JOP20170057B1 - مركبات ترابطية مستهدفة كمركبات علاجية - Google Patents

مركبات ترابطية مستهدفة كمركبات علاجية

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Publication number
JOP20170057B1
JOP20170057B1 JOP/2017/0057A JOP20170057A JOP20170057B1 JO P20170057 B1 JOP20170057 B1 JO P20170057B1 JO P20170057 A JOP20170057 A JO P20170057A JO P20170057 B1 JOP20170057 B1 JO P20170057B1
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Jordan
Prior art keywords
targeting ligands
expression
compounds
target
inhibiting oligomeric
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JOP/2017/0057A
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English (en)
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Pei Tao
B Rozema David
H Wakefield Darren
V Blokhin Andrei
D Benson Jonathan
Li Zhen
Fleitz Fred
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Arrowhead Pharmaceuticals Inc
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
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    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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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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Abstract

يتعلق الاختراع الحالي بمركبات ترابطية مقترنة والتي يمكن ربطها بمركبات أوليجومرية مثبطة للتعبير الجيني، مثل عوامل تداخل RNA (RNAi)، والتي تكون مفيدة في توجيه المركبات الأوليجومرية المثبطة للتعبير الجيني إلى خلايا الكبد لتعديل التعبير الجيني. يمكن استخدام المركبات الترابطية المقترنة التي تم الكشف عنها في هذه الوثيقة، عند اقترانها مع مركب أوليجومري مثبط للتعبير الجيني، في مجموعة متنوعة من التطبيقات، التي تتضمن الاستخدام في تطبيقات علاجية، تشخيصية، التحقق من الهدف، وتطبيقات اكتشاف مجموعة العوامل الوراثية. تكون التركيبات التي تشتمل على المركبات الترابطية المقترنة المرتبطة بمركبات أوليجومرية مثبطة للتعبير الجيني والتي تم الكشف عنها هنا قادرة على التوسط فى التعبير الجيني لمتواليات حمض نووي مستهدفة في خلايا الكبد، مثل الخلايا الكبدية، التي يمكن أن تكون مفيدة في علاج الأمراض أو الحالات التي تستجيب لتثبيط التعبير الجيني أو النشاط في خلية، نسيج أو كائن حي.
JOP/2017/0057A 2016-03-07 2017-03-07 مركبات ترابطية مستهدفة كمركبات علاجية JOP20170057B1 (ar)

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US201662304652P 2016-03-07 2016-03-07
US201662370754P 2016-08-04 2016-08-04
US201662426916P 2016-11-28 2016-11-28

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JOP/2017/0057A JOP20170057B1 (ar) 2016-03-07 2017-03-07 مركبات ترابطية مستهدفة كمركبات علاجية
JOP/2021/0231A JOP20210231A1 (ar) 2016-03-07 2021-08-19 مركبات ترابطية مستهدفة كمركبات علاجية

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US (4) US10246709B2 (ar)
EP (1) EP3426261A4 (ar)
JP (4) JP6983797B2 (ar)
KR (3) KR102348425B1 (ar)
CN (2) CN109069529B (ar)
AU (3) AU2017229469B2 (ar)
BR (1) BR112018068230B1 (ar)
CA (1) CA3011946A1 (ar)
IL (3) IL296377A (ar)
JO (2) JOP20170057B1 (ar)
MX (3) MX2018009854A (ar)
TW (3) TWI783434B (ar)
UY (1) UY37146A (ar)
WO (1) WO2017156012A1 (ar)

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