2
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Danon disease | autologous human CD34+ hematopoietic stem and progenitor cells (HSPCs), derived from G-CSF/plerixafor mobilized peripheral blood stem cells (PBSCs) of patients with Danon disease (DD) | Orphan Designation | - |
| Friedreich ataxia | autologous human CD34+ hematopoietic stem and progenitor cells isolated from mobilized peripheral blood stem cell of patients with Friedreich's ataxia, modified ex vivo using CRISPR/Cas9 technology to remove the GAA hyper-expansion mutation in the intron 1 of frataxin | Orphan Designation | - |
| Friedreich ataxia 2 | autologous human CD34+ hematopoietic stem and progenitor cells isolated from mobilized peripheral blood stem cell of patients with Friedreich's ataxia, modified ex vivo using CRISPR/Cas9 technology to remove the GAA hyper-expansion mutation in the intron 1 of frataxin | Orphan Designation | - |