1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 5 areas
0
News (30d)
Quiet
Ascend Biopharmaceuticals Ltd is a company with 1 orphan drug designation across 3 rare diseases. gene therapy candidates in 1 disease.
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 |
|---|---|---|
| Diamond-Blackfan anemia 1 | Recombinant adenovirus carrying the cDNA coding sequence for the human interferon gamma gene | Des.TrialAppr. |
| thyroid cancer, nonmedullary, 4 | Recombinant adenovirus carrying the cDNA coding sequence for the human interferon gamma gene | Des.TrialAppr. |
| xeroderma pigmentosum group B | Recombinant adenovirus carrying the cDNA coding sequence for the human interferon gamma gene | Des.TrialAppr. |
100% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
100% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/3
candidate diseases
0
avg importance: 0
0
affecting portfolio