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Recombinant adeno-associated viral vector serotype 5 encodingStaphylococcus aureusCas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of theCEP290gene |
|---|---|
| Trade Name | |
| Orphan Indication | Leber congenital amaurosis |
| EU Market Approval | EU |
| EU Designation Date | 2017-10-16 00:00:00 |
| Sponsor | Pharma Gateway AB |
