Aim and objectives of Net4CGD

The aim of this project is the clinical development of an orphan drug that can rapidly become a new treatment option to X-CGD patients. This aim will be achieved through these objectives :

  • Conducting phase I/II trial in eligible X-CGD patients, with lentiviral vector gene-modified autologous HSC to test the safety and efficacy of the technology,
  • Rapidly accruing high-quality data  by conducting trials in 4 expert Institutions, testing the same lentiviral vector product with harmonized procedures and protocols to facilitate product registration,
  • Assessing functional innate immune restoration obtained by hematopoietic gene therapy,
  • Obtaining large-scale “omics” and vector genome stability analysis, including bioinformatic data mining, dynamic sequence data storage and whole integrome sequencing, to evaluate vector safety in man.