1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 6 areas
0
News (30d)
Quiet
UNeMed Corporation 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, 6B (Kufs type) | scAAV9.MeCP2.hCLN3, a self-complementary AAV serotype 9 expressing human CLN3 (hCLN3) | Des.TrialAppr. |
| neuronal ceroid lipofuscinosis 3 | scAAV9.MeCP2.hCLN3, a self-complementary AAV serotype 9 expressing human CLN3 (hCLN3) | Des.TrialAppr. |
| progressive myoclonic epilepsy type 3 | scAAV9.MeCP2.hCLN3, a self-complementary AAV serotype 9 expressing human CLN3 (hCLN3) | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio