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AVR-RD-02 is an investigational ex vivo lentiviral gene therapy developed by AVROBIO Inc. It consists of autologous CD34+ enriched hematopoietic stem cells (HSCs) that have been genetically modified ex vivo with a lentiviral vector (LV) to contain a ribonucleic acid (RNA) transcript that, after reverse transcription, results in codon-optimized, complementary deoxyribonucleic acid (cDNA) that, upon its integration into human genome, encodes for functional human glucocerebrosidase (GCase). AVR-RD-02 is being investigated in Gaucher Disease.
AVR-RD-02 is an investigational ex vivo lentiviral gene therapy developed by AVROBIO Inc. It consists of autologous CD34+ enriched hematopoietic stem cells (HSCs) that have been genetically modified ex vivo with a lentiviral vector (LV) to contain a ribonucleic acid (RNA) transcript that, after reverse transcription, results in codon-optimized, complementary deoxyribonucleic acid (cDNA) that, upon its integration into human genome, encodes for functional human glucocerebrosidase (GCase). AVR-RD-02 is being investigated in Gaucher Disease.
每项发明一条，涵盖美国、欧洲、PCT 与中国的同族专利。
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专利文献通常使用化学名或研发代号指代药物，而非通用名；且专利申请在提交约 18 个月后才公开，较新的药物可能尚未出现在这里。
