4
Orphan Designations
0
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Au-Kline syndrome | Adeno-associated viral vector serotype 9 (AAV9) containing human RPE65 gene | Orphan Designation | - |
| Becker muscular dystrophy | A recombinant adeno-associated virus serotype 9 (rAAV9) vector carrying a compact high-fidelity Cas12i (hfCas12Max) and a CRISPR RNA (crRNA) targeting human DMD exon 51 SD site | Orphan Designation | - |
| Duchenne muscular dystrophy | A recombinant adeno-associated virus serotype 9 (rAAV9) vector carrying a compact high-fidelity Cas12i (hfCas12Max) and a CRISPR RNA (crRNA) targeting human DMD exon 51 SD site | Orphan Designation | - |
| HSD10 disease, infantile type | recombinant adeno-associated virus serotype 9 vector carrying high-fidelity Cas13Y (hfCas13Y), an RNA editor, gene expression cassette, and guide RNAs targeting human MECP2 | Orphan Designation | - |
| HSD10 disease, neonatal type | recombinant adeno-associated virus serotype 9 vector carrying high-fidelity Cas13Y (hfCas13Y), an RNA editor, gene expression cassette, and guide RNAs targeting human MECP2 | Orphan Designation | - |
| auditory neuropathy | a novel CRISPR/Cas13X RNA base-editing therapy drug | Orphan Designation | - |
| autosomal recessive nonsyndromic hearing loss 9 | a novel CRISPR/Cas13X RNA base-editing therapy drug | Orphan Designation | - |
| branchio-oto-renal syndrome | a novel CRISPR/Cas13X RNA base-editing therapy drug | Orphan Designation | - |
| fetal methylmercury syndrome | recombinant adeno-associated virus serotype 9 vector carrying high-fidelity Cas13Y (hfCas13Y), an RNA editor, gene expression cassette, and guide RNAs targeting human MECP2 | Orphan Designation | - |
| inherited kidney disorder | Adeno-associated viral vector serotype 9 (AAV9) containing human RPE65 gene | Orphan Designation | - |
| inherited renal tubular disease | Adeno-associated viral vector serotype 9 (AAV9) containing human RPE65 gene | Orphan Designation | - |
| symptomatic form of muscular dystrophy of Duchenne and Becker in female carriers | A recombinant adeno-associated virus serotype 9 (rAAV9) vector carrying a compact high-fidelity Cas12i (hfCas12Max) and a CRISPR RNA (crRNA) targeting human DMD exon 51 SD site | Orphan Designation | - |