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 1 indication, with its most advanced program in Preclinical.[1]
Exa-cel is one of 17 gene therapy programs targeting Beta-Thalassemia. It is currently in Preclinical.
1
Indications
2
Total Trials
0
Active Trials
16
Competitors
Pipeline
Status by Indication[3][1]
Indication
Competition
Competitive Landscape
22 competitors in Beta-Thalassemia
#DrugCompanyPhaseTrials
1Exjade
NovartisApproved
Thalomid
Bristol-Myers SquibbApproved
Droxia
TTheravia
Approved
Desferal
Jazz PharmaceuticalsApproved
Glucophage
EMD SeronoApproved
Viagra
VViatris Specialty LLC
Approved
Vidaza
KKaruna Therapeutics, Inc., a Bristol Myers Squibb company
Approved
Ferriprox
Chiesi Farmaceutici S.p.A.Approved
CTX001
Vertex PharmaceuticalsPhase 3
ACE-536
Bristol-Myers SquibbPhase 2
Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.
Clinical
Trials by Indication[1]
Sickle Cell Disease2 trials
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