6
Orphan Designations
0 approved, 4 designated
0
FDA Approvals
4
Active Trials
200 recruiting
8
Rare Diseases
across 18 areas
2
News (30d)
uniQure Biopharma BV is a company with 6 orphan drug designations across 8 rare diseases. Active clinical trials in 4 indications. gene therapy candidates in 1 disease. 7 active policy bills affect their portfolio. 2 news articles in the past 30 days.
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 |
|---|---|---|
| Duchenne muscular dystrophy | adeno-associated viral vector containing modified U11 snRNA | Des.TrialAppr. |
| Fabry disease | adeno-associated viral vector serotype 5 encoding human alpha-galactosidase A | Des.TrialAppr. |
| Huntington disease | adeno-associated viral vector serotype 5 encoding a microRNA targeted to human huntingtin gene | Des.TrialAppr. |
| amyotrophic lateral sclerosis | adeno-associated virus serotype rh10 encoding artificial anti-SOD1 miRNA | Des.TrialAppr. |
| epilepsy | - | Des.TrialAppr. |
| familial lipoprotein lipase deficiency | Adeno-associated vector expressing the human lipoprotein lipase protein | Des.TrialAppr. |
| hemophilia B | an adeno-associated viral vector containing a codon-optimised human factor IX gene (AAV5-hFIXco) | Des.TrialAppr. |
| multiple sclerosis | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
57
overlap in 2+ diseases
1/8
candidate diseases
2
avg importance: 65
7
affecting portfolio
0% of portfolio targets high unmet need diseases
57
overlap in 2+ diseases
1/8
candidate diseases
2
avg importance: 65
7
affecting portfolio