Clinical Trial to test gene therapy treatment for Haemophilia B

On 21 November 2018, the Environmental Protection Agency (EPA) granted consent to uniQure Biopharma BV, Paasheuvelweg 25A, 1105 BP Amsterdam, The Netherlands, for the deliberate release of a Genetically Modified Organism (GMO) during a clinical trial under Part B of the GMO (Deliberate Release) Regulations S.I. No 500 of 2003.  The GMO is a recombinant serotype 5 adeno-associated viral vector containing the Padua variant of a codon optimised human factor IX gene (AAV5-hFIXco-Padua).  The GMO is also known as AMT-061.  The clinical trial will assess the safety and efficacy of the administration of a single dose of AMT-061 to adult patients with severe or moderately severe haemophilia B who meet study inclusion criteria.

Location and Duration

The clinical trial will be carried out in St James's Hospital, James's Street, Dublin 8, from the date of grand of the consent conditions to 31st December 2021.  Consent was granted, subject to conditions.

Legislation

Any person or organisation proposing to carry out a deliberate release of a genetically modified organism (GMO) for purposes other than placing on the market (for purposes of research or performing trials) must comply with the legislative requirements set out under Part II of the GMO (Deliberate Release) Regulations S.I. No 500 of 2003.

Relevant Documentation

Requests for further information and replies

Consent Conditions

Inspector's Report

Board Minute Extract

GMO Register Entry  

GMO Register Entry

The proposed clinical trial has been assigned GMO Register No G0667-01.