uniQure Presents New Data Demonstrating Successful and Effective Transduction of AAV5 Vector in the Presence of Pre-Existing Neutralizing Antibodies in Non-Human Primates

-- Strong Dose-Dependent Transduction in All Animals Tested Regardless of the Level of Anti-AAV5 Antibodies at Pre-Administration --
-- Data Suggests AAV5 Gene Therapies May be Viable Treatments for Nearly All Patients --

-- Preclinical Data Presented Today at American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Washington, D.C.
-- Investor Webcast Friday, May 12, 2017 at 7:00 a.m. ET --

LEXINGTON, MA, USA and AMSTERDAM, The Netherlands I May 10, 2017 I uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today presented new preclinical data demonstrating successful and effective transduction of AAV5 in non-human primates with pre-existing anti-AAV5 neutralizing antibodies (NABs). At all observed levels, pre-existing neutralizing antibodies for AAV5 did not have a negative impact on the transduction effectiveness of the AAV5 vector. This suggests a much broader potential population of eligible patients than previously expected for AAV5-based gene therapies, including AMT-060, uniQure’s investigational gene therapy in patients with severe hemophilia B.

The data were presented today at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Washington, D.C., the preeminent gene and cell therapy conference in the world.

In ongoing gene therapy clinical trials using adeno-associated virus (AAV) vectors, patients who present levels of anti-AAV NABs are excluded from treatment due to concern that the efficacy of AAV vectors may be negatively influenced by their presence. In this study, uniQure researchers sought to assess the impact of pre-existing anti-AAV5 NAB levels on the liver transduction efficacy of an AAV5-based vector. 

Preclinical Data Findings

Fourteen non-human primates with pre-existing anti-AAV5 NABs at titers ranging from 1:57 to 1:1031, tested using a highly-sensitive, luciferase-based assay, received intravenous administration of an AAV5 vector (AAV5-hFIX). Within each dose group, successful and comparable transduction was achieved, independent of the level of pre-existing anti-AAV5 NABs. The data show that anti-AAV5 NAB titers of up to at least 1:1031 have no impact on the transduction of the liver by AAV5 in non-human primates.

Low levels of NABs have been reported to block liver transduction for other vector serotypes. This finding was not seen in the present study with AAV5 in non-human primates, where transduction was not impacted by moderate levels of NABs. Titers of NABs against AAV5 in the general population are generally characterized as low, suggesting that a greater portion of patients can be effectively treated with AAV5 gene therapy, compared to other AAV-based gene therapies. 

“This important data suggests that patients with pre-existing anti-AAV5 NABs may be able to be successfully treated with AAV5 gene therapies, such as our product candidates AMT-060 in hemophilia B and AMT-130 in Huntington’s disease,” stated Matthew Kapusta, chief executive officer at uniQure. “This development has the potential to significantly expand the applicability of AAV5 gene therapies to nearly all patients, regardless of pre-existing antibodies. In addition, AAV5 also appears to have a more favorable immunogenicity profile, with no immune responses detected across two clinical studies involving intravenous administration to 18 patients.  We believe these factors make AAV5 a highly differentiated, best-in-class vector with the potential to more effectively and safely deliver gene therapies to a greater portion of patients in need of treatment.”

Investor/Analyst Breakfast and Webcast on Friday, May 12, 2017

An investor and analyst breakfast meeting will be held on Friday, May 12 at 7:00 a.m.  Senior members of uniQure's research and development team will discuss the results of this study along with the four additional abstracts that are being presented at ASGCT.  The meeting will be webcast live along with slides and can be accessed by visiting the investor relations section of the Company's website at www.uniQure.com. 

Date and Time:  Friday, May 12 at 7:00 a.m. EDT
Location: Omni Shoreham Hotel, The Congressional Room, 2500 Calvert Street NW, Washington, D.C.
The Omni Shoreham hotel is located directly across from the conference venue.

To request attendance at the meeting, please RSVP to This email address is being protected from spambots. You need JavaScript enabled to view it. as space is limited.

About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and cardiovascular diseases. www.uniQure.com

SOURCE: uniQure

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