DK3058071T3 - : dynamin-2-inhibitor til behandlingen af centronukleære myopatier - Google Patents
: dynamin-2-inhibitor til behandlingen af centronukleære myopatier Download PDFInfo
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- DK3058071T3 DK3058071T3 DK14786893.9T DK14786893T DK3058071T3 DK 3058071 T3 DK3058071 T3 DK 3058071T3 DK 14786893 T DK14786893 T DK 14786893T DK 3058071 T3 DK3058071 T3 DK 3058071T3
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- dynamin
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- inhibitor
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Claims (14)
1. Inhibitor af Dynamin 2 til anvendelse i behandlingen af en centronukleær myopati, hvor dynamin 2-inhibitoren er valgt fra gruppen bestående af et antistof rettet mod Dynamin 2, et nukleinsyremolekyle, som interagerer direkte med en nukleinsyre, der koder for Dynamin 2, og et lille molekyle, som inhiberer Dynamin 2-aktiviteten, -ekspressionen eller -funktionen, hvor nævnte lille molekyle er valgt fra gruppen bestående af 3-Hydroxynaphthalen-2-carboxylsyre (3,4-dihydroxybenzyliden)hydrazid, 3-Hydroxy-N'-[(2,4,5-trihydroxyphenyl)methyliden]naphthalen-2-carbohydrazid, Tetradecyltrimethylammoniumbromid, 4-Chlor-2-((2-(3-nitrophenyl)-l,3-dioxo-2,3-dihydro-lH-isoindol-5-carbonyl)-amino)-benzoesyre, 2-Cyano-/V-octyl-3-[l-(3-dimethylaminopropyl)-lH-indol-3-yl]acrylamid, 3-(2,4-Dichlor-5-methoxyphenyl)-2-sulfanylquinazolin-4(3H)-on, /V,/V'-(Propan-l,3-diyl)bis(7,8-dihydroxy-2-imino-2H-chromen-3-carboxamid), /V/ZV-CEthan-l^-diyObisC?^-dihydroxy-2-imino-2H-chromen-3-carboxamid), OctadecylTriMethylAmmoniumBromid, Dynamin-inhiberende peptid med aminosyresekvens: QVPSRPNRAP, 3-Hydroxy-/V'-[(2,4,5- trihydroxyphenyl)methyliden]naphthalen-2-carbohydrazid, og 4-(N,N-Dimethyl-N-octadecyl-N-ethyl)-4-aza-10-oxatricyclo-[5.2.1]decan-3,5-dionbromid.
2. Inhibitoren af Dynamin 2 til anvendelse ifølge krav 1, hvor den centronukleære myopati er valgt fra gruppen bestående af X-forbundet CNM, autosomal recessiv CNM, og autosomal dominant CNM.
3. Inhibitoren af Dynamin 2 til anvendelse ifølge krav 1, hvor den centronukleære myopati er X-forbundet CNM eller autosomal recessiv CNM.
4. Inhibitoren af Dynamin 2 til anvendelse ifølge krav 1, hvor den centronukleære myopati er en centronukleær myopati forårsaget af BINl-mutationer.
5. Inhibitoren af Dynamin 2 til anvendelse ifølge et hvilket som helst af kravene 1, 2, 3 og 4, hvor dynamin 2-inhibitoren er valgt fra gruppen bestående af et antistof rettet mod Dynamin 2, eller et nukleinsyremolekyle, som interagerer direkte med en nukleinsyre, der koder for Dynamin 2.
6. Inhibitoren af Dynamin 2 til anvendelse ifølge et hvilket som helst af kravene 1-5, hvor dynamin 2-inhibitoren er et RNAi, en antisense-nukleinsyre eller et ribozym, der interfererer specifikt med Dynamin 2-ekspression, fortrinsvis hvor dynamin 2-inhibitoren er et siRNA eller shRNA.
7. Inhibitoren af Dynamin 2 til anvendelse ifølge et hvilket som helst af kravene 1-6, hvor dynamin 2-inhibitoren er et antisense-nukleotid, som inducerer exon-skipping i et Dynamin 2 præ-mRNA.
8. Inhibitoren af Dynamin 2 til anvendelse ifølge krav 7, hvor dynamin 2-inhibitoren er et antisense-nukleotid udviklet til specifikt at inducere DNM2 exon 2 eller exon 8 skipping, og fortrinsvis omfatter eller består af en af de følgende sekvenser: U7-Ex2 (target skipping af DNM2 exon 2), omfattende den følgende sekvens: SEQ ID No 26: GTCACCCGGAGGCCTCTCATTCTGCAGCTC U7-Ex8 (target skipping af DNM2 exon 8), omfattende den følgende sekvens: SEQ ID No 27: ACACACTAGAGTTGTCTGGTGGAGCCCGCATCA.
9. Inhibitoren af Dynamin 2 til anvendelse ifølge et hvilket som helst af kravene 1-6, hvor dynamin 2-inhibitoren er et nukleinsyremolekyle, som specifikt interfererer med Dynamin 2 og omfatter eller består af en sekvens valgt fra gruppen bestående af SEQ ID 2-25.
10. Inhibitoren af Dynamin 2 til anvendelse ifølge et hvilket som helst af kravene 1-5, hvor dynamin 2-inhibitoren er et DNA, mRNA eller en nuklease fremstillet målrettet mod DNM2-genet og til at levere nukleaser under anvendelse af genomediteringsterapi.
11. Inhibitoren af Dynamin 2 til anvendelse ifølge et hvilket som helst af kravene 1-10, hvor inhibitoren er administreret i en mængde tilstrækkelig til at reducere Dynamin 2-ekspressionen eller Dynamin 2-aktiviteten, -ekspressionen eller -funktionen til et niveau lig med eller fortrinsvis mindre end det normale niveau.
12. Fremgangsmåde til at identificere eller screene molekyler anvendelige til behandlingen afen centronukleær myopati, fortrinsvis XLCNM, omfattende trinnene: a. at tilvejebringe eller opnå en kandidatforbindelse; og b. at bestemme hvorvidt nævnte kandidatforbindelse inhiberer aktiviteten, funktionen og/eller ekspressionen af Dynamin 2, c. at vælge nævnte kandidatforbindelse, hvis den inhiberer aktiviteten/ekspression/funktionen af Dynamin 2.
13. Fremgangsmåden ifølge krav 12, hvor den yderligere omfatter trinnet at administrere in vitro det valgte molekyle i en centronukleær myopati, ikke-human dyremodel eller en del deraf, og at analysere virkningen på myopati-indtræden eller -udviklingen.
14. Farmaceutisk sammensætning omfattende en inhibitor af Dynamin 2 og en farmaceutisk acceptabel bærer/excipiens til anvendelse til behandlingen afen centronukleær myopati, og mere fortrinsvis hvor inhibitoren af Dynamin 2 er som defineret i et af kravene 1 til 10.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP13306440.2A EP2862928A1 (en) | 2013-10-18 | 2013-10-18 | Dynamin 2 inhibitor for the treatment of centronuclear myopathies |
| PCT/EP2014/072466 WO2015055859A1 (en) | 2013-10-18 | 2014-10-20 | Dynamin 2 inhibitor for the treatment of centronuclear myopathies |
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| DK3058071T3 true DK3058071T3 (da) | 2018-10-22 |
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| DK14786893.9T DK3058071T3 (da) | 2013-10-18 | 2014-10-20 | : dynamin-2-inhibitor til behandlingen af centronukleære myopatier |
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| Country | Link |
|---|---|
| US (2) | US10647986B2 (da) |
| EP (2) | EP2862928A1 (da) |
| JP (2) | JP6641270B2 (da) |
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| AU (1) | AU2014336070B2 (da) |
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| WO (1) | WO2015055859A1 (da) |
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| EP2862928A1 (en) | 2013-10-18 | 2015-04-22 | Université de Strasbourg | Dynamin 2 inhibitor for the treatment of centronuclear myopathies |
| EP3085785A1 (en) * | 2015-04-22 | 2016-10-26 | Université de Strasbourg | Dynamin 2 inhibitor for the treatment of duchenne's muscular dystrophy |
| US10947540B2 (en) * | 2016-11-29 | 2021-03-16 | Association Institut De Myologie | Allele-specific silencing therapy for Dynamin 2-related diseases |
| WO2018115477A1 (en) * | 2016-12-23 | 2018-06-28 | Universite De Strasbourg | Dynamin 2 inhibitor for the treatment of myotonic dystrophy |
| EP3610016A1 (en) | 2017-04-10 | 2020-02-19 | Genethon | Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies |
| EP3706772A1 (en) * | 2017-11-11 | 2020-09-16 | Université de Strasbourg | Compositions and method for the treatment of x-linked centronuclear myopathy |
| AU2019206731A1 (en) | 2018-01-15 | 2020-07-30 | Ionis Pharmaceuticals, Inc. | Modulators of DNM2 expression |
| EP3830130A4 (en) * | 2018-08-02 | 2022-05-18 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating centronuclear myopathy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| EP3941505A1 (en) * | 2019-03-20 | 2022-01-26 | Centre national de la recherche scientifique | Amphiphysin / bin1 for the treatment of autosomal dominant centronuclear myopathy |
| CA3172179A1 (en) | 2020-03-20 | 2021-09-23 | Jamal Tazi | Compounds for treating or preventing a coronaviridae infection & methods and uses for assessing the occurrence of a coronaviridae infection |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| EP4311546A1 (en) | 2022-07-26 | 2024-01-31 | Dynacure | Combination therapy for myopathies |
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| US6506559B1 (en) | 1997-12-23 | 2003-01-14 | Carnegie Institute Of Washington | Genetic inhibition by double-stranded RNA |
| GB9811692D0 (en) * | 1998-06-01 | 1998-07-29 | Medical Res Council | Improvements in or relating to uptake of substances by cells |
| US8491914B2 (en) * | 2004-02-13 | 2013-07-23 | Ibc Pharmaceuticals, Inc. | Dock-and-lock (DNL) complexes for delivery of interference RNA |
| WO2012106313A2 (en) | 2011-01-31 | 2012-08-09 | Oklahoma Medical Research Foundation | Ubiquitin interacting motif peptides as cancer therapeutics |
| CA2840222A1 (en) * | 2011-07-01 | 2013-01-10 | Board Of Regents, The University Of Texas System | Compositions and methods for treating skeletal myopathy |
| WO2013006555A1 (en) | 2011-07-01 | 2013-01-10 | Waltrip Mark A | Bottle holder and related methods |
| EP2862928A1 (en) | 2013-10-18 | 2015-04-22 | Université de Strasbourg | Dynamin 2 inhibitor for the treatment of centronuclear myopathies |
| US10947540B2 (en) * | 2016-11-29 | 2021-03-16 | Association Institut De Myologie | Allele-specific silencing therapy for Dynamin 2-related diseases |
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| CN105940108B (zh) | 2019-06-28 |
| ES2689862T3 (es) | 2018-11-16 |
| US11499155B2 (en) | 2022-11-15 |
| US20200308589A1 (en) | 2020-10-01 |
| EP3058071B1 (en) | 2018-08-15 |
| CN105940108A (zh) | 2016-09-14 |
| BR112016008644A2 (pt) | 2017-10-03 |
| WO2015055859A1 (en) | 2015-04-23 |
| JP2016540732A (ja) | 2016-12-28 |
| LT3058071T (lt) | 2018-10-25 |
| PT3058071T (pt) | 2018-10-25 |
| JP6920408B2 (ja) | 2021-08-18 |
| EP2862928A1 (en) | 2015-04-22 |
| RS57860B1 (sr) | 2018-12-31 |
| PL3058071T3 (pl) | 2018-12-31 |
| US10647986B2 (en) | 2020-05-12 |
| HRP20181450T1 (hr) | 2018-11-16 |
| AU2014336070B2 (en) | 2020-11-19 |
| US20160264976A1 (en) | 2016-09-15 |
| SI3058071T1 (sl) | 2018-11-30 |
| CA2927240A1 (en) | 2015-04-23 |
| AU2014336070A1 (en) | 2016-05-19 |
| CA2927240C (en) | 2022-12-13 |
| EP3058071A1 (en) | 2016-08-24 |
| JP6641270B2 (ja) | 2020-02-05 |
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