Infusion Of Autologous Efs-Ada Lv Cd34+ Cells
Severe Combined Immunodeficiency, Autosomal Recessive, T Cell-Negative, B Cell-Negative, NK Cell-Negative, Due To Adenosine Deaminase Deficiency
Great Ormond Street Hospital for Children NHS Foundation Trustgene_therapyPhase 1/2Dormant
CCC
52/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 37/ 100
- Competitive Position
- 95/ 100
Competitive context
1 Active assets in the Severe Combined Immunodeficiency, Autosomal Recessive, T Cell-Negative, B Cell-Negative, NK Cell-Negative, Due To Adenosine Deaminase Deficiency cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Adagen Leadiant Biosciences, Inc. | Phase 3 | BB |
Elapegademase Leadiant Biosciences, Inc. | Phase 3 | BB |
Peg-Ada Great Ormond Street Hospital for Children NHS Foundation Trust | Phase 1/2 | CCC |
Busulfanum Great Ormond Street Hospital for Children NHS Foundation Trust | Phase 1/2 | CCC |
Cyclosporin A Fred Hutchinson Cancer Center | Phase 1 | CC |
Simoladagene Autotemcel Lentiviral Vector University of California, Los Angeles | Unknown | D |
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