1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 8 areas
0
News (30d)
Quiet
Shanghai Unicar-Therapy Bio-medicine Technology Co.,Ltd is a company with 1 orphan drug designation across 3 rare diseases.
Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.
Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA
| Disease | Drug(s) | Stage |
|---|---|---|
| acute lymphoblastic leukemia | Autologous anti-CD19 Chimeric Antigen Receptors (CAR)-T cells expressing a short hairpin RNA (shRNA) against interleukin-6 (IL-6) (also termed ssCART-19 cells) | Des.TrialAppr. |
| adult acute lymphoblastic leukemia | Autologous anti-CD19 Chimeric Antigen Receptors (CAR)-T cells expressing a short hairpin RNA (shRNA) against interleukin-6 (IL-6) (also termed ssCART-19 cells) | Des.TrialAppr. |
| childhood acute lymphoblastic leukemia | Autologous anti-CD19 Chimeric Antigen Receptors (CAR)-T cells expressing a short hairpin RNA (shRNA) against interleukin-6 (IL-6) (also termed ssCART-19 cells) | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio