Vertex Pharmaceuticals¹«Ë¾ÈÕÇ°Ðû²¼ £¬£¬ £¬£¬FDAÒѾÔÊÐí¸Ã¹«Ë¾µÄÔÚÑÐÁÆ·¨VX-264Æô¶¯ÁÙ´²ÊÔÑé¡£¡£¡£VX-264ÊÇÒ»¿î¸Éϸ°û·Ö½â±¬·¢µÄÒȵºÏ¸°ûÁÆ·¨ £¬£¬ £¬£¬°ü×°ÔÚVertex¿ª·¢µÄ¶ÀÍÌÃâÒß±£»£»£»£»£»¤Ò½ÁÆÆ÷еÖÐ £¬£¬ £¬£¬¾ßÓÐÔÚ²»ÐèҪʹÓÃÃâÒßÒÖÖƼÁµÄÇéÐÎÏ £¬£¬ £¬£¬¹¦Ð§ÐÔÖÎÓú1ÐÍÌÇÄò²¡µÄDZÁ¦¡£¡£¡£
1ÐÍÌÇÄò²¡»¼ÕßÓÉÓÚÃâÒßϵͳÆÆËðÒÈÏÙÖб¬·¢ÒȵºËصÄÒȵºÏ¸°û £¬£¬ £¬£¬µ¼ÖÂÒȵºËر¬·¢ïÔ̺͸ßѪÌÇ¡£¡£¡£¸ßѪÌǻᵼÖÂÌÇÄò²¡ÍªÖ¢ËáÖж¾ £¬£¬ £¬£¬Ëæ×Åʱ¼äµÄÍÆÒÆ £¬£¬ £¬£¬»áÒýÆð²¢·¢Ö¢ £¬£¬ £¬£¬ÈçÉö²¡/Ë¥½ß¡¢ÑÛ¼²¡¢ÐÄÔಡ¡¢Öз硢Éñ¾ËðÉË £¬£¬ £¬£¬ÉõÖÁéæÃü¡£¡£¡£ÓÉÓÚÒȵºËظøҩϵͳµÄ¾ÖÏÞÐÔºÍÖØ´óÐÔ £¬£¬ £¬£¬»¼ÕߺÜÄѵִïºÍά³ÖѪÌÇƽºâ¡£¡£¡£ÔÚ½ÓÊÜÒȵºËØÖÎÁƵĻ¼ÕßÖÐ £¬£¬ £¬£¬ÒȵºËØÓÃÁ¿¹ý¸ß»áµ¼ÖµÍѪÌDZ¬·¢¡£¡£¡£
2021Äê £¬£¬ £¬£¬Vertex¹«Ë¾Ðû²¼ £¬£¬ £¬£¬Æä»ùÓÚ¸Éϸ°û·Ö½âµÄÒȵºÏ¸°ûÁÆ·¨VX-880ÔÚÊ×λ½ÓÊÜÖÎÁƵĻ¼ÕßÌåÏÖ³ö¡°Ê·ÎÞÇ°Àý¡±µÄÊÔÑéЧ¹û¡£¡£¡£ÔÚ½ÓÊܵ¥¼ÁÖÎÁƺóµÚ90Ììʱ £¬£¬ £¬£¬»¼Õ߻ָ´ÒȵºËØÉú²ú £¬£¬ £¬£¬²¢ÇÒ½«ÌìÌìÒȵºËØʹÓÃÁ¿ïÔÌ91%¡£¡£¡£¸Ã¹«Ë¾Ö¸³ö £¬£¬ £¬£¬ÕâÊÇÊ×´ÎÊÔÑéÊý¾Ý֤ʵÕâһϸ°ûÁÆ·¨¿ÉÏÔÖø»Ö¸´1ÐÍÌÇÄò²¡»¼ÕßµÄÒȵºÏ¸°û¹¦Ð§¡£¡£¡£²»¹ýÓÉÓÚVX-880ÊÇʹÓÿµ½¡¹©ÌåÌṩµÄ¸Éϸ°û·Ö½â±¬·¢µÄͬÖÖÒìÌåϸ°ûÁÆ·¨ £¬£¬ £¬£¬1ÐÍÌÇÄò²¡»¼ÕßÐèÒªºã¾Ã·þÓÃÃâÒßÒÖÖƼÁÀ´ÒÖÖÆÉíÌåµÄÃâÒßÇãÔþ·´Ó¦¡£¡£¡£
VX-264ÊÇÒ»ÖÖϸ°ûÁÆ·¨ºÍÒ½ÁÆÆ÷еµÄ×éºÏ £¬£¬ £¬£¬½«VX-880·â×°ÔÚVertex¿ª·¢µÄÒ½ÁÆÆ÷еÖС£¡£¡£ÕâÒ»×°ÖÿÉÒÔͨ¹ýÊÖÊõÖ²Èëµ½»¼ÕßÌåÄÚ £¬£¬ £¬£¬ÔÚ±£»£»£»£»£»¤Ï¸°û²»ÊÜÃâÒßÇãÔþµÄͬʱ £¬£¬ £¬£¬ÊÍ·Åϸ°ûÌìÉúµÄÒȵºËØ¡£¡£¡£VertexÍýÏëÔÚδÀ´¼¸¸öÔÂÀïÔÚ1ÐÍÌÇÄò²¡»¼ÕßÖÐÆô¶¯1/2ÆÚÁÙ´²ÊÔÑé¡£¡£¡£±ðµÄ £¬£¬ £¬£¬¸Ã¹«Ë¾»¹ÔÚʹÓÃCRISPR»ùÒò±à¼ÊÖÒÕ £¬£¬ £¬£¬¿ª·¢ÌÓ±ÜÈËÌåÃâÒßϵͳ¹¥»÷µÄÒȵºÏ¸°ûÁÆ·¨¡£¡£¡£
¡°VX-264ʹÓõĸÉϸ°ûÑÜÉúÒȵºÏ¸°ûÓëÎÒÃÇÒѾ»ñµÃ¿´·¨ÑéÖ¤µÄVX-880ÏîÄ¿Ïàͬ¡£¡£¡£ÐÂÔöµÄ¶ÀÍÌÃâÒß±£»£»£»£»£»¤Æ÷еÈÃÎÒÃÇ¿ÉÒÔÃâÈ¥ÃâÒßÒÖÖƵÄÒªÇ󡣡£¡£¡±VertexÖ´Ðи±×ܲüæϸ°ûºÍ»ùÒòÁÆ·¨ÈÏÕæÈËBastiano Sanna²©Ê¿Ëµ £¬£¬ £¬£¬¡°ÎÒÃǺÜÐË·ÜÄܹ»½«µÚ¶þ¿îÖÎÁÆ1ÐÍÌÇÄò²¡µÄÏîÄ¿ÍÆÈëÁÙ´² £¬£¬ £¬£¬ÆÚ´ý½«ÕâÒ»Àå¸ïÐÔ £¬£¬ £¬£¬Ç±ÔÚÖÎÓúÐÔÁÆ·¨´ø¸ø»¼Õß¡£¡£¡£¡±
£¨ÔÎÊÌ⣺Ïò¹¦Ð§ÐÔÖÎÓúÌÇÄò²¡½ø·¢£¡ÐÂÒ»´ú¸Éϸ°ûÁÆ·¨²½ÈëÁÙ´²£©
ȪԴ£ºÍ¬»¨Ë³²Æ¾
