5
Orphan Designations
0 approved, 5 designated
0
FDA Approvals
0
Active Trials
4
Rare Diseases
across 5 areas
0
News (30d)
Quiet
HemaCell Biotechnology Inc. is a company with 5 orphan drug designations across 4 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 |
|---|---|---|
| Bernard-Soulier syndrome | induced pluripotent stem cell (iPSC)-derived platelet injectionplatelets produced in vitro from proplatelets derived from peripheral bloodumbilical cord blood derived megakaryocyte injection | Des.TrialAppr. |
| Horner syndrome | platelets produced in vitro from proplatelets derived from peripheral blood | Des.TrialAppr. |
| congenital Horner syndrome | platelets produced in vitro from proplatelets derived from peripheral blood | Des.TrialAppr. |
| congenital amegakaryocytic thrombocytopenia | platelets produced in vitro from proplatelets derived from peripheral bloodmegatemlocel | Des.TrialAppr. |
100% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio
100% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio