1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 7 areas
0
News (30d)
Quiet
Blue Gen Therapeutics Foundation is a company with 1 orphan drug designation across 3 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 |
|---|---|---|
| Acrodysostosis 1 with or without hormone resistance | adeno-associated virus vector carrying complementary deoxyribonucleic acid encoding the human long-wave-sensitive opsin 1 (AAV.7m8-L-opsin) | Des.TrialAppr. |
| blue cone monochromacy | adeno-associated virus vector carrying complementary deoxyribonucleic acid encoding the human long-wave-sensitive opsin 1 (AAV.7m8-L-opsin) | Des.TrialAppr. |
| microcephalic osteodysplastic dysplasia, Saul-Wilson type | adeno-associated virus vector carrying complementary deoxyribonucleic acid encoding the human long-wave-sensitive opsin 1 (AAV.7m8-L-opsin) | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio