Transforming drug discovery for human therapeutics with antisense technology

来源 :The 2nd Canton Nucleic Acids Forum(第二届广州核酸国际论坛) | 被引量 : 0次 | 上传用户:a1234578
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  The antisense oligonucleotide drug platform has now emerged as an important, mechanistically validated and therapeutically proven approach for drug discovery.This achievement is the result of a twenty-five year, multi-billion dollar investment involving academic research, biotechnology and large pharmaceutical companies.The first systemic, chronically administered antisense oligonucleotide drug has now been FDA approved.Moreover, dozens of antisense-based drugs are under evaluation in the final stages of clinical testing for a broad range of diseases, and registration applications are imminent.Thus, the antisense drug platform has now emerged as the third drug platform for human therapeutics, alongside small molecules and antibodies.This presentation will highlight the key achievements leading to the success of the antisense platform, and a vision toward its future role in human therapeutics.
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