Science & discoveries / Development in perspective
CRISPR: when gene editing became an approved therapy
A milestone from 2023 explained: what the approval of Casgevy brought, and its limits.
Key takeaway
An approved CRISPR therapy is an important achievement for specific indications. It is not a universal treatment for genetic diseases.
What happened on 8 December 2023
The FDA approved Casgevy for patients aged 12 and older with sickle cell disease and recurrent vaso-occlusive crises. It was the first FDA-approved therapy using CRISPR/Cas9. This article explains that historical milestone rather than presenting it as breaking news.
What is changed
The process uses the patient’s own blood-forming stem cells. They are modified outside the body and returned to increase production of fetal haemoglobin, helping prevent the characteristic sickling of red blood cells.
An achievement involving a complex process
Treatment requires cell collection, preparatory chemotherapy and transplantation. It is not a routine injection and does not promise the same outcome for everyone. Long-term follow-up is needed to assess safety and treatment effects.
Why it matters
For the Journal, this development matters because a research technology became an approved treatment for a defined patient group. EMA documentation provides further European information on Casgevy’s use and safety.
This is information about an international development. It does not establish availability in Albania or describe a service offered by Anna Maria Lab.
Sources
- FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease · 8 dhjetor 2023 ↗U.S. Food and Drug Administration · Accessed 5 September 2026
- Casgevy — European public assessment report ↗European Medicines Agency · Accessed 5 September 2026
An editorial explanation referring to the listed sources. General information; individual interpretation and treatment require clinical assessment.
Updated 5 September 2026. How we prepare our content