Busulfanum

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 TrustSmall moleculePhase 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
Infusion Of Autologous Efs-Ada Lv Cd34+ Cells 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