SG11201804166YA - Materials and methods for treatment of titin-based myopathies and other titinopaties - Google Patents

Materials and methods for treatment of titin-based myopathies and other titinopaties

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Publication number
SG11201804166YA
SG11201804166YA SG11201804166YA SG11201804166YA SG11201804166YA SG 11201804166Y A SG11201804166Y A SG 11201804166YA SG 11201804166Y A SG11201804166Y A SG 11201804166YA SG 11201804166Y A SG11201804166Y A SG 11201804166YA SG 11201804166Y A SG11201804166Y A SG 11201804166YA
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SG
Singapore
Prior art keywords
international
titin
domain
methods
band
Prior art date
Application number
SG11201804166YA
Inventor
Louise Rodino-Kalapac
Rachael Potter
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Res Inst Nationwide Childrens Hospital
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Application filed by Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of SG11201804166YA publication Critical patent/SG11201804166YA/en

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    • 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
    • A61K38/39—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
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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
    • C12N15/11—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
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • 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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
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Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau 111111111M 11111111111111111111111111111111111111111111111111111111111111R111111111111111 (10) International Publication Number WO 2017/087395 Al WIPO I PCT (43) International Publication Date 26 May 2017 (26.05.2017) 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, JP, KE, KG, 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. (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). Published: with international search report (Art 21(3)) with sequence listing part of description (Rule 5.2(a)) (51) International Patent Classification: A61K 9/00 (2006.01) A61K 48/00 (2006.01) A61K 38/46 (2006.01) (21) International Application Number: PCT/US2016/062052 (22) International Filing Date: 15 November 2016 (15.11.2016) Filing Language: English (25) (26) (30) English Publication Language: Priority Data: 62/255,887 16 November 2015 (16.11.2015) US (71) Applicant: RESEARCH INSTITUTE AT NATION- WIDE CHILDREN'S HOSPITAL [US/US]; 700 Chil- dren's Drive, Room W172, Cumbus, OH 43205 (US). (72) Inventors: RODINO-KALAPAC, Louise; 4912 Bixby Ridge Drive, E. Groveport, OH 43125 (US). POTTER, Rachael; 7467 Achill Dr., Dublin, OH 43017 (US). (74) Agent: NOLAND, Greta, E.; Marshall, Gerstein & Dorun LLP, 233 S.wacker Drive, 6300 Willis Tower, Chicago, IL 60606-6357 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (54) Title: MATERIALS AND METHODS FOR TREATMENT OF TITIN-BASED MYOPATHIES AND OTHER TITINO - PATIES I-band M-line A-band se - Nebulin 5,55.5, 555 2.S.S... 5 . 5 £ < 1.1 ye/ a -Actinin Myosin TacLTITV., ;, , a,Nrawo:u Actin Thin Filament \"Titan domain: tee N2 -B AM. PEVK eras* N2-A o kinase domain (TK) 5 FIGURE 1A I-band region A-band region M-line region a y co ro EnIENIMEECOOH Myosin MyBPC Calpan-1 -0-crystalin oactinin HSP27 Nebulin „I m P D P I r sirl -FHL1 Filamin c uuscunn -DRAUFHL2 sAnk 1 PKG T-cap/Tolothonin -PKA Z-disk region 1 6.1 illnailnlkillSignigliinigniniainnilinn 013-crYstalin S100A1 HSP27 Actin Smyd2-mHSP90 Nebulin i C g ain3/p94 -Calpain-1 -Tropomyosin MARPs: CARP nig-like domain /Z-repeat 1PEVK element _Protein binding site DARR IFN3 domain X12-bus finterdoman section Kinase domain I. Ankrd-2/arpp W O 20 17 /087 395 Al i m - yi tcunn -L Lamm CalpainMp94 M B i r itiein -Mvmesin ca gt ir rL2 Nbr1Ip62 MUR I FT F-I FIGURE 113 (57) : The present application provides materials and methods for treating a patient with a titin-based myopathy, particu- larly a titin-based cardiomyopathy, and/or other titinopathy. In addition, the present application provides materials and methods for editing the titin gene in a cell by genome editing.
SG11201804166YA 2015-11-16 2016-11-15 Materials and methods for treatment of titin-based myopathies and other titinopaties SG11201804166YA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562255887P 2015-11-16 2015-11-16
PCT/US2016/062052 WO2017087395A1 (en) 2015-11-16 2016-11-15 Materials and methods for treatment of titin-based myopathies and other titinopaties

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Publication Number Publication Date
SG11201804166YA true SG11201804166YA (en) 2018-06-28

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US (2) US11617783B2 (en)
EP (1) EP3377042A4 (en)
JP (3) JP7418957B2 (en)
KR (1) KR20180081600A (en)
CN (1) CN108463211B (en)
AU (1) AU2016358172B2 (en)
BR (1) BR112018009954A2 (en)
CA (1) CA3005633C (en)
CO (1) CO2018006133A2 (en)
EA (1) EA201891192A1 (en)
HK (1) HK1257955A1 (en)
IL (1) IL259441B2 (en)
MX (1) MX2018006116A (en)
SG (1) SG11201804166YA (en)
WO (1) WO2017087395A1 (en)

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JP7418957B2 (en) 2024-01-22
US11617783B2 (en) 2023-04-04
JP2022009734A (en) 2022-01-14
US20230233651A1 (en) 2023-07-27
BR112018009954A2 (en) 2018-11-13
EP3377042A4 (en) 2019-05-29
EP3377042A1 (en) 2018-09-26
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