AKOPIAN, Violetta,MINOMI, Kenjiro,NIITSU, Yoshiro,PAYNE, Joseph E.,KNOPOV, Victor,WITTE, Richard P.,AHMADIAN, Mohammad,PERELMAN, Loren A.,TANAKA, Yasunobu,FEINSTEIN, Elena,AVKIN-NACHUM, Sharon,KALINSK
申请号：
ES16169874
公开号：
ES2689696T3
申请日:
2012.06.08
申请国别(地区):
ES
年份:
2018
代理人:
摘要：
Pharmaceutical composition comprising (i) a double stranded nucleic acid molecule and a drug carrier comprising a mixture of a retinoid and a lipid, in which the double stranded nucleic acid molecule comprises the structure (A1): (A1) 5 ' (N) x - Z 3 '(antisense chain) 3' Z '- (N') and -z "5 '(sense chain) in which each of N and N' is a nucleotide that may not be modified or be modified, or an unconventional moiety； in which each of (N) xy (N ') and is an oligonucleotide in which each consecutive N or N' is linked to the next N or N 'by a covalent bond； in which each of Z and Z 'is independently present or absent, but if present independently includes 1-5 nucleotides or residues other than consecutive nucleotides or a combination thereof covalently linked to the 3'-terminal end of the chain in the that is present； in which z "may be present or absent, but if present it is a remainder of occupation of reactive centers covalently linked to the 5'-terminal end of (N ') and； in which each of x and y is independently an integer between 18 and 40； wherein the sequence of (N ') y has complementarity with respect to the sequence of (N) x； wherein (N) x includes an antisense sequence with respect to the mRNA coding sequence for human hsp47 shown by way of example by SEQ ID NO: 1； and wherein the lipid comprises a lipid selected from the group consisting of ** Formula ** or (ii) a double stranded nucleic acid molecule comprising a sense chain and an antisense chain in which the sense and antisense chains are selected from the oligonucleotides described as SERPINH1_2 (SEQ ID NO: 60 and 127), SERPINH1_45a (SEQ ID NO: 98 and 165) and SERPINH1_51 (SEQ ID NO: 101 and 168), and a drug carrier comprising a mixture of a lipid vesicle and a retinoid or a retinoid conjugate, in which the lipid vesicle comprises a lipid selected from the group consisting of ** Formula **Composición farmacéutica que comprende (i) una molécula de ácido nucleico bicatenario y un portador de fármaco que com
