Exa-cel
PreclinicalCell therapy
Vertex PharmaceuticalsExa-cel is a cell therapy that works as a BCL11A Gene Editing (CRISPR).[2] It is being investigated across 2 indications, with its most advanced program in Preclinical.[1]
Exa-cel is one of 67 gene therapy programs targeting Sickle Cell Disease. It is currently in Preclinical.
2
Indications
1
Total Trials
0
Active Trials
66
Competitors
Pipeline
Status by Indication[3][1]
Competition
Competitive Landscape
272 competitors in Sickle Cell Disease
#DrugCompanyPhaseTrials
1Adakveo
NovartisPhase 3
Exjade
NovartisPhase 3
Brilinta
AstraZenecaPhase 3
Campath
GGenzyme, a Sanofi Company
Phase 3
Ritalin
NovartisPhase 3
Cysteine Hydrochloride
NovartisPhase 3
Eliquis
Bristol-Myers SquibbPhase 3
Oxbryta
GGlobal Blood Therapeutics
Phase 3
Aqvesme
AAgios Pharmaceuticals, Inc.
Phase 3
Effient
CCosette Pharmaceuticals, Inc.
Phase 3
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Clinical
Trials by Indication[1]
Sources
References
- [1]
- [2]
- [3]Open Targets Platform — Drug–disease associations for Exa-cel. EMBL-EBI & Wellcome Sanger Institute.
- [4]
- [5]
Details
Drug Info
Quick Facts
- Mechanism of Action
- BCL11A Gene Editing (CRISPR)[2]
- Modality
- Cell therapy[2]
- Highest Phase
- Preclinical[1]
- Company