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Exa-cel — Competitive Intelligence Brief

Exa-cel (Exa-cel) competitive landscape: 0 comparators, recent regulatory actions, upcoming PDUFA, patent timeline. Class: CRISPR gene-editing therapy. Area: Hematology/Rare Genetic Disorders.

phase 3 CRISPR gene-editing therapy BCL11A gene Hematology/Rare Genetic Disorders Biologic Live · refreshed every 30 min

Target snapshot

Exa-cel (Exa-cel) — Vertex Pharmaceuticals Incorporated. Exa-cel is an in vivo CRISPR gene-editing therapy that modifies the BCL11A gene to reactivate fetal hemoglobin production in patients with sickle cell disease or beta-thalassemia.

Comparator set

Auto-detected by same drug class / molecular target / therapeutic area, ranked by phase + recency. Override with ?vs=slug1,slug2,….

DrugGenericSponsorPhaseClassTargetApproval / PDUFA
Exa-cel TARGET Exa-cel Vertex Pharmaceuticals Incorporated phase 3 CRISPR gene-editing therapy BCL11A gene

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this set.

Patent timeline (forward window)

No upcoming patent expiries for this set.

Sponsor landscape (CRISPR gene-editing therapy class)

  1. Vertex Pharmaceuticals Incorporated · 1 drug in this class

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Cite this brief

Drug Landscape (2026). Exa-cel — Competitive Intelligence Brief. https://druglandscape.com/ci/exa-cel. Accessed 2026-05-13.

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