Replies to post #192936 on Biotech Values

[DewDiligence]

QURE—Sort of plays off the monogenic comment you had made on Twitter [wrt AAVL’s program in AMD] and which I had been thinking about in regards to preclinical models of HF.

Yes—noticed the connection. I concur with your skepticism of the myriad preclinical models that show great success for various gene therapies in such multifaceted diseases as HF.

If it seems too good to be true…

[DewDiligence]

QURE reports updated phase-1/2 data in Sanfilippo B syndrome:

http://finance.yahoo.com/news/positive-topline-results-announced-phase-121245814.html

uniQure N.V…today announced the topline results of one-year follow-up data from a Phase I/II clinical trial conducted by Institut Pasteur…in partnership with the French Muscular Dystrophy Association and Vaincre les Maladies Lysosomales…in four Sanfilippo B syndrome (MPSIIIB) patients treated with a novel gene therapy, AMT-110. In all four patients, researchers verified the restoration of catalytical activity of the NaGlu protein in the cerebrospinal fluid (CSF) from 0% at baseline up to 14-17% of normal at 3 months with persistent effect at 12 months. [The vagueness of the “up to” phrase will presumably be addressed on the CC.]

…In the trial, four patients received a one-time administration of AAV5 gene therapy dosed over two hours directly into the brain. All patients were maintained under coverage of a continuous immune suppression regimen. No local inflammation or other safety concerns related to the therapy or the procedure have been identified. In addition to establishing the safety of the procedure and the AAV5 viral vector, the most important result was the presence of catalytically active NaGlu protein in the CSF measured at 1, 3, and 12 months after treatment. The fact that all treated Sanfilippo B subjects continued to gain skills throughout the study is extremely encouraging.

CC at 8:30am ET.