Biotech Breakthroughs
↑ Positive
CRISPR Gene Therapy Shows Complete Remission in Sickle Cell Trial
Nature
by Heidi Ledford
February 11, 2026 · 05:59
22 hours ago
Summary
Phase 3 clinical results show 100% of treated patients achieved sustained remission from sickle cell disease symptoms.
Results from a pivotal Phase 3 clinical trial published today in Nature Medicine demonstrate that a one-time CRISPR-based gene therapy achieved complete and sustained remission in all 45 patients treated for severe sickle cell disease.
Patients who received the therapy, designated CTX001, showed elimination of vaso-occlusive crises and no longer required blood transfusions, with benefits sustained for more than 24 months in the longest-followed patients.
The results are expected to accelerate the already-submitted regulatory applications in the US, EU, and UK, with FDA approval potentially coming within months.
Patients who received the therapy, designated CTX001, showed elimination of vaso-occlusive crises and no longer required blood transfusions, with benefits sustained for more than 24 months in the longest-followed patients.
The results are expected to accelerate the already-submitted regulatory applications in the US, EU, and UK, with FDA approval potentially coming within months.
Analysis
Relevance Score
Critical
Sentiment
↑ +0.88
Positive
Source Reliability
9.5
/ 10
High
Source
Nature
nature.com
Independent
UK