What GenSight Biologics’ French CUA decision means for LHON gene therapy
France grants compassionate use access to GenSight Biologics’ LUMEVOQ for LHON. Explore what this decision changes and what risks remain.
Waskyra approved for Wiskott-Aldrich syndrome: What it means for stem cell alternatives
Fondazione Telethon has received regulatory approval from the United States Food and Drug Administration for Waskyra (etuvetidigene autotemcel), an ex vivo gene therapy designed to treat Wiskott-Aldrich syndrome, a rare X-linked primary immunodeficiency. This approval makes Waskyra the first gene therapy for this condition cleared for use in the United States and solidifies Fondazione Telethon’s […]