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SG11201900085TA - Modified ligand-gated ion channels and methods of use - Google Patents

Modified ligand-gated ion channels and methods of use

Info

Publication number
SG11201900085TA
SG11201900085TA SG11201900085TA SG11201900085TA SG11201900085TA SG 11201900085T A SG11201900085T A SG 11201900085TA SG 11201900085T A SG11201900085T A SG 11201900085TA SG 11201900085T A SG11201900085T A SG 11201900085TA SG 11201900085T A SG11201900085T A SG 11201900085TA
Authority
SG
Singapore
Prior art keywords
methods
international
modified
lgic
maryland
Prior art date
Application number
SG11201900085TA
Inventor
Scott Sternson
Peter Lee
Christopher Magnus
Original Assignee
Hughes Howard Med Inst
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 Hughes Howard Med Inst filed Critical Hughes Howard Med Inst
Publication of SG11201900085TA publication Critical patent/SG11201900085TA/en

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    • 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
    • C07K14/4713—Autoimmune diseases, e.g. Insulin-dependent diabetes mellitus, multiple sclerosis, rheumathoid arthritis, systemic lupus erythematosus; Autoantigens
    • 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
    • C12N15/62—DNA sequences coding for fusion proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/01—Hydrolysed proteins; Derivatives thereof
    • A61K38/012—Hydrolysed proteins; Derivatives thereof from animals
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
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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
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    • 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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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Cell Biology (AREA)
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  • Microbiology (AREA)
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  • Plant Pathology (AREA)
  • Marine Sciences & Fisheries (AREA)
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Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111011111111101 0111011101111101001H011101111111111111111011111111111110111111 Organization International Bureau (10) International Publication Number (43) International Publication Date ......0\" WO 2018/009832 Al 11 January 2018 (11.01.2018) W I PO I PCT (51) International Patent Classification: C07K 14/705 (2006.01) Cl 2N 5/071 (2010.01) Published: G01N 33/58 (2006.01) A61K 48/00 (2006.01) — with international search report (Art. 21(3)) G01N 33/68 (2006.01) A61K 38/00 (2006.01) — with sequence listing part of description (Rule 5.2(a)) (21) International Application Number: PCT/US2017/041147 (22) International Filing Date: 07 July 2017 (07.07.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/359,534 07 July 2016 (07.07.2016) US 62/486,779 18 April 2017 (18.04.2017) US (72) Inventors; and (71) Applicants: STERNSON, Scott [US/US]; 4000 Jones Bridge Road, Chevy Chase, Maryland 20815-6789 (US). LEE, Peter [US/US]; 4000 Jones Bridge Road, Chevy _ Chase, Maryland 20815-6789 (US). MAGNUS, Christo- pher — [US/US]; 4000 Jones Bridge Road, Chevy Chase, Maryland 20815-6789 (US). = Agent: WILLIS, Margaret S. J. et al.; Fish & Richardson (74) = _ P.C., P.O. Box 1022, Minneapolis, Minnesota 55440-1022 = (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. = Designated States (unless otherwise indicated, for every (84) = 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, = — Il KM, ML, MR, NE, SN, TD, TG). . 4 ei M X ) (54) Title: MODIFIED LIGAND-GATED ION CHANNELS AND METHODS OF USE 01 C ::: : (57) : This document relates to materials and methods for controlling ligand gated ion channel (LGIC) activity. For example, 0 —. modified LGICs including at least one LGIC subunit having a modified ligand binding domain (LBD) and/or a modified ion pore GC , 1 domain (IPD) are provided. Also provided are exogenous LGIC ligands that can bind to and activate the modified LGIC, as well as © methods of modulating ion transport across the membrane of a cell of a mammal, methods of modulating the excitability of a cell in N a mammal, and methods of treating a mammal having a channelopathy. 0
SG11201900085TA 2016-07-07 2017-07-07 Modified ligand-gated ion channels and methods of use SG11201900085TA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662359534P 2016-07-07 2016-07-07
US201762486779P 2017-04-18 2017-04-18
PCT/US2017/041147 WO2018009832A1 (en) 2016-07-07 2017-07-07 Modified ligand-gated ion channels and methods of use

Publications (1)

Publication Number Publication Date
SG11201900085TA true SG11201900085TA (en) 2019-02-27

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SG11201900085TA SG11201900085TA (en) 2016-07-07 2017-07-07 Modified ligand-gated ion channels and methods of use

Country Status (14)

Country Link
US (5) US11084853B2 (en)
EP (1) EP3481858A4 (en)
JP (3) JP7755922B2 (en)
KR (2) KR20190044617A (en)
CN (2) CN110023331B (en)
AU (2) AU2017293925C1 (en)
BR (1) BR112019000182A2 (en)
CA (1) CA3030147A1 (en)
IL (3) IL304040B2 (en)
MX (2) MX2019000175A (en)
NZ (1) NZ749744A (en)
SG (1) SG11201900085TA (en)
WO (1) WO2018009832A1 (en)
ZA (2) ZA201900456B (en)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110023331B (en) * 2016-07-07 2024-09-17 霍华休斯医学研究院 Modified ligand-gated ion channels and methods of use
WO2018175443A1 (en) * 2017-03-20 2018-09-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Targeted gene therapies for pain and other neuro-related disorders
WO2019094778A1 (en) * 2017-11-10 2019-05-16 Scott Sternson Modified ligand-gated ion channels and methods of use
KR20200112811A (en) * 2017-11-27 2020-10-05 코다 바이오테라퓨틱스 인코포레이티드 Composition and method for neurodegenerative diseases
KR102489109B1 (en) * 2020-06-02 2023-01-16 부산대학교 산학협력단 Composition for treating dystonia comprising acetylcholine receptor gene variants
WO2022040535A2 (en) * 2020-08-21 2022-02-24 Coda Biotherapeutics, Inc. Compositions and methods for neurological diseases
WO2022255915A1 (en) * 2021-06-01 2022-12-08 Agneta Nordberg Pet radiotracers
US20250145687A1 (en) * 2022-01-31 2025-05-08 Howard Hughes Medical Institute Modified ligand-gated ion channels and methods of use
WO2023159247A1 (en) * 2022-02-18 2023-08-24 Trames Bio, Inc. Ligand gated ion channels and methods of use
CA3250470A1 (en) * 2022-04-27 2023-11-02 Howard Hughes Medical Institute Chemogenetic receptors and methods of making and using
US20240390521A1 (en) * 2023-04-28 2024-11-28 Kriya Therapeutics, Inc. Chimeric modified ion channels and uses thereof for treatment of trigeminal nerve disorders
CN116731008B (en) * 2023-06-15 2025-10-24 西北大学 Wash-free fluorescent probe for labeling lung cancer target nicotinic acetylcholine receptor and preparation method
WO2025015092A1 (en) 2023-07-10 2025-01-16 Kriya Therapeutics, Inc. Novel cam-kinase promoter and uses thereof for treating nervous system disorders
GB202401492D0 (en) * 2024-02-05 2024-03-20 Ucl Business Ltd Chloride channels and uses thereof

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WO2019094778A1 (en) * 2017-11-10 2019-05-16 Scott Sternson Modified ligand-gated ion channels and methods of use

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Publication number Publication date
ZA201900456B (en) 2021-06-30
ZA202101867B (en) 2022-08-31
US20180009862A1 (en) 2018-01-11
JP2023040251A (en) 2023-03-22
AU2022202948B2 (en) 2024-03-07
IL304040A (en) 2023-08-01
US20210403522A1 (en) 2021-12-30
CA3030147A1 (en) 2018-01-11
JP7755922B2 (en) 2025-10-17
US10981962B2 (en) 2021-04-20
IL311984A (en) 2024-06-01
IL304040B1 (en) 2024-05-01
US20220041671A1 (en) 2022-02-10
AU2024203888A1 (en) 2024-06-27
US11124554B2 (en) 2021-09-21
MX2019000175A (en) 2020-01-20
AU2017293925C1 (en) 2025-02-27
WO2018009832A1 (en) 2018-01-11
AU2022202948A1 (en) 2022-06-16
NZ749744A (en) 2025-11-28
IL264098B1 (en) 2023-08-01
IL304040B2 (en) 2024-09-01
CN110023331B (en) 2024-09-17
US20190375807A1 (en) 2019-12-12
KR20190044617A (en) 2019-04-30
AU2017293925B2 (en) 2022-02-03
JP2019530468A (en) 2019-10-24
JP7538902B2 (en) 2024-08-22
MX2022013766A (en) 2022-11-30
US20200377566A1 (en) 2020-12-03
CN110023331A (en) 2019-07-16
AU2017293925A1 (en) 2019-01-24
IL264098B2 (en) 2023-12-01
KR102874080B1 (en) 2025-10-23
IL264098A (en) 2019-01-31
AU2022202948C1 (en) 2024-09-26
EP3481858A1 (en) 2019-05-15
BR112019000182A2 (en) 2019-04-16
EP3481858A4 (en) 2020-06-24
CN119120484A (en) 2024-12-13
KR20230145505A (en) 2023-10-17
US11084853B2 (en) 2021-08-10
JP2025069252A (en) 2025-04-30

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