6
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Danon disease | Adeno-associated virus serotype 9 vector containing the human lysosome-associated membrane glycoprotein 2 isoform B transgene (AAV9.LAMP2B) | Orphan Designation | - |
| Fanconi anemia complementation group A | Fancalen (lentiviral vector containing the Fanconi anemia-A (FANCA) gene | Orphan Designation | - |
| Fanconi anemia complementation group C | Fancalen (lentiviral vector containing the Fanconi anemia-A (FANCA) gene | Orphan Designation | - |
| Fanconi anemia complementation group Q | Fancalen (lentiviral vector containing the Fanconi anemia-A (FANCA) gene | Orphan Designation | - |
| arrhythmogenic right ventricular dysplasia 9 | Adeno-associated virus serotype rh.74 vector containing the human Plakophilin-2a transgene (AAVrh.74-PKP2a) | Orphan Designation | - |
| autosomal recessive osteopetrosis | Autologous genetically modified CD34+ hematopoietic cells transduced with a lentiviral vector (LV-EFS-TCIRG1-WPRE) that encodes for the human TCIRG1 transgene | Orphan Designation | - |
| leukocyte adhesion deficiency 1 | hematopoietic stem cells modified with a lentiviral vector containing the CD18 (Integrin Beta 2) gene | Orphan Designation | - |
| polycystic kidney disease 2 | Adeno-associated virus serotype rh.74 vector containing the human Plakophilin-2a transgene (AAVrh.74-PKP2a) | Orphan Designation | - |
| pyruvate kinase deficiency of red cells | lentiviral vector containing the human liver and erythroid pyruvate kinase gene | Orphan Designation | - |