1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
1
Rare Diseases
across 1 areas
0
News (30d)
Quiet
BlackfinBio Limited is a company with 1 orphan drug designation across 1 rare disease. gene therapy candidates in 1 disease.
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 |
|---|---|---|
| hereditary spastic paraplegia 47 | adeno-associated virus serotype 9 gene transfer vector expressing human AP4B1 cDNA | Des.TrialAppr. |
100% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/1
candidate diseases
0
avg importance: 0
0
affecting portfolio
100% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/1
candidate diseases
0
avg importance: 0
0
affecting portfolio