2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
4
Rare Diseases
across 7 areas
0
News (30d)
Quiet
Rznomics is a company with 2 orphan drug designations across 4 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 |
|---|---|---|
| adult hepatocellular carcinoma | replication-incompetent human mastadenovirus C serotype 5, with E1 and partial E3 deletions that encodes a hTERT RNA-targeting trans-splicing ribozyme with HSVtk transgene | Des.TrialAppr. |
| hepatocellular carcinoma | replication-incompetent human mastadenovirus C serotype 5, with E1 and partial E3 deletions that encodes a hTERT RNA-targeting trans-splicing ribozyme with HSVtk transgene | Des.TrialAppr. |
| malignant glioma | a replication-incompetent adenoviral therapy that expresses a ribozyme (RNA enzyme) that is capable of cleaving cancer-specific human telomerase reverse transcriptase (hTERT) RNA, and trans-ligating herpes simplex virus thymidine kinase (HSVtk) onto the hTERT RNA | Des.TrialAppr. |
| pediatric hepatocellular carcinoma | replication-incompetent human mastadenovirus C serotype 5, with E1 and partial E3 deletions that encodes a hTERT RNA-targeting trans-splicing ribozyme with HSVtk transgene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
23
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
23
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio