Ó¢¹úÒ©Æ·ºÍ±£½¡Æ·ÖÎÀí¾Ö£¨Ï³ÆMHRA£©¿ËÈÕÔÚ¹ÙÍøÐû²¼Í¨¸æ³Æ£¬£¬£¬£¬£¬Åú×¼ÃÀ¹ú¸£Ì©ÖÆÒ©£¨Vertex Pharmaceuticals£©ÓëÈðÊ¿CRISPR TherapeuticsÏàÖú¿ª·¢µÄCRISPR»ùÒò±à¼ÁÆ·¨examglogene autotemcel£¨¼ò³ÆExa-cel£¬£¬£¬£¬£¬Æ·ÅÆÃû³ÆCasgevy£©ÓÐÌõ¼þÉÏÊÐÔÊÐí£¬£¬£¬£¬£¬ÓÃÓÚÖÎÁÆ12Ëê¼°ÒÔÉÏÁµ¶ÐÍϸ°ûѪÐ鲡£¡£¡£¡£¨SCD£©°é¸´·¢ÐÔѪ¹Ü±ÕÈûΣÏó£¨VOCs£©»¼Õߣ¬£¬£¬£¬£¬ÒÔ¼°ÎÞ·¨»ñµÃÈËÀà°×ϸ°û¿¹Ô£¨HLA£©Æ¥ÅäÔìѪ¸Éϸ°ûÒÆÖ²ÖÎÁƵÄÊäѪÒÀÀµÐԦµØÖк£ÑªÐ飨TDT£©»¼Õß¡£¡£¡£¡£
MHRA³Æ£¬£¬£¬£¬£¬ÕâÊÇÈ«ÇòÊ׸ö»ñÅúÖÎÁƸÃ˳Ӧ֢µÄ»ùÒò±à¼ÁÆ·¨£¬£¬£¬£¬£¬Ò²ÊÇÈ«ÇòÊ׸ö»ñÅúÓ¦Óá°»ùÒò½Â¼ô¡±CRISPRµÄÁÆ·¨¡£¡£¡£¡£CRISPRÁÆ·¨ÊÇ2020Äêŵ½±µÃÖ÷Emmanuelle CharpentierºÍJennifer DoudnaÔÚ2012ÄêһƪÂÛÎÄÖÐÌá³öµÄÊÖÒÕ¡£¡£¡£¡£
ËäÈ»Á½¼ÒÒ©ÆóÉÐδÐû²¼ÊÛ¼Û£¬£¬£¬£¬£¬µ«Æ¾Ö¤´ËǰÃÀ¹úʳҩ¼à¾Ö£¨FDA£©Åú×¼µÄÀ¶ÄñÉúÎBluebird Bio£©µÄ»ùÒòÁÆ·¨²úÆ·ZyntegloÀ´ÍÆË㣬£¬£¬£¬£¬Casgevy¼ÛÇ®»òÐíÂÊ»áÊÇ¡°°ÙÍòÃÀÔª¡±¼¶±ðµÄ¡°Ìì¼ÛÒ©Î¡£¡£¡£¡£
¾ÝϤ£¬£¬£¬£¬£¬´Ë´ÎMHRA¶ÔCasgevy¸øÓèÓÐÌõ¼þÉÏÊÐÔÊÐíÊÇÓÉÓÚ¸ÃÁÆ·¨ÓÐÖØ´óµÄ¡°Î´Öª×ãÐèÇó¡±»òÓëÏÖÓеÄÁÆ·¨Ïà±È¾ßÓÐǿʢÖÎÁÆÓÅÊÆ¡£¡£¡£¡£MHRAµÄÓÐÌõ¼þÔÊÐíÓÐÓÃÆÚΪһÄ꣬£¬£¬£¬£¬µ«ÊÜÅúÖ÷Ìå¿ÉÒÔ»ùÓÚÁÙ´²Êý¾ÝµÄÒ»Á¬¸üÐÂͨ¹ýÄêÉ󡣡£¡£¡£
MHRAµÄÒ½ÁƱ£½¡ÖÊÁ¿ºÍ¿É¼°ÐÔÔÝʱִÐÐÖ÷ÈÎJulian BeachÔÚͨ¸æÖÐÌåÏÖ£¬£¬£¬£¬£¬SCDºÍTDTÊÇÍ´¿àµÄÖÕÉú¼²²¡£¬£¬£¬£¬£¬ÔÚijЩÇéÐÎÏ¿ÉÄÜÒ²ÊÇÖÂÃüµÄ¡£¡£¡£¡£Æù½ñΪֹ£¬£¬£¬£¬£¬¹ÇËèÒÆÖ²£¨±ØÐèÀ´×ÔÇ×½üÆ¥ÅäµÄ¾èÔùÕß²¢±£´æÇãÔþΣº¦£©ÊÇΨһµÄÓÀÊÀÐÔÖÎÁÆÑ¡Ôñ¡£¡£¡£¡£
ƾ֤MHRAÖÜËÄÐû²¼µÄÅú׼ͨ¸æ£¬£¬£¬£¬£¬CasgevyµÄÓÐÌõ¼þÉÏÊÐÔÊÐí»ùÓÚÁ½ÏîÈ«ÇòÁÙ´²ÊÔÑéµÄЧ¹û¡£¡£¡£¡£
ÆäÖУ¬£¬£¬£¬£¬SCDÑо¿ÕÐļÁË45ÃûÊÜÊÔÕߣ¬£¬£¬£¬£¬µ«Õâµ±ÖÐÖ»ÓÐ29ÃûÊÜÊÔÕßÌṩÁË×ãÒÔ¾ÙÐÐÖ÷ÒªÁÆÐ§ÖÐÆÚÆÊÎöµÄ³ÉÊìÊý¾Ý¡£¡£¡£¡£ÔÚÕâЩÊÜÊÔÕßÖУ¬£¬£¬£¬£¬³ý1ÈËÍ⣬£¬£¬£¬£¬ÆäËûÈ˾ùÔÚ½ÓÊÜCasgevyÖÎÁƺóÔÚÖÁÉÙ12¸öÔÂÄÚÏû³ýÁËÑÏÖØµÄÌÛÍ´¸Ð¡£¡£¡£¡£¾ÝĬɳ¶«ÕïÁÆÊÖ²áÏÈÈÝ£¬£¬£¬£¬£¬SCDÊÇѪºìÂѰ׵ÄÒ»ÖÖÒÅ´«ÐÔ»ùÒòÒì³££¬£¬£¬£¬£¬ÌØÕ÷ÊǺìϸ°û³ÊÁµ¶£¨ÐÂÔ£©ÐΡ£¡£¡£¡£ÓÉÓÚÁ״ϸ°û½©Ó²£¬£¬£¬£¬£¬ÒÔÊÇÄÑÒÔͨ¹ýÉíÌåÖÐ×îСµÄѪ¹Ü£¨Ã«Ï¸Ñª¹Ü£©£¬£¬£¬£¬£¬µ¼ÖÂѪÁ÷ÛÕ±Õ£¬£¬£¬£¬£¬Òý·¢ÌÛÍ´¡¢Æ÷¹ÙË𺦵ÈЧ¹û¡£¡£¡£¡£
ÔÚTDTÊÜÊÔÕßµÄÁÙ´²ÊÔÑéÖУ¬£¬£¬£¬£¬ÓÐ42Ãû»¼ÕßÌṩÁ˳ÉÊìÊý¾Ý£¬£¬£¬£¬£¬ÆäÖÐ39Ãû»¼ÕßÔÚ½ÓÊÜCasgevyÖÎÁƺóÖÁÉÙ12¸öÔÂÄÚ²»ÔÙÐèÒªÊäºìϸ°û£¬£¬£¬£¬£¬ÁíÍâ3Ãû»¼ÕßµÄÊäѪÐèÇóϽµÁË70%¡£¡£¡£¡£
ÉÏÊöÁ½ÏîÑо¿»¹ÏÔʾ£¬£¬£¬£¬£¬CasgevyµÄÇå¾²ÐÔÓÅÒ죬£¬£¬£¬£¬Æä¸±×÷ÓÃÓë×ÔÌå¸Éϸ°ûÒÆÖ²»ù±¾Ò»Ö¡£¡£¡£¡£³£¼ûµÄ²»Á¼·´Ó¦°üÀ¨Æ£ÀÍ¡¢·¢ÈÈ¡¢¶ñÐĺÍѬȾΣº¦ÔöÌí¡£¡£¡£¡£