uniQure says gene therapy helps clotting in two hemophilia patients

8 January 2016

Shares in Dutch human gene therapy specialist uniQure NV (Nasdaq: QURE) were up 4.2% in pre-market trade on Nasdaq Friday following the company’s Thursday announcement of data indicating its experimental treatment for hemophilia B increased blood clotting ability in two patients.

Shares in the company jumped as much as 21% Thursday.

The company said the therapy, AMT-060, was designed to fix a genetic flaw caused by missing or defective factor IX, a protein that clots blood. The two adult patients received a low dose of the treatment. Factor IX rose to 5.5% and 4.5% of the normal level from less than 2% in the patients. Three other people who received a low dose of the therapy in the trial haven’t yet reached the full 12 weeks of follow-up.

This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Try before you buy

Free

7 day trial access

Take a Free Trial

All the news that moves the needle in pharma and biotech
Exclusive features, podcasts, interviews, data analyses and commentary from our global network of life sciences reporters.
Receive The Pharma Letter daily news bulletin, free forever.

Become a subscriber

£820

Or £77 per month

Subscribe Now

Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
Daily roundup of key events in pharma and biotech.
Monthly in-depth briefings on Boardroom appointments and M&A news.
Choose from a cost-effective annual package or a flexible monthly subscription

The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK