Sickle cell anemia: AFM-Telethon supports gene therapy trial
Thanks to the financial support of the AFM-Téléthon, a decisive step has just been taken in the fight against sickle cell disease. In collaboration with AP-HP, Institut Imagine and SK pharmteco, the AFM- Telethon will enable the production of lentiviral vectors for the development of a gene therapy clinical trial. This represents hope for the 5 million patients worldwide affected by this incurable blood disease.
A project made possible by the AFM- Telethon
The AFM- Telethon is funding the final pre-clinical phases and technical production of lentiviral vectors, which are necessary to achieve this key milestone before the launch of a clinical trial.
“We are proud to support this key validation phase for this gene therapy drug candidate for sickle cell disease, a rare and extremely serious blood disorder that affects thousands of people in France and millions worldwide, and to enable the researchers we have supported for many years, thanks in particular to the Telethon, to take the final step towards treating these patients” said Laurence Tiennot-Herment, President of AFM-Telethon
This commitment is in line with AFM- Telethon's general interest strategy to accelerate the development of innovative treatments for rare genetic diseases. By supporting this project, the association is contributing to a decisive step towards the clinical application of gene therapy for sickle cell disease.
A promising gene therapy in development
This project is based on an approach to ex vivo genetic modification of patients' hematopoietic stem cells using a lentiviral vector, newly developed by Annarita Miccio and Marina Cavazzana's labs at Institut Imagine, following earlier work carried out in collaboration with Mario Amendola of Genethon, the AFM-Telethon lab.
The project is based on SK pharmteco's LentiSure™ platform and analytical tests carried out in France. This production represents a strategic step forward to ensure that the research conducted at Institut Imagine can be translated into clinical applications.
Why is this trial crucial?
Sickle cell disease, a genetic disorder affecting red blood cells, affects thousands of people in France and nearly 5 million worldwide. It causes severe multi-organ pain, anemia and increased susceptibility to infection. To date, there is no cure for this chronic disease.
The production of lentiviral vectors represents a major step forward in bringing this gene therapy to the clinical stage.