Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as voretigene neparvovec-rzyl, AAV2-hRPE65v2, gene therapy vector
Voretigene neparvovec · 3 trials · 2 indications
The MLMT measures changes in functional vision, as assessed by the ability to navigate a course accurately and at a reasonable pace at different levels of environmental illumination. MLMT was assessed using both eyes at 1 or more of 7 levels of illumination, ranging from 400 lux (a brightly lit office) to 1 lux (a moonless summer night). Each light level was assigned a score code ranging from 0 to 6. A higher score indicated that a subject was able to pass the MLMT at a lower light level. A score of -1 was assigned to those who could not pass MLMT at 400 lux. The MLMT of each subject was videotaped and assessed by independent graders. The MLMT score was determined by the lowest light level at which the subject was able to pass the MLMT. The MLMT score change was defined as the difference between the score at Baseline and the score at Year 1. A positive MLMT score change from Baseline to Year 1 visit indicated that the subject was able to complete the MLMT at a lower light level.
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
| Arm | Type | Description |
|---|---|---|
| AAV2-hRPE65v2,voretigene neparvovec-rzyl | EXPERIMENTAL | voretigene neparvovec rzyl, 1.5 E11 vector genomes, per eye, administered by subretinal injection in a volume of 0.3mL, 6-18 days apart |
| Control | NO_INTERVENTION | No intervention |
| voretigene neparvovec-rzyl (AAV2-hRPE65v2) | EXPERIMENTAL | Administration of study agent (AAV2-hRPE65v2) to the previously, uninjected contralateral eye: |
| dose cohort 1 | EXPERIMENTAL | 1.5E10 vector genomes voretigene neparvovec-rzyl in 150 microliters administered subretinally |
| dose cohort 2 | EXPERIMENTAL | 4.8E10 vector genomes voretigene neparvovec-rzyl in 150 microliters administered subretinally |
| dose cohort 3 | EXPERIMENTAL | 1.5E11 vector genomes voretigene neparvovec-rzyl in 300 microliters administered subretinally |
| Name | Type | Description |
|---|---|---|
| AAV2-hRPE65v2,voretigene neparvovec-rzyl | BIOLOGICAL | Subretinal administration of gene therapy vector AAV2-hRPE65v2 (1.5E11 vector genomes per eye) to both eyes via surgical procedures on separate days. |
| voretigene neparvovec-rzyl | BIOLOGICAL | One time, subretinal administration of 1.5E11 vg AAV2-hRPE65v2 vector in 300 microliters to the contralateral, previously uninjected eye. |
Inclusion Criteria: * Willingness to adhere to protocol and long-term follow-up as evidenced by written informed consent or parental permission and subject assent (where applicable). * Diagnosis of LCA due to RPE65 mutations; molecular diagnosis is to be performed, or confirmed, by a CLIA-approved ...
Voretigene neparvovec is an investigational gene therapy developed for inherited retinal dystrophy caused by RPE65 mutations, including Leber congenital amaurosis. It is designed to deliver a functional copy of the RPE65 gene to retinal cells. It is being developed by Roche Holding AG under ticker RHHBY.
Voretigene neparvovec is being studied for inherited retinal dystrophy due to RPE65 mutations and for Leber congenital amaurosis, a rare inherited form of severe vision loss. These conditions are caused by mutations in the RPE65 gene, and the therapy is intended for patients with those confirmed mutations.
Voretigene neparvovec targets RPE65, an exogenous gene. It is a gene therapy designed to introduce a working copy of the RPE65 gene into retinal cells, aiming to restore the protein needed for normal visual function in patients whose disease is driven by RPE65 mutations.
Voretigene neparvovec is being developed by Roche Holding AG, which trades under the ticker RHHBY. The program is in the rare disease therapeutic area and uses a gene therapy modality, with clinical activity focused on inherited retinal dystrophy due to RPE65 mutations and Leber congenital amaurosis.
Voretigene neparvovec is in Phase 3 development. It is an investigational gene therapy and is not described as approved. The program includes a Phase 3 safety and efficacy study along with earlier Phase 1 work, and one trial remains active while another has completed.
Voretigene neparvovec has been studied in trials including NCT00999609, a Phase 3 safety and efficacy study in subjects with Leber congenital amaurosis and inherited retinal dystrophy due to RPE65 mutations, and NCT01208389, a Phase 1 follow-on study. A completed Phase 1 safety study, NCT00516477, enrolled 12 subjects.
Yes. Voretigene neparvovec is also known as voretigene neparvovec-rzyl and by the research designation AAV2-hRPE65v2. These names refer to the same gene therapy program targeting RPE65 for inherited retinal dystrophy due to RPE65 mutations and Leber congenital amaurosis.