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UniQure gene therapy shows promising result in mid-stage trial

(Reuters) – Gene therapy company UniQure NV said on Friday its treatment for hemophilia B increased the levels of a protein that helps in blood clotting after 12 weeks in a relatively small study.

Common treatments for hemophilia B, in which a lack of the protein factor IX (FIX) prevents clotting that could lead to severe bleeding, include factor replacement therapy where the patient gets an injection of clotting protein.

Spark Therapeutics and Pfizer Inc are also developing a gene therapy for treating hemophilia B.

UniQure’s treatment aims to reduce bleeding by raising the FIX levels, and no patient suffered bleeding or needed replacement therapy during the trial period.

“With the FIX activity observed to date, we believe these patients could feel better without seeing signs of disease,” Cantor Fitzgerald analyst Elemer Piros said.

The updated data from a mid-stage study showed that all three patients treated with a single dose of the therapy, AMT-061, showed increased and sustained levels of factor IX (FIX), the company said.

FIX activity at 12 weeks increased to 38 percent of normal, which exceeded the threshold levels generally considered enough to eliminate or cut down the risk of bleeding cases, UniQure said.

The company, on a conference call, said it was confident of getting a speedy approval from U.S. regulators, considering the data.

UniQure in November reported initial data from the same trial, showing FIX activity of 31 percent of normal after six weeks of administration.

The positive data follows a bleak day for companies developing gene therapies and gene editing technology, after drugs developed by two small firms failed to show promise in separate clinical trials.

Interest in gene therapies, which use engineered viruses to carry healthy genetic material into the cells of sick people, has gone up greatly as the first few of such treatments get approval.

“There is, generally speaking, a large and high interest in gene therapy among patients, simply because of the hope of a cure,” Annette von Drygalski, an investigator who treated the first patient administered with AIM-061, said.

The company’s shares were up 4.6 percent at $34.72.

Reporting by Manas Mishra in Bengaluru; Editing by Sriraj Kalluvila and James Emmanuel

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