1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
1
Rare Diseases
across 2 areas
0
News (30d)
Quiet
Neogene Therapeutics, Inc., a member of the AstraZeneca Group is a company with 1 orphan drug designation across 1 rare 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 |
|---|---|---|
| malignant pancreatic neoplasm | autologous T cells genetically engineered to express an HLA-C*08:02-restricted TCR specific to KRAS G12D and a modified dihydrofolate reductase gene (mDHFR) sequence, and to knock out endogenous human TCR alpha and beta chains and the gene encoding the transforming growth factor-beta receptor 2 (TGFBR2) | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/1
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/1
candidate diseases
0
avg importance: 0
0
affecting portfolio