2
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
200 recruiting
2
Rare Diseases
across 4 areas
0
News (30d)
Quiet
Targeted Genetics Corporation is a company with 2 orphan drug designations across 2 rare diseases. 1 active policy bill affects their portfolio.
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 |
|---|---|---|
| cystic fibrosis | Adeno-associated viral-based vector cystic fibrosis gene therapy | Des.TrialAppr. |
| ovarian cancer | RGG0853, E1A lipid complex | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
18
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
1
affecting portfolio
0% of portfolio targets high unmet need diseases
18
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
1
affecting portfolio