1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 5 areas
0
News (30d)
Quiet
Choroideremia Research Foundation, Inc. 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 |
|---|---|---|
| choroideremia | aadeno-associated viral vector, serotype 2, containing the human choroideremia gene encoding human Rab escort protein 1 | Des.TrialAppr. |
| choroideremia hypopituitarism | aadeno-associated viral vector, serotype 2, containing the human choroideremia gene encoding human Rab escort protein 1 | Des.TrialAppr. |
| choroideremia-deafness-obesity syndrome | aadeno-associated viral vector, serotype 2, containing the human choroideremia gene encoding human Rab escort protein 1 | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio