2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 2 areas
0
News (30d)
Quiet
Holostem S.r.l. is a company with 2 orphan drug designations across 2 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 |
|---|---|---|
| epidermolysis bullosa | epidermal stem cells genetically modified with a gamma-retroviral (rv) vector expressing the full-length LAMB3 cDNA | Des.TrialAppr. |
| limbal stem cell deficiency | ex vivo expanded autologous human corneal epithelial cells containing stem cells | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio