Interventions of Interest

  • sonpiretigene isteparvovec (Nanoscope Therapeutics)

ICER assessed the comparative clinical effectiveness and value of sonpiretigene isteparvovec (Nanoscope Therapeautics) for the treatment of advanced retinitis pigmentosa. An independent appraisal committee voted that the current evidence is adequate to demonstrate a net health benefit of sonpiretigene isteparvovec compared to usual care.

Final Documents

For questions please contact info@icer.org.

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ICER’s Chief Medical Officer David Rind, MD stated:

“Many individuals with retinitis pigmentosa develop severe vision loss as the disease progresses and photoreceptor cells are lost. Researchers have examined the idea of inserting proteins into other cells that remain in the back of the eye to allow those cells to react to light. Sonpiretigene isteparvovec is a one-time gene therapy that codes for such proteins in remaining retinal bipolar cells. We have a number of uncertainties about the efficacy and durability of this therapy given the limited evidence to date, but the underlying scientific approach is remarkable.”