肽​载体​上​的​多个​寡​核苷酸​部分

源语言标题
(CN106459975) 肽​载体​上​的​多个​寡​核苷酸​部分
公开号/公开日
CN106459975 A 2017-02-22 [CN106459975] / 2017-02-22
申请号/申请日
2015CN-80027369 / 2015-05-22
发明人
WEEDEN TIMOTHY E;NELSON CAROL A;WENTWORTH BRUCE M;CLAYTON NICHOLAS P;LEGER ANDREW;
申请人
GENZYME;
主分类号
IPC分类号
C12N-015/113
摘要
(CN106459975) 本文​涉及​反义​寡​核苷酸(AON),例如​磷​二​酰胺​吗​啉​代​寡​核苷酸(PMO)。本文​进步​涉及​多个​PMO​与​阳离子​细胞​穿透​肽(CPP)缀合​以​增强​PMO​吸收​至​骨骼肌​和​心肌​细胞。
地址
代理人
代理机构
;
优先权号
2014US-62002296 2014-05-23 2015WO-US32142 2015-05-22
主权利要求
(CN106459975) 1. One AON a peptide-conjugated compound, a compound comprising at least two AON, AON compound of formula (I), (II), (III), (IV), (V) or (VI) 15 to 30 subunits in length to, Wherein B is selected from C, G, A or T naturally occurring purine or pyrimidine nucleotide base; Wherein at least two AON compounds have the target front-mRNA or mRNA nucleotide complementary to the nucleotide base sequence of at least 75%; Wherein at least two AON 0 to 3 repeating subunits of a compound having, wherein B is a G; Wherein at least two AON compound has less than 60% of the subunits, wherein B is a C or G; Wherein at least two compounds having a non-self-complementary sequence AON; with Wherein at least two AON compound with conjugated poly-cationic peptide.  2. One a peptide-conjugated phosphorus diamide compound mo, comprising at least two phosphorous diamide a morpholino compound, the phosphorus compound the diamide mo formula (I) 15 to 30 subunits of the: Wherein R is a alkyl and B is selected from C, G, A or T naturally occurring purine or pyrimidine nucleotide base; Wherein at least two phosphorous diamide mo target compound has the front-mRNA or mRNA of at least 8 contiguous bases complementary to the nucleotide base sequence; Wherein at least two phosphorus diamide compound having repeating subunits of 0 to 3 mo, wherein B is a G; Wherein at least two phosphorus compound has less than 60% of the subunits of the diamide mo, wherein B is a C or G; Wherein at least two phosphorus diamide compound having a non-self-complementary sequence mo; with Wherein at least two phosphorus diamide compound with the poly-cationic peptide conjugated mo.
法律状态
(CN106459975) LEGAL DETAILS FOR CN106459975  Actual or expected expiration date=2035-05-22    Legal state=ALIVE    Status=PENDING     Event publication date=2015-05-22  Event code=CN/APP  Event indicator=Pos  Event type=Examination events  Application details  Application country=CN CN201580027369  Application date=2015-05-22  Standardized application number=2015CN-80027369     Event publication date=2017-02-22  Event code=CN/A  Event type=Examination events  Published application  Publication country=CN  Publication number=CN106459975  Publication stage Code=A  Publication date=2017-02-22  Standardized publication number=CN106459975     Event publication date=2017-02-22  Event code=CN/C06  Event indicator=Pos  Event type=Examination events  Publication    Event publication date=2017-04-05  Event code=CN/SE01  Event type=Examination events  ENTRY INTO FORCE OF REQUEST FOR SUBSTANTIVE EXAMINATION
专利类型码
A
国别省市代码
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