近日,美国康涅狄格大学的研究人员在药物代谢领域的著名期刊Drug Metabolism and Disposition上发表了题为《Absorption, Distribution, Metabolism, and Excretion of FDA approved Antisense Oligonucleotide Drugs》的综述[1]。他们选择了已被FDA批准的10种反义寡核苷酸(Antisense oligonucleotide,ASO)药物,Fomivirsen、Pegaptanib...
1.FDA-Approved Oligonucleotide Therapies in 2017.Mol Ther. 2017 May 3;25 (5):1069-1075. doi: 10.1016/j.ymthe.2017.03.023. 2.Pharmacology of Antisense Drugs. Annu Rev Pharmacol Toxicol 2017 Jan;57 81-105. 3.Molecular Mechanisms of Antisense Oligonucleotides. Nucleic Acid Ther. 2017 April 1...
Distribution, Metabolism, and Excretion of FDA approved Antisense Oligonucleotide Drugs》的综述[1]中,他们选择了已被FDA批准的10种反义寡核苷酸(Antisense oligonucleotide,ASO)药物,Fomivirsen、Pegaptanib、Mipomersen、Nusinersen、Inotersen、Defibrotide、Eteplirsen、Golodirsen、Viltolarsen和Casimersen...
参考文献: 1.FDA-Approved Oligonucleotide Therapies in 2017.Mol Ther. 2017 May 3;25 (5):1069-1075. doi: 10.1016/j.ymthe.2017.03.023. 2.Pharmacology of Antisense Drugs. Annu Rev Pharmacol Toxicol 2017 Jan;57 81-105. 3.Molecular Mechanisms o...
美国康涅狄格大学研究人员在《Drug Metabolism and Disposition》期刊上发表综述，题为《Absorption, Distribution, Metabolism, and Excretion of FDA approved Antisense Oligonucleotide Drugs》。文章聚焦于已被美国食品和药物管理局（FDA）批准的10种反义寡核苷酸（ASO）药物的ADME特征。通过集合分析，总结了ASO...
However, newly developed large molecule biological drugs, including antisense oligonucleotide (ASO) drugs, have substantially different ADME parameters. ASO drugs are single‐stranded antisense nucleic acids with diverse modes of drug action that induce mRNA degradation, exon s...
2020年,FDA批准53个新药,其中包含“Small Molecule”35个,“Antibody”10个,“Radioconjugate”1个,“Peptide”1个,“siRNA”1个,“Protein”1个,“Oligonucleotide”1个,“Antibody-drug Conjugate”2个和“Lipid”1个。 虽与2018年的59个新药获批有一段小距离,但FDA在2020年保持了新药审批的惊人步伐,同时对COVI...
Tab.2 Classifications of the Approved Orphan Drugs 3、 讨论 Evaluate Pharma数据库预测,2028年全球罕见病市场规模将达到3 000亿美元,占全球处方药市场规模的20%[11],大部分销售额将来自于“重磅炸弹”的抗肿瘤药孤儿药。因此,在评审孤儿药时更应综合考虑患者未满足的临床需求,引导企业去开发无药物治疗的罕见病...
Qalsody is an antisense oligonucleotide (ASO) designed to bind toSOD1mRNA to reduce SOD1 protein production. Qalsody is indicated for the treatment of ALS in adults who have a mutation in theSOD1gene in the U.S. This indication is approved under accelerated approval based on reduction in...
2020年,FDA批准53个新药,其中包含“Small Molecule”35个,“Antibody”10个,“Radioconjugate”1个,“Peptide”1个,“siRNA”1个,“Protein”1个,“Oligonucleotide”1个,“Antibody-drug Conjugate”2个和“Lipid”1个。 虽与2018年的59个新药获批有一段小距离,但FDA在2020年保持了新药审批的惊人步伐,同时对COVI...