2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Hirschsprung disease | autologous neurospheres containing enteric neural stem cells, enteric neurons, and glia | Orphan Designation | - |
| LAMA5-related multisystemic syndrome | a gene editing therapy that uses a CRISPR (clustered regularly interspaced short palindromic repeats)-based adenosine base editor (ABE), delivered using a smooth muscle cell targeting adeno-associated virus (AAV) capsid called AAV-PR (comprised of the parent AAV9 capsid modified with a unique 7-mer amino acid after amino acid 588 of VP1 protein) | Orphan Designation | - |
| autoinflammatory disease, multisystem, with immune dysregulation, X-linked | a gene editing therapy that uses a CRISPR (clustered regularly interspaced short palindromic repeats)-based adenosine base editor (ABE), delivered using a smooth muscle cell targeting adeno-associated virus (AAV) capsid called AAV-PR (comprised of the parent AAV9 capsid modified with a unique 7-mer amino acid after amino acid 588 of VP1 protein) | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | a gene editing therapy that uses a CRISPR (clustered regularly interspaced short palindromic repeats)-based adenosine base editor (ABE), delivered using a smooth muscle cell targeting adeno-associated virus (AAV) capsid called AAV-PR (comprised of the parent AAV9 capsid modified with a unique 7-mer amino acid after amino acid 588 of VP1 protein) | Orphan Designation | - |