2018-05-03
HK1248723B (en) 2019-11-22
ZA201802462B (en) 2021-01-27
IL284577B2 (en) 2023-12-01
CN116370655A (zh) 2023-07-04
BR112018008731A2 (en) 2018-11-06
RU2018118954A (ru) 2019-12-02
IL284577A (en) 2021-08-31
JP7126944B2 (ja) 2022-08-29
EP3262066B1 (en) 2018-02-07
PL3262066T5 (pl) 2023-10-30
AU2022203675A1 (en) 2022-06-16

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