3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
5
Rare Diseases
across 7 areas
0
News (30d)
Quiet
Ronald Reagan UCLA Medical Center is a company with 3 orphan drug designations across 5 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 |
|---|---|---|
| AGAT deficiency | adeno-associated vector expressing human codon-optimized arginase 1 under a liver-specific promoter | Des.TrialAppr. |
| arginase deficiency | adeno-associated vector expressing human codon-optimized arginase 1 under a liver-specific promoter | Des.TrialAppr. |
| creatine transporter deficiency | adeno-associated vector-based gene therapy expressing codon-optimized human SLC6A8 | Des.TrialAppr. |
| guanidinoacetate methyltransferase deficiency | adeno-associated vector expressing human codon-optimized guanidinoacetate N-methyltransferase under a ubiquitous-expressing promoter | Des.TrialAppr. |
| hyperlysinemia | adeno-associated vector expressing human codon-optimized arginase 1 under a liver-specific promoter | Des.TrialAppr. |
100% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio
100% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio