Drug Type Gene therapy |
Synonyms A001 MeiraGTx, AAV RPE65 MeiraGTx, AAV2/5 OPTIRPE65 + [2] |
Target |
Action- |
Mechanism RPE65 gene transference |
Therapeutic Areas |
Active Indication- |
Originator Organization |
Active Organization- |
Inactive Organization |
License Organization- |
Drug Highest PhasePendingPhase 1/2 |
First Approval Date- |
RegulationRare Pediatric Disease (United States) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Amaurosis Congenita of Leber, Type 1 | Phase 2 | United States | 01 Apr 2016 | |
Amaurosis Congenita of Leber, Type 1 | Phase 2 | United Kingdom | 01 Apr 2016 | |
Biallelic RPE65 mutation associated retinal dystrophy | Phase 2 | United States | 01 Apr 2016 | |
Biallelic RPE65 mutation associated retinal dystrophy | Phase 2 | United Kingdom | 01 Apr 2016 | |
Leber Congenital Amaurosis | Phase 2 | United Kingdom | 09 Mar 2016 |
Phase 1/2 | 15 | (Low Dose AAV-RPE65) | gitgayxszo = oapqszmwce lmtjdxcsog (inmjfhfwtu, ubvdaltitv - dntlpdicja) View more | - | 08 Apr 2021 | ||
(Intermediate Dose AAV-RPE65) | gitgayxszo = bmlbnrehor lmtjdxcsog (inmjfhfwtu, ojoonoceao - lvzhhxocgh) View more | ||||||
Phase 1/2 | RPE65 gene | - | lkanpfbkuz(ogqusbxcaj) = qdtxhatebg pycrwwkvaa (hyshaicxie ) | Positive | 17 Oct 2017 |