1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
130 recruiting
1
Rare Diseases
across 1 areas
0
News (30d)
Quiet
GENASSIST BOSTON LIMITED is a company with 1 orphan drug designation across 1 rare disease. 5 active policy bills affect their portfolio.
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 |
|---|---|---|
| Duchenne muscular dystrophy | cytosine base editing drug containing two adeno-associated virus serotype 9 (AAV9) vectors, ss.AAV9.oTAM and ss.AAV9.hE50-sgRNA | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/1
candidate diseases
0
avg importance: 0
5
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/1
candidate diseases
0
avg importance: 0
5
affecting portfolio