2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
27
Active Trials
200 recruiting
3
Rare Diseases
across 34 areas
0
News (30d)
Quiet
University of Texas Southwestern Medical Center is a company with 2 orphan drug designations across 3 rare diseases. Active clinical trials in 27 indications. gene therapy candidates in 1 disease. 29 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 |
|---|---|---|
| Alzheimer disease | - | Des.TrialAppr. |
| Leigh syndrome | TGTX-102, an adeno-associated virus serotype 9 (AAV9) vector with engineered transgene encoding the human SURF1 protein. | Des.TrialAppr. |
| Lichtenstein-Knorr syndrome | adeno-associated viral vector serotype 9 encoding a codon-optimized human SLC6A1 transgene | Des.TrialAppr. |
| Parkinson disease | - | Des.TrialAppr. |
| SLC26A2-related skeletal dysplasia | adeno-associated viral vector serotype 9 encoding a codon-optimized human SLC6A1 transgene | Des.TrialAppr. |
| amyloidosis | - | Des.TrialAppr. |
| brain neoplasm | - | Des.TrialAppr. |
| cancer | - | Des.TrialAppr. |
| cardiovascular disorder | - | Des.TrialAppr. |
| craniosynostosis | - | Des.TrialAppr. |
| cystic fibrosis | - | Des.TrialAppr. |
| diabetes mellitus | - | Des.TrialAppr. |
| dystonic disorder | - | Des.TrialAppr. |
| epilepsy | - | Des.TrialAppr. |
| hemophilia A | - | Des.TrialAppr. |
| lupus erythematosus | - | Des.TrialAppr. |
| multiple sclerosis | - | Des.TrialAppr. |
| multiple system atrophy | - | Des.TrialAppr. |
| sarcoidosis | - | Des.TrialAppr. |
| sarcoma | - | Des.TrialAppr. |
| sickle cell disease | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
102
overlap in 2+ diseases
1/21
candidate diseases
0
avg importance: 0
29
affecting portfolio
0% of portfolio targets high unmet need diseases
102
overlap in 2+ diseases
1/21
candidate diseases
0
avg importance: 0
29
affecting portfolio