Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa - Concepedia

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Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa

DOI Full Paper Access

29

Citations

18

References

2024

Year

Michel Michaelides, Cagri G. Besirli, Yesa Yang, Thales A. C. de Guimarães, Sui Chien Wong, Rachel M. Huckfeldt, Jason Comander, José‐Alain Sahel, Syed Mahmood Shah, James Tee,

American Journal of Ophthalmology

Abstract

AAV5-hRKp.RPGR demonstrated an anticipated and manageable AE profile through 52 weeks. Safety and efficacy findings support investigation in a phase 3 trial.

References

	Year	Citations

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