4
Orphan Designations
0 approved, 4 designated
0
FDA Approvals
13
Active Trials
200 recruiting
14
Rare Diseases
across 24 areas
0
News (30d)
Quiet
Nationwide Children's Hospital is a company with 4 orphan drug designations across 14 rare diseases. Active clinical trials in 13 indications. gene therapy candidates in 1 disease. 24 active policy bills affect their portfolio.
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 |
|---|---|---|
| Duchenne muscular dystrophy | non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNArecombinant adeno-associated virus serotype rhesus 74 (rh74) expressing the human CT GalNac transferase (GALGT2) gene, also called the Sda or Cad GAlNac transferase or b4Galnt2, under control of the muscle creatine kinase (MCK) promoter | Des.TrialAppr. |
| Ewing sarcoma | - | Des.TrialAppr. |
| adult neuronal ceroid lipofuscinosis | Non-replicating recombinant, self-complementary adeno-associated virus serotype 9 containing the human CLN3 gene | Des.TrialAppr. |
| brain neoplasm | - | Des.TrialAppr. |
| cancer | - | Des.TrialAppr. |
| childhood malignant neoplasm | - | Des.TrialAppr. |
| congenital heart disease | - | Des.TrialAppr. |
| juvenile neuronal ceroid lipofuscinosis | Non-replicating recombinant, self-complementary adeno-associated virus serotype 9 containing the human CLN3 gene | Des.TrialAppr. |
| lymphoma | - | Des.TrialAppr. |
| malignant glioma | Human cytomegalovirus (HCMV) internal repeat short 1 (IRS1)-expressing oncolytic Herpes Simplex Virus | Des.TrialAppr. |
| muscular dystrophy | - | Des.TrialAppr. |
| neuronal ceroid lipofuscinosis 3 | Non-replicating recombinant, self-complementary adeno-associated virus serotype 9 containing the human CLN3 gene | Des.TrialAppr. |
| sarcoma | - | Des.TrialAppr. |
| sickle cell disease | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
108
overlap in 2+ diseases
1/14
candidate diseases
0
avg importance: 0
24
affecting portfolio
0% of portfolio targets high unmet need diseases
108
overlap in 2+ diseases
1/14
candidate diseases
0
avg importance: 0
24
affecting portfolio