Karyna Lutsyk, Tristan Gicquel, Lucas Cortial, Sylvain Forget, Serge Braun, Pierre-Olivier Boyer, Vincent Laugel, Olivier Blin
Rare diseases are chronic, serious and generally genetic conditions affecting a small number of people, and their therapeutic management is a real challenge. They represent a considerable burden for patients, caregivers and society alike. Compared with existing symptomatic treatments, gene therapies represent a promising new approach aimed at treating these diseases by replacing a defective gene, or by abolishing or reviving a gene-derived function. France is considered one of the leading countries in the research and development of drugs for rare diseases, yet the position of French public and private stakeholders in the research and development of gene therapies for rare diseases at global and European level remains unclear...
February 15, 2024: Thérapie