1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Tern Therapeutics LLC is a company with 1 orphan drug designation across 3 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 |
|---|---|---|
| ceroid lipofuscinosis, neuronal, 4 (Kufs type) | non-replicating, recombinant adeno-associated virus (AAV) serotype 9 (AAV9) vector containing an hCLN2 expression cassette encoding for the soluble lysosomal enzyme tripeptidyl peptidase I (TPP1) | Des.TrialAppr. |
| ceroid lipofuscinosis, neuronal, 6B (Kufs type) | non-replicating, recombinant adeno-associated virus (AAV) serotype 9 (AAV9) vector containing an hCLN2 expression cassette encoding for the soluble lysosomal enzyme tripeptidyl peptidase I (TPP1) | Des.TrialAppr. |
| neuronal ceroid lipofuscinosis 2 | non-replicating, recombinant adeno-associated virus (AAV) serotype 9 (AAV9) vector containing an hCLN2 expression cassette encoding for the soluble lysosomal enzyme tripeptidyl peptidase I (TPP1) | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
2
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
2
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio