News

NET4CGD Newsletter 2017 : Net4CGD Consortium - Gene Therapy for C-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 receive autologous CD34+ blood stem cells transduced with the G1XCGD lentiviral vector.

In Europe, two clinical studies are ongoing: G1XCGD.01 study (NCT01855685) and G1XCGD.02 study (NCT02757911). Three patients have already been treated and results are encouraging.

Notably, a study is also ongoing in USA (NCT02234934) and three patient benefitted from the same therapeutic approach with good tolerance and clinical improvement.

For more information, please refer to the Net4CGD Newsletter 2017 that can be found here.

Participation of NET4CGD at the 3rd International Rare Diseases Research Consortium Conference - Paris, France (February 8-9, 2017)

Net4CGD poster presentation at the 3rd International Rare Diseases Research Consortium Conference - Conference Center University Pierre et Marie Curie - Paris, France (February 8-9, 2017)

The International Rare Diseases Research Consortium (IRDiRC) brings together members that share common goals and principles and have agreed to work in a collaborative manner within a multinational consortium. IRDiRC teams up researchers and organizations investing in rare diseases research to achieve two main objectives by the year 2020, namely to deliver 200 new therapies for rare diseases and the means to diagnose most rare diseases.

For further information, please click here (abstract and poster).

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.