LUXTURNA (voretigene neparvovec-rzyl) is a recombinant adeno-associated virus serotype 2 (AAV2) vector with a cytomegalovirus (CMV) enhancer and chicken beta actin (CβA) promoter driving expression of the gene for human retinal pigment epithelium 65 kDa protein (hRPE65), which is an isomerohydrolase converting all-trans-retinyl ester to 11-cis-retinol in the retinoid visual cycle.
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PARAMETER |
DATA |
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Manufacturer |
Spark Therapeutics, Inc. |
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Transgene |
Retinal pigment epithelial 65 kDa protein [RPE65] |
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Indication |
Luxturna is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s). |
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Virus and Serotype |
Adeno-associated virus serotype 2 (AAV2) |
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Cell Substrate |
Mammalian cell-substrate |
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Manufacturing platform |
Transient transfection |
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Dose in vial/final container |
5 x 1012 vector genomes (vg) per mL (0.05 mg vector/ml) |
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Dose/patient |
1.5 x 1011 vector genomes (vg), administered by sub-retinal injection in a total volume of 0.3 mL for each eye. |
SUPPORTING CLINICAL TRIALS
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NCT |
TRIAL PHASE |
SUBJECTS ENROLLED |
STUDY TITLE |
COUNTRIES |
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NCT00516477 |
1 |
12 |
United States |
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NCT01208389 |
1, 2 |
12 |
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects with Leber Congenital Amaurosis (LCA) 2 |
United States |
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NCT00999609 |
3 |
31 |
Safety and Efficacy Study in Subjects with Leber Congenital Amaurosis |
United States |
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12/19/2017 |
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11/22/2018 |
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10/15/2020 |
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| 08/04/2020 |
A meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) was held on October 12, 2017 to provide feedback to FDA regarding clinical efficacy and safety, and an overall benefit-risk assessment of LUXTURNA.