$uniQure N.V. - Ordinary Shares(QURE)$ The first patient achieved FIX activity of 37% of normal at ten weeks after administration. FIX activity in the second patient was 23% of normal at eight weeks following administration and in the third patient was 30% of normal at six weeks after administration. The second and third patients had previously screen-failed another gene therapy study due to pre-existing neutralizing antibodies to a different AAV vector.
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PharmSeeker2018-11-16 04:45

市场大的Hem B 送人, Hem A 没搞定, 自己留一个市场特别小的眼科,当时的决策太失败了。Hem A的vector的确很厉害,我很喜欢那个用转人肝细胞的小鼠模型,没想到在immunogenicity 上翻船。

感觉ONCE 有点像BMS, 一招错,步步错,满盘皆输。

usbiostock2018-11-16 02:02

COO只好辞职了。

PharmSeeker2018-11-16 01:44

once如果自己留着Hemo B,目前的市值大大低估。不明白为啥把这么好的东西给了Pfizer

usbiostock2018-11-15 23:46

ONCE其实技术并不差,就是点背。没想到QURE来这一手,后面在专利问题上估计要纠缠一大阵子。A型血友病BMRN完全压倒性优势。眼科产品也有几家公司虎视眈眈。

淘沙见金2018-11-15 23:37

这个可能ONCE COO辞职的一个原因?Long QURE/Short ONCE应该不错