Forecast horizon — calibration-scored at resolution.
By 2028, exa-cel or a comparable ex vivo gene-edited therapy will have treated >500 SCD patients globally with a durable VOC-free rate >90% at 3 years.
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Evidence stream
1 event · 0 snapshots
Registry data
Apr 18, 2026
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Source publication
A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
Semantically related
Nearest claims in the expert-corpus vector space. Ordered by cosine distance — lower is closer.
0.1449
By 2028, at least two sickle cell gene-therapy products will compete head-to-head on real-world VOC-free survival and cost-effectiveness.
0.1501
By 2028, one-time AAV gene therapy for severe hemophilia B will show ≥5-year FIX expression durability in >70% of treated patients.
0.1929
The CLIMB-121 (exa-cel, sickle cell) trial's stated primary conclusion — CRISPR-Cas9 BCL11A editing (exa-cel) eliminates vaso-occlusive crises in severe sickle cell disease over multi-year follow-up. — replicates in independent cohorts.
0.1939
The HGB-206 (lovo-cel) trial's stated primary conclusion — Lentiviral β-globin gene therapy reduces or eliminates VOC events in severe sickle cell disease. — replicates in independent cohorts.
0.1984
Recent follow-up analyses of CLIMB-121 (exa-cel, sickle cell) are confirming the original effect size in real-world data.
0.2197
Recent follow-up analyses of HOPE-B (Hemgenix / etranacogene) are confirming the original effect size in real-world data.