NET4CGD Newsletter 2016 : Focus on the Gene Therapy Studies for X-CGD

The Net4CGD European Consortium is focused on the clinical development of Gene Therapy as a new Orphan Drug for patients with the X-linked form of Chronic Granulomatous Disease (X-CGD). Patients will receive autologous CD34+ blood stem cells transduced with the G1XCGD lentiviral vector. In 2016, Phase I/II Clinical Trials are ongoing in several clinical centers in Europe. For more information, please refer to the Net4CGD Newsletter 2016 that can be found here.

NET4CGD News  : First CGD patient to be treated - Dana-Farber/Boston Children’s Gene Therapy Programme.

On December 18, 2015, 22-year-old Brenden Whittaker became the first CGD patient to be treated with gene therapy for chronic granulomatous disease (CGD) - Dana-Farber/Boston Children’s Gene Therapy Programme.

Further information can be found on the Boston Children's Hospital website by clicking here.

XCGD Multicentric European Study - Q&A

As part of the NET4CGD dissemination strategy and in order to provide information to patients, a Q&A titled "New gene therapy trial for X-CGD" has been created that can be found on the NET4CGD website under the "NET4CGD Project" tab.

NET4CGD Results In Brief

As the European Commission's primary public repository and portal to disseminate information on all EU-funded research projects and their results in the broadest sense, CORDIS has published a summary on the NET4CGD project. This project summary  is available in several languages (French, English, German, Spanish, Italian and Polish) that can be found on the NET4CGD website under the "NET4CGD Project" tab.

Net4CGD new scientific article to be published in Journal of Allergy and Clinical Immunology - 2016

Net4CGD scientists in Frankfurt, Paris and London report that hyperinflammation in CGD leads to impairment of hematopoietic stem cell functions in the prestigious Journal of Allergy and Clinical Immunology. Persistent chronic inflammation in CGD is associated with hematopoietic proliferative stress leading to a decrease in the functional activity of hematopoietic stem cells. Proactive treatment of CGD patients with anti-inflammatory drugs might therefore reduce chronic sterile inflammation in CGD and in addition could have an impact on the quantity and quality of hematopoietic stem cells available for autologous gene therapy.

The article will be published in 2016.