2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 7 areas
1
News (30d)
National Center for Advancing Translational Sciences, National Institutes of Health is a company with 2 orphan drug designations across 2 rare diseases. gene therapy candidates in 1 disease. 1 news article 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 |
|---|---|---|
| mucopolysaccharidosis | adeno-associated virus 8 vector expressing functional human cDNA encoding the N-acetylgalactosamine-6-sulfate sulfatase | Des.TrialAppr. |
| mucosulfatidosis | recombinant serotype 9 adeno-associated virus encoding codon optimized human sulfatase modifying factor 1 | Des.TrialAppr. |
100% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/2
candidate diseases
1
avg importance: 40
0
affecting portfolio
100% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/2
candidate diseases
1
avg importance: 40
0
affecting portfolio