Official FDA announcements or press releases

Kendric Cromer Receives First Sickle Cell Gene Therapy; CRISPR Shows Safety, Efficacy in Blindness

May 6, 2024, 02:00 PM

Recent advancements in gene therapy have shown significant progress in treating various inherited diseases. Kendric Cromer, a 12-year-old boy from the Washington, D.C. area, has become the first person to receive a newly approved gene therapy for sickle cell disease, expressing his desire to be cured. Clinical trials have demonstrated the efficacy of CRISPR gene editing in treating inherited forms of blindness, with improvements in vision for several patients and showing safety and early evidence of efficacy. Additionally, gene therapies are also being developed for other conditions, such as skeletal defects and inherited retinal diseases, showing early signs of effectiveness and safety.