GB201519086D0 - Gene Therapy - Google Patents

Gene Therapy

Info

Publication number
GB201519086D0
GB201519086D0 GBGB1519086.1A GB201519086A GB201519086D0 GB 201519086 D0 GB201519086 D0 GB 201519086D0 GB 201519086 A GB201519086 A GB 201519086A GB 201519086 D0 GB201519086 D0 GB 201519086D0
Authority
GB
United Kingdom
Prior art keywords
gene therapy
therapy
gene
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.)
Ceased
Application number
GBGB1519086.1A
Other languages
English (en)
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.)
Syncona Partners LLP
Original Assignee
Syncona Partners LLP
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=55130363&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=GB201519086(D0) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Syncona Partners LLP filed Critical Syncona Partners LLP
Priority to GBGB1519086.1A priority Critical patent/GB201519086D0/en
Publication of GB201519086D0 publication Critical patent/GB201519086D0/en
Priority to KR1020187015024A priority patent/KR20180066247A/ko
Priority to CN202310132380.3A priority patent/CN116370655A/zh
Priority to JP2018541571A priority patent/JP7126944B2/ja
Priority to AU2016344523A priority patent/AU2016344523B2/en
Priority to EP16788764.5A priority patent/EP3262066B2/en
Priority to DK16788764.5T priority patent/DK3262066T4/da
Priority to NO16788764A priority patent/NO3262066T3/no
Priority to US15/771,849 priority patent/US20190255193A1/en
Priority to DK17210269.1T priority patent/DK3360890T3/da
Priority to EP20187535.8A priority patent/EP3786176A1/en
Priority to IL284577A priority patent/IL284577B2/en
Priority to ES17210269T priority patent/ES2828050T3/es
Priority to ES16788764T priority patent/ES2666204T5/es
Priority to CA3002125A priority patent/CA3002125A1/en
Priority to CN202310134734.8A priority patent/CN116271104A/zh
Priority to PL17210269T priority patent/PL3360890T3/pl
Priority to CN201680073421.4A priority patent/CN108431030B/zh
Priority to PCT/GB2016/053343 priority patent/WO2017072515A1/en
Priority to MX2018005145A priority patent/MX366343B/es
Priority to RU2018118954A priority patent/RU2740038C2/ru
Priority to PL16788764.5T priority patent/PL3262066T5/pl
Priority to CN202311083674.8A priority patent/CN117343960A/zh
Priority to BR112018008731-9A priority patent/BR112018008731B1/pt
Priority to HK18108313.3A priority patent/HK1248723B/en
Priority to SG11201802934WA priority patent/SG11201802934WA/en
Priority to EP17210269.1A priority patent/EP3360890B1/en
Priority to ZA2018/02462A priority patent/ZA201802462B/en
Priority to IL258806A priority patent/IL258806B/en
Priority to AU2022203675A priority patent/AU2022203675B2/en
Priority to JP2022103103A priority patent/JP2022141670A/ja
Priority to US18/067,283 priority patent/US20230277689A1/en
Priority to IL304084A priority patent/IL304084B1/en
Priority to JP2024112812A priority patent/JP2024156706A/ja
Priority to AU2025200979A priority patent/AU2025200979A1/en
Priority to US19/093,102 priority patent/US20250319210A1/en
Ceased 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • 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
    • 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
    • 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
    • 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
    • A61K38/1725Complement proteins, e.g. anaphylatoxin, C3a or C5a
    • 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
    • A61K48/0008Medicinal 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
    • 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
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525Virus
    • A61K2039/5258Virus-like particles
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/60Medicinal preparations containing antigens or antibodies characteristics by the carrier linked to the antigen
    • A61K2039/6031Proteins
    • A61K2039/6075Viral proteins
    • 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
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Emergency Medicine (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
GBGB1519086.1A 2015-10-28 2015-10-28 Gene Therapy Ceased GB201519086D0 (en)

Priority Applications (36)

Application Number Priority Date Filing Date Title
GBGB1519086.1A GB201519086D0 (en) 2015-10-28 2015-10-28 Gene Therapy
MX2018005145A MX366343B (es) 2015-10-28 2016-10-27 Terapia genetica.
SG11201802934WA SG11201802934WA (en) 2015-10-28 2016-10-27 Gene therapy
PCT/GB2016/053343 WO2017072515A1 (en) 2015-10-28 2016-10-27 Gene therapy
CN202310132380.3A CN116370655A (zh) 2015-10-28 2016-10-27 基因疗法
JP2018541571A JP7126944B2 (ja) 2015-10-28 2016-10-27 遺伝子治療
AU2016344523A AU2016344523B2 (en) 2015-10-28 2016-10-27 Gene therapy
EP16788764.5A EP3262066B2 (en) 2015-10-28 2016-10-27 Gene therapy
DK16788764.5T DK3262066T4 (da) 2015-10-28 2016-10-27 Genterapi
NO16788764A NO3262066T3 (enExample) 2015-10-28 2016-10-27
US15/771,849 US20190255193A1 (en) 2015-10-28 2016-10-27 Gene Therapy
DK17210269.1T DK3360890T3 (da) 2015-10-28 2016-10-27 Genterapi
EP20187535.8A EP3786176A1 (en) 2015-10-28 2016-10-27 Gene therapy
IL284577A IL284577B2 (en) 2015-10-28 2016-10-27 Gene therapy
ES17210269T ES2828050T3 (es) 2015-10-28 2016-10-27 Terapia génica
ES16788764T ES2666204T5 (es) 2015-10-28 2016-10-27 Terapia génica
CA3002125A CA3002125A1 (en) 2015-10-28 2016-10-27 Gene therapy
CN202310134734.8A CN116271104A (zh) 2015-10-28 2016-10-27 基因疗法
PL17210269T PL3360890T3 (pl) 2015-10-28 2016-10-27 Terapia genowa
KR1020187015024A KR20180066247A (ko) 2015-10-28 2016-10-27 유전자 요법
CN201680073421.4A CN108431030B (zh) 2015-10-28 2016-10-27 基因疗法
EP17210269.1A EP3360890B1 (en) 2015-10-28 2016-10-27 Gene therapy
RU2018118954A RU2740038C2 (ru) 2015-10-28 2016-10-27 Генная терапия
PL16788764.5T PL3262066T5 (pl) 2015-10-28 2016-10-27 Terapia genowa
CN202311083674.8A CN117343960A (zh) 2015-10-28 2016-10-27 基因疗法
BR112018008731-9A BR112018008731B1 (pt) 2015-10-28 2016-10-27 Terapia gênica
HK18108313.3A HK1248723B (en) 2015-10-28 2016-10-27 Gene therapy
ZA2018/02462A ZA201802462B (en) 2015-10-28 2018-04-13 Gene therapy
IL258806A IL258806B (en) 2015-10-28 2018-04-18 Gene therapy
AU2022203675A AU2022203675B2 (en) 2015-10-28 2022-05-30 Gene therapy
JP2022103103A JP2022141670A (ja) 2015-10-28 2022-06-28 遺伝子治療
US18/067,283 US20230277689A1 (en) 2015-10-28 2022-12-16 Gene therapy
IL304084A IL304084B1 (en) 2015-10-28 2023-06-27 Gene healing
JP2024112812A JP2024156706A (ja) 2015-10-28 2024-07-12 遺伝子治療
AU2025200979A AU2025200979A1 (en) 2015-10-28 2025-02-13 Gene therapy
US19/093,102 US20250319210A1 (en) 2015-10-28 2025-03-27 Gene therapy

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB1519086.1A GB201519086D0 (en) 2015-10-28 2015-10-28 Gene Therapy

Publications (1)

Publication Number Publication Date
GB201519086D0 true GB201519086D0 (en) 2015-12-09

Family

ID=55130363

Family Applications (1)

Application Number Title Priority Date Filing Date
GBGB1519086.1A Ceased GB201519086D0 (en) 2015-10-28 2015-10-28 Gene Therapy

Country Status (19)

Country Link
US (3) US20190255193A1 (enExample)
EP (3) EP3262066B2 (enExample)
JP (3) JP7126944B2 (enExample)
KR (1) KR20180066247A (enExample)
CN (4) CN116271104A (enExample)
AU (3) AU2016344523B2 (enExample)
CA (1) CA3002125A1 (enExample)
DK (2) DK3262066T4 (enExample)
ES (2) ES2666204T5 (enExample)
GB (1) GB201519086D0 (enExample)
HK (1) HK1248723B (enExample)
IL (3) IL284577B2 (enExample)
MX (1) MX366343B (enExample)
NO (1) NO3262066T3 (enExample)
PL (2) PL3262066T5 (enExample)
RU (1) RU2740038C2 (enExample)
SG (1) SG11201802934WA (enExample)
WO (1) WO2017072515A1 (enExample)
ZA (1) ZA201802462B (enExample)

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AU2016326627B2 (en) * 2015-09-24 2022-12-15 The Trustees Of The University Of Pennsylvania Composition and method for treating complement-mediated disease
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
WO2017180936A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of 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
EP4445954A3 (en) * 2017-07-31 2025-01-08 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
AU2018351491A1 (en) * 2017-10-20 2020-05-07 Gemini Therapeutics Inc. Compositions and methods for treating 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
WO2020086735A1 (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
EP4667578A2 (en) * 2019-04-03 2025-12-24 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
WO2022056531A1 (en) * 2020-09-09 2022-03-17 Homology Medicines, Inc. Vectorized antibodies and uses thereof
US20230390296A1 (en) 2020-10-30 2023-12-07 Keio University Novel treatment and prevention of sarcopenia-related diseases
GB202018320D0 (en) * 2020-11-20 2021-01-06 Univ Newcastle Methods of producing recombinant complement proteins
TW202323270A (zh) 2021-11-04 2023-06-16 英商自由行療法有限公司 多肽
US20250161255A1 (en) 2022-01-25 2025-05-22 Motoshi Hayano New therapy and prevention for suppressing aging-related disorders including sarcopenia

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