3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Vor Biopharma is a company with 3 orphan drug designations across 2 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 |
|---|---|---|
| acute myeloid leukemia | lentiviral-transduced CD33-directed CAR-T cell therapyallogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell (HSPC) therapy product lacking the CD33 protein | Des.TrialAppr. |
| myasthenia gravis | telitacicept | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
5
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
5
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio