4
Orphan Designations
0 approved, 4 designated
0
FDA Approvals
0
Active Trials
21 recruiting
2
Rare Diseases
across 7 areas
0
News (30d)
Quiet
NeuroGT, Inc. is a company with 4 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 |
|---|---|---|
| mucopolysaccharidosis | Adeno-associated virus vector expressing human iduronate-2-sulfatase | Des.TrialAppr. |
| mucopolysaccharidosis type 1 | Self-complementary adeno-associated virus serotype 9 vector harboring a miniaturized codon-optimized human IDUA gene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio