1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
4
Rare Diseases
across 5 areas
0
News (30d)
Quiet
Elixirgen Therapeutics, Inc. is a company with 1 orphan drug designation 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 |
|---|---|---|
| ACD-related long telomere syndrome | autologous CD34+ cells contacted ex vivo with EXG-001 Sendai virus vector | Des.TrialAppr. |
| ACD-related short telomere syndrome | autologous CD34+ cells contacted ex vivo with EXG-001 Sendai virus vector | Des.TrialAppr. |
| ACD-related telomere biology disorder | autologous CD34+ cells contacted ex vivo with EXG-001 Sendai virus vector | Des.TrialAppr. |
| dyskeratosis congenita | autologous CD34+ cells contacted ex vivo with EXG-001 Sendai virus vector | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio