SG11201907606XA - Humanized model of kidney and liver disorders - Google Patents

Humanized model of kidney and liver disorders

Info

Publication number
SG11201907606XA
SG11201907606XA SG11201907606XA SG11201907606XA SG11201907606XA SG 11201907606X A SG11201907606X A SG 11201907606XA SG 11201907606X A SG11201907606X A SG 11201907606XA SG 11201907606X A SG11201907606X A SG 11201907606XA SG 11201907606X A SG11201907606X A SG 11201907606XA
Authority
SG
Singapore
Prior art keywords
tarrytown
international
saw mill
river road
regeneron pharmaceuticals
Prior art date
Application number
SG11201907606XA
Inventor
Kishor Devalaraja-Narashimha
Lori Morton
Yifan Luo
Cong Huang
Karolina Meagher
Sarah Casanova
Original Assignee
Regeneron Pharma
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 Regeneron Pharma filed Critical Regeneron Pharma
Publication of SG11201907606XA publication Critical patent/SG11201907606XA/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • C07K14/472—Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5082—Supracellular entities, e.g. tissue, organisms
    • G01N33/5088—Supracellular entities, e.g. tissue, organisms of vertebrates
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • 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
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/054—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
    • 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
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • 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
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • 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
    • 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
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0368—Animal model for inflammation
    • 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
    • A01K2267/0393—Animal model comprising a reporter system for screening tests
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8527—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8527—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
    • C12N2015/8536—Animal models for genetic diseases
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Urology & Nephrology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Environmental Sciences (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Pathology (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • General Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Animal Husbandry (AREA)
  • Food Science & Technology (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 30 August 2018 (30.08.2018) WIPO I PCT onion °nolo HE loonliolommo oimIE (10) International Publication Number WO 2018/157027 Al (51) International Patent Classification: A61K 49/00 (2006.01) CO7K 14/47 (2006.01) A01K 67/027 (2006.01) G01N 33/50 (2006.01) (21) International Application Number: PCT/US2018/019651 (22) International Filing Date: 26 February 2018 (26.02.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/464,262 27 February 2017 (27.02.2017) US 62/529,916 07 July 2017 (07.07.2017) US 62/583,780 09 November 2017 (09.11.2017) US (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii)) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: with international search report (Art. 21(3)) with sequence listing part of description (Rule 5.2(a)) 1-1 N N O N kr) 00 O N C (71) Applicant: REGENERON PHARMACEUTICALS, INC. [US/US]; 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). (72) Inventors: DEVALARAJA-NARASHIMHA, Kishor; c/ o Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). MOR- TON, Lori; c/o Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). LUO, Yifan; c/o Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). HUANG, Cong; c/o Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). MEAGHER, Karolina; c/o Regeneron Pharmaceu- ticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). CASANOVA, Sarah; c/o Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tar- rytown, NY 10591-6707 (US). (74) Agent: GROLZ, Edward, W.; Scully, Scott, Murphy & Presser, 400 Garden City Plaza, Suite 300, Garden City, NY 11530 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (54) Title: HUMANIZED MODEL OF KIDNEY AND LIVER DISORDERS (57) : Provided herein, inter alia, are non-human animals comprising nucleic acid sequences encoding a C3 protein that com- prises a human sequence as well as transgenic non-human animals comprising a C3 gene that is human in whole or in part as well as methods for using the same to screen for candidate therapeutic molecules to treat complement-related nephropathies. Also provided herein are methods for improving kidney function in an individual diagnosed with or thought to have a complement-related nephropathy.
SG11201907606XA 2017-02-27 2018-02-26 Humanized model of kidney and liver disorders SG11201907606XA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762464262P 2017-02-27 2017-02-27
US201762529916P 2017-07-07 2017-07-07
US201762583780P 2017-11-09 2017-11-09
PCT/US2018/019651 WO2018157027A1 (en) 2017-02-27 2018-02-26 Humanized model of kidney and liver disorders

Publications (1)

Publication Number Publication Date
SG11201907606XA true SG11201907606XA (en) 2019-09-27

Family

ID=61617150

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11201907606XA SG11201907606XA (en) 2017-02-27 2018-02-26 Humanized model of kidney and liver disorders
SG10202111663PA SG10202111663PA (en) 2017-02-27 2018-02-26 Humanized model of kidney and liver disorders

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG10202111663PA SG10202111663PA (en) 2017-02-27 2018-02-26 Humanized model of kidney and liver disorders

Country Status (11)

Country Link
US (1) US10765762B2 (en)
EP (1) EP3585445B1 (en)
JP (1) JP7169979B2 (en)
KR (1) KR102602199B1 (en)
CN (1) CN110494168B (en)
AU (1) AU2018225745B2 (en)
CA (1) CA3054167A1 (en)
IL (1) IL268748A (en)
MX (1) MX2019010219A (en)
SG (2) SG11201907606XA (en)
WO (1) WO2018157027A1 (en)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7139419B2 (en) 2017-09-29 2022-09-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Non-Human Animals Containing Humanized TTR Loci and Methods of Use
WO2020018511A1 (en) 2018-07-16 2020-01-23 Regeneron Pharmaceuticals, Inc. Non-human animal models of ditra disease and uses thereof
CA3112612C (en) * 2018-09-13 2024-02-27 Regeneron Pharmaceuticals, Inc. Complement factor h gene knockout rat as a model of c3 glomerulopathy
ES2966625T3 (en) 2019-04-04 2024-04-23 Regeneron Pharma Rodents comprising a humanized coagulation factor 12 locus
CA3137761A1 (en) 2019-06-04 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use
EP3796776A1 (en) 2019-06-07 2021-03-31 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
WO2021112235A1 (en) * 2019-12-05 2021-06-10 国立大学法人宮崎大学 Medicine and medicinal composition for treating or preventing c3 nephropathy, and complement c3b degradation accelerator
WO2022072671A1 (en) * 2020-10-01 2022-04-07 Regeneron Pharmaceuticals, Inc. Rodent animals expressing human cr1

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4708871A (en) 1983-03-08 1987-11-24 Commonwealth Serum Laboratories Commission Antigenically active amino acid sequences
NZ207394A (en) 1983-03-08 1987-03-06 Commw Serum Lab Commission Detecting or determining sequence of amino acids
WO1984003506A1 (en) 1983-03-08 1984-09-13 Commw Serum Lab Commission Antigenically active amino acid sequences
US6492107B1 (en) 1986-11-20 2002-12-10 Stuart Kauffman Process for obtaining DNA, RNA, peptides, polypeptides, or protein, by recombinant DNA technique
JP2584613B2 (en) 1985-03-30 1997-02-26 バリベ、マール Method for obtaining DNA, RNA, peptide, polypeptide or protein by recombinant DNA technology
NZ215865A (en) 1985-04-22 1988-10-28 Commw Serum Lab Commission Method of determining the active site of a receptor-binding analogue
DE3883899T3 (en) 1987-03-18 1999-04-22 Sb2 Inc CHANGED ANTIBODIES.
US5266684A (en) 1988-05-02 1993-11-30 The Reagents Of The University Of California Peptide mixtures
US5571689A (en) 1988-06-16 1996-11-05 Washington University Method of N-acylating peptide and proteins with diheteroatom substituted analogs of myristic acid
US5223409A (en) 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
US5663143A (en) 1988-09-02 1997-09-02 Dyax Corp. Engineered human-derived kunitz domains that inhibit human neutrophil elastase
US5427908A (en) 1990-05-01 1995-06-27 Affymax Technologies N.V. Recombinant library screening methods
US5723286A (en) 1990-06-20 1998-03-03 Affymax Technologies N.V. Peptide library and screening systems
US5698426A (en) 1990-09-28 1997-12-16 Ixsys, Incorporated Surface expression libraries of heteromeric receptors
US5770434A (en) 1990-09-28 1998-06-23 Ixsys Incorporated Soluble peptides having constrained, secondary conformation in solution and method of making same
ATE176239T1 (en) 1990-11-21 1999-02-15 Iterex Pharma Lp SYNTHESIS OF EQUIMOLAR MIXTURES OF VARIOUS OLIGOMERS, SPECIFICALLY OLIGOPEPTIDE MIXTURES
ATE164395T1 (en) 1990-12-03 1998-04-15 Genentech Inc METHOD FOR ENRICHMENT OF PROTEIN VARIANTS WITH MODIFIED BINDING PROPERTIES
US5270170A (en) 1991-10-16 1993-12-14 Affymax Technologies N.V. Peptide library and screening method
US5644048A (en) 1992-01-10 1997-07-01 Isis Pharmaceuticals, Inc. Process for preparing phosphorothioate oligonucleotides
CA2163345A1 (en) 1993-06-16 1994-12-22 Susan Adrienne Morgan Antibodies
GB9521943D0 (en) 1995-10-26 1996-01-03 Univ Hertfordshire Coded particles for process sequence tracking in combinatorial compound library preparation
ATE375365T1 (en) 1998-04-02 2007-10-15 Genentech Inc ANTIBODIES VARIANTS AND FRAGMENTS THEREOF
US6335155B1 (en) 1998-06-26 2002-01-01 Sunesis Pharmaceuticals, Inc. Methods for rapidly identifying small organic molecule ligands for binding to biological target molecules
US6280946B2 (en) 1998-08-07 2001-08-28 Boston Probes, Inc. PNA probes, probe sets, methods and kits pertaining to the universal detection of bacteria and eucarya
CA2355215A1 (en) 1998-12-28 2000-07-06 Jim Wells Identifying small organic molecule ligands for binding
JP2009523237A (en) 2006-01-11 2009-06-18 ノヴォ ノルディスク アクティーゼルスカブ Improved spherical coded beads
MX2009013082A (en) * 2007-06-07 2010-01-15 Genentech Inc C3b antibodies and methods for the prevention and treatment of complement- associated disorders.
WO2010054403A1 (en) 2008-11-10 2010-05-14 Alexion Pharmaceuticals, Inc. Methods and compositions for treating complement-associated disorders
WO2013152024A1 (en) * 2012-04-03 2013-10-10 Novelmed Therapeutics, Inc Humanized and chimeric anti-factor c3 antibodies and uses thereof
ES2647124T3 (en) * 2012-06-28 2017-12-19 Novartis Ag Pyrrolidine derivatives and their use as modulators of the complement pathway
KR102192596B1 (en) * 2013-08-07 2020-12-18 알렉시온 파마슈티칼스, 인코포레이티드 Atypical hemolytic uremic syndrome (ahus) biomarker proteins
US9659904B2 (en) * 2013-12-12 2017-05-23 Intel Corporation Distributed on-package millimeter-wave radio
KR20160116018A (en) * 2014-02-25 2016-10-06 아칠리온 파르마세우티칼스 인코포레이티드 Amide compounds for treatment of complement mediated disorders
WO2015171523A1 (en) 2014-05-05 2015-11-12 Regeneron Pharmaceuticals, Inc. Humanized c5 and c3 animals

Also Published As

Publication number Publication date
CN110494168A (en) 2019-11-22
RU2019126691A (en) 2021-03-29
US20180243450A1 (en) 2018-08-30
CN110494168B (en) 2023-02-03
KR20190122231A (en) 2019-10-29
RU2019126691A3 (en) 2021-10-04
JP2020510827A (en) 2020-04-09
AU2018225745A1 (en) 2019-09-19
EP3585445A1 (en) 2020-01-01
MX2019010219A (en) 2020-02-07
CA3054167A1 (en) 2018-08-30
SG10202111663PA (en) 2021-12-30
JP7169979B2 (en) 2022-11-11
EP3585445B1 (en) 2024-02-07
KR102602199B1 (en) 2023-11-15
AU2018225745B2 (en) 2023-12-07
IL268748A (en) 2019-10-31
WO2018157027A1 (en) 2018-08-30
EP3585445C0 (en) 2024-02-07
US10765762B2 (en) 2020-09-08

Similar Documents

Publication Publication Date Title
SG11201907606XA (en) Humanized model of kidney and liver disorders
SG11201903882VA (en) Il-2 variants for the treatment of autoimmune diseases
SG11201907299XA (en) Multi-specific binding proteins for activation of natural killer cells and therapeutic uses thereof to treat cancer
SG11202000274RA (en) Oligonucleotide compositions and methods thereof
SG11201906200WA (en) Nucleic acid-polypeptide compositions and methods of inducing exon skipping
SG11201903167QA (en) Compounds and methods for reducing atxn3 expression
SG11201808196UA (en) Neoantigens and methods of their use
SG11201808619QA (en) Nucleic acid-polypeptide compositions and uses thereof
SG11201805784PA (en) Molecules that selectively activate regulatory t cells for the treatment of autoimmune diseases
SG11201906161VA (en) Microorganisms programmed to produce immune modulators and anti-cancer therapeutics in tumor cells
SG11201908489XA (en) De novo synthesized combinatorial nucleic acid libraries
SG11201906319XA (en) Compositions and methods for the depletion of cd137+ cells
SG11201804713VA (en) TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
SG11201810757YA (en) Non-human animals expressing exogenous terminal deoxynucleotidyltransferase
SG11201407370QA (en) Humanized il-7 rodents
SG11201807279QA (en) Binding proteins and methods of use thereof
SG11201804070XA (en) Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
SG11201907490XA (en) Non-human animal models of retinoschisis
SG11201810927QA (en) Pd-l1-specific antibodies and methods of using the same
SG11201908238SA (en) Anti-c5 antibodies and uses thereof
SG11201806496SA (en) Antigen binding proteins that bind pd-l1
SG11201809374VA (en) Cd40l-fc fusion polypeptides and methods of use thereof
SG11201806453TA (en) Improved blood-brain barrier endothelial cells derived from pluripotent stem cells for blood-brain barrier models
SG11201807881VA (en) Compositions and methods for making antibodies based on use of an expression-enhancing locus
SG11201407486PA (en) Compositions and methods for modulating utrn expression