Please login to the form below

Not currently logged in
Email:
Password:

Severe immune deficiency trial approved

A clinical trial of a gene therapy for the rare immunodeficiency, Wiskott-Aldrich syndrome, has been approved in France and the UK

A clinical trial of a gene therapy for the rare immunodeficiency, Wiskott-Aldrich syndrome (WAS), has been approved in France and the UK. It will be sponsored by Généthon, the not-for-profit biotherapy laboratory operated by the French Muscular Dystrophy Association (AFM), with funding from France's annual Telethon. 

The French and British health authorities have authorised the phase I/II trial, which will be conducted both at Necker Children's Hospital, Paris, and at London's Great Ormond Street Hospital.   

WAS is a severe, X-linked immunodeficiency which results in haemorrhage, recurrent infection and eczema. It is almost always fatal in affected children. Although a bone marrow transplant can re-establish the patient's immune system, this requires a compatible donor and carries a risk of failure. Gene therapy is a major source of hope, therefore.   

This trial is the result of research initiated in 2002 in the Généthon laboratory. The researchers have developed an ex-vivo approach that uses an HIV-derived lentiviral vector to transfer genes into autologous CD34+ haematopoietic stem cells from the patient.  

"This trial is enabling the Généthon laboratory to demonstrate its world-class expertise in the production of gene-based medicines and the preparation and implementation of clinical trials." stated AFM and Généthon president, Laurence Tiennot-Herment. "It confirms Généthon's ability to become a major international player in demonstrating the efficacy of gene therapy for rare diseases."  

Généthon is a leading centre for preclinical and clinical research and development in the field of gene therapy. It has a biomanufacturing platform for clinical-grade vectors and is involved in building the world's largest facility for pre-industrial pilot production at Evry, France, that is scheduled to open in early 2011.     

24th February 2010

Share

Featured jobs

Subscribe to our email news alerts

PMHub

Add my company
Page & Page

Page & Page is a new kind of marketing communications consultancy. We change beliefs and behaviours to improve health outcomes...

Latest intelligence

What pharma marketers can learn from behavioural science
Pharma behavioural science and traditional emotional marketing create a powerful mix of techniques that have impact on real lives....
NEW EU DATA PROTECTION REGULATION POISED TO CHANGE DATA PRIVACY LANDSCAPE
NEW EU DATA PROTECTION REGULATION POISED TO CHANGE DATA PRIVACY LANDSCAPE The bold digital clock featured on the EUGDPR (EU General Data Protection Regulation)1 website indicates – to the second...
Health knowledge: Why is it so important?
Health knowledge plays an important role in population health, but by itself is rarely enough to prompt a change in the behaviours that cause the risks....

Infographics