2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
1
Active Trials
200 recruiting
2
Rare Diseases
across 7 areas
0
News (30d)
Quiet
Cellectis S.A. is a company with 2 orphan drug designations across 2 rare diseases. Active clinical trials in 1 indication.
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 lymphoblastic leukemia | alemtuzumaballogeneic non-alloreactive T cells edited with mRNA to disrupt TRAC and CD52 genes and transduced with lentiviral vector expressing a chimeric antigen receptor against CD22 and RQR8 depletion mechanism | Des.TrialAppr. |
| lymphoma | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
9
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
9
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio