Clinical trial to test gene therapy treatment for Haemophilia B

GMO Register Reference: G0498-01

Reference: B/IE/13/01

On 3 July 2013, the Environmental Protection Agency (EPA) granted consent to The Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia, USA, to carry out a clinical trial in patients suffering from severe Haemophilia B (Factor IX deficiency).  The clinical trial will involve the administraation of a Genetically Modified Organism (GMO).  The GMO is a recombinant hybrid Adeno-Associated Virus (AAV) which has been engineered to express human coagulation Factor IX (hFIX) for the treatment of patients with severe Haemophilia B (Factor IX deficiency). The GMO is called or abbreviated to AAV8-hFIX19.

Location and Duration

The clinical trial will be carried out in the National Centre for Hereditary Coagulation Disorders, St James's Hospital, James's Street, Dublin 8, during the period 3 July 2013 to 30 September 2015.  Consent was granted, subject to conditions.


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 (performance of clinical 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 G0498-01.  The Register provides significant details about the trial.  The relevant entry is updated as details become available.