Gene therapy trial launched for sickle cell disease
For the 7 million people living with sickle cell disease worldwide, the authorisation granted by the French National Agency for Medicines and Health Products Safety (ANSM) to launch the DREPAMIR phase I/II clinical trial represents real hope. Supported by AFM-Téléthon and led by Professor Marina Cavazzana and Dr Anne Galy (ART-TG), the aim of this trial is to provide a long-term therapeutic solution for affected patients.
This project is the result of close collaboration between Institut Imagine, the scientific driver of the programme, AP-HP, sponsor of the trial, and AFM-Téléthon, a key supporter and funder of the project. This commitment is fully aligned with AFM-Téléthon’s public-interest strategy to accelerate the development of innovative treatments for rare genetic diseases.
Science at the heart of innovation
DREPAMIR is the name of this clinical trial, which consists of delivering a therapeutic gene combined with a small RNA designed to inhibit the production of toxic haemoglobin.
This ex vivo gene therapy strategy involves collecting blood stem cells from patients, modifying them in the laboratory using a bifunctional lentiviral vector designed by Annarita Miccio’s team in collaboration with Mario Amendola from the Généthon laboratory, and then reinfusing the corrected cells into patients. The objective is to restore functional haemoglobin production and reduce severe complications associated with sickle cell disease.
Anne Galy’s perspective
Anne Galy, Inserm research director and expert in gene therapy for blood disorders, presents the innovative approaches being developed for sickle cell disease, including the DREPAMIR trial. (In French)