1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
1
Active Trials
142 recruiting
2
Rare Diseases
across 5 areas
0
News (30d)
Quiet
Genecombio Ltd. is a company with 1 orphan drug designation across 2 rare diseases. Active clinical trials in 1 indication.
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 |
|---|---|---|
| Rett syndrome | Self-complementary Adeno-Associated Virus 9 containing human methyl-CpG binding protein 2 | Des.TrialAppr. |
| spinal muscular atrophy | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio