MeiraGTx Posts Encouraging Gene Therapy Data From Eye Disorder Trial
- MeiraGTx Holdings plc announced topline data from the Phase 1/2 study MGT009 of botaretigene sparoparvovec, gene therapy for X-linked retinitis pigmentosa (XLRP).
- Treatment with botaretigene sparoparvovec was found to be generally safe and well-tolerated.
- Significant improvements were demonstrated across retinal, visual functions, and functional vision in participants treated with botaretigene sparoparvovec compared to the randomized untreated control arm of the study at six months post-treatment.
- MeiraGTx and Janssen Pharmaceuticals Inc, a unit of Johnson & Johnson , are jointly developing botaretigene sparoparvovec.
- In the dose-escalation and expansion phases of the MGT009 study, improvements in vision were seen in subjects treated with low and intermediate doses of botaretigene sparoparvovec compared to the randomized concurrent control arm at 6 months.
- Improvements were demonstrated in retinal sensitivity on static perimetry, functional vision in a vision-guided mobility assessment, and other visual function and functional vision measures.
- The Phase 3 Lumeos study of botaretigene sparoparvovec for XLRP with disease-causing variants in the RPGR gene is actively dosing patients.
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