HUE027169T2 - Komplement antagonisták és azok alkalmazása - Google Patents

Komplement antagonisták és azok alkalmazása Download PDF

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HUE027169T2
HUE027169T2 HUE09788222A HUE09788222A HUE027169T2 HU E027169 T2 HUE027169 T2 HU E027169T2 HU E09788222 A HUE09788222 A HU E09788222A HU E09788222 A HUE09788222 A HU E09788222A HU E027169 T2 HUE027169 T2 HU E027169T2
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oligomer
rna
lna
seq
nucleotide
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HUE09788222A
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Frank Baas
Kees Fluiter
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Regenesance B V
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    • 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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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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Claims (4)

  1. S» Oligomer a 2. igénypont szerint* ahol a módosítóit tatern-ukleozld kötés egy foszfort*oát huenm kieozkl kötés, ő. Oligomer a 2. Igénypont szerinL ahol a módosított oukleobázis az őmteíihebozm,
    7, Oligomer az I , igénypont szeriek ahol az oligomer 12~?ői 45ág mtkieotld hosszúságii
    8, CMIgomer az i. igénypont szerint, ahol az oligomer í 0-től 18-ig nukleotkl hosszúságú. % Oligomer az 1. igénypont szerint, ahol az oligomer a 132, 453,. 566, /26 vagy 1035 mmleotkiokra van irányítva a SEQ ID NO: I -nek az ΛΤΟ start helyéből, I#. Oligomer az L vagy 9, igénypont szerint, ahol az oligomer legalább 90'%-os, 5X>%-os, 97%-os, 98%-os vagy 99%-os szekvencia-identitással rendelkezik a Komplement-Kotoponerts €Ö szekvenciához viszonyítva, amely a SEQ ID NO: 1 által van képviselve, Π. Gyóg yszerészeti készítmény, amely tartalmazza az L vagy 2, igénypont szerinti oiigomeo és egy gyógyszerészeti leg elfogadható Mgitét, hordozol sőt vagy adjuvánst. .12, Legalább egy az, i, igénypont szeriinti oligomer alkalmazása egy gyógyszernek az előállítása céljából egy állapotnak a kezelésére, amely axonáiis regenerációt igényel, 13» Legalább egy az: !, igénypont szerinti oligomer alkalmazása egy gyógyszernek az előállítása céljából egy kréniknsdemielMzáC'iás állapot, kezelésére, 14 Alkalmazás a 1B. igénypont szerint, ahol a krónikus demieiinixacios állapot a szklerózis multiplex, egy krónikus detmeiinrzádos neto'opartu, iá és IB típusú liMSN (CMT) betegség, Aizhelmemkőr, iitmtingion-kor, Charcoi-Marté-Tooíh betegség, amioirolMs lateráiszklerózis ( A11..S}, Gtuilam-Barre szindróma (G 88.. amely szintén .ismert a km gyulladásos deoueiiuizáctős polmeuropáíta vagy A1DP), leofeodtsziróíta vagy Parkinson· kór. KOMPLEMENT ANTAGONISTÂK ÉS AZOK ALKALMAZÁSA. Szabadalmi igény pirítok P .Egyszálú aotiszensz oligomer, amely 10-tél 5ö-ig' npkjeotid hosszúságú, és amely rendelkezik egy összefüggő rmkleobázis-szekvenelával, amely legalább 80%-os szekvencia-identitással rmlelkezlk egy nukleiosav egy komplementer régiójához viszonyítva, amely kódolja a KOMFIJ'IA^:iNTKOMIX)NÉNS 6 (€6) szekvenciát, amely a SEQ ID NO; l által van képviselve; ahol az oligomer tartalmaz legalább egy nukleont! analógot és képes arra, hogy a €6 mRNS expressziónak a szintje legyen csökkentve egy emlősben legalább 20%-ak ahogyan az egy qfÇR-vizsgálat révén van meghatározva, ahol az oligomer az 112-152, 433-473, 546-586, 706-746, vagy 1015-1055 nukleoüdokra van irányítva a SEQ !D NO; 1-nek az A TG start helyéből
  2. 2, Oligomer az L igénypont szerint, ahol az oligomer továbbá tartalmazza legalább az egyiket egy Módosított irrternukleozid kötés és egy módosított. nnkleobázis kőzni.
  3. 3, Oligomer az i, igénypont szerint,, ahol a nukkobd analóg egy módosított cukor-rész, amely a. következőknél álló csoportból van választva; egy 2'-0-tnetoxl-etil módosított cukor-rész, egy T’-metoxi módosított cukor-rész, egy 2'-0- atkái módosított cukor-rész és egy bi ciklusos ott kor-rész,
  4. 4, Obgomer a 3. igénypont szerint, ahol a biciilusos cukor-rész egy zárt nukleinsav (LNA, angolul; „locked nucleic ackP) monomer.
HUE09788222A 2008-07-10 2009-07-10 Komplement antagonisták és azok alkalmazása HUE027169T2 (hu)

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US (2) US8703730B2 (hu)
EP (1) EP2320925B1 (hu)
JP (1) JP2011527568A (hu)
KR (1) KR20110044983A (hu)
CN (2) CN103740713A (hu)
AU (1) AU2009270020B2 (hu)
CA (1) CA2730203C (hu)
CY (1) CY1117476T1 (hu)
DK (1) DK2320925T3 (hu)
ES (1) ES2566008T3 (hu)
HR (1) HRP20160261T1 (hu)
HU (1) HUE027169T2 (hu)
IL (1) IL210506A (hu)
NZ (1) NZ591057A (hu)
PL (1) PL2320925T3 (hu)
SI (1) SI2320925T1 (hu)
SM (1) SMT201600098B (hu)
WO (1) WO2010005310A2 (hu)

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