Nanoscope advances gene-agnostic therapy for severe vision loss in RP A failed RNA drug, major job cuts, a win for myeloma CAR T, and more led the news this week. Brought to you byDDN editorial team

Nanoscope advances gene-agnostic therapy for severe vision loss in RP A failed RNA drug, major job cuts, a win for myeloma CAR T, and more led the news this week. Brought to you byDDN editorial team https://www.drugdiscoverynews.com/nanoscope-advances-gene-agnostic-therapy-for-severe-vision-loss-in-rp-17521 The FDA has accepted Nanoscope Therapeutics’ Biologics License Application for MOGENRY (sonpiretigene isteparvovec), an investigational optogenetic gene therapy for people with retinitis pigmentosa (RP) and severe vision loss. If approved, MOGENRY would be the first gene-agnostic treatment for this stage of RP, allowing patients to receive treatment independent of their individual mutation. RP encompasses more than 100 genes and more than 1,000 known mutations, creating a challenge for therapies designed around individual genetic causes. MOGENRY instead delivers a synthetic opsin gene to surviving bipolar retinal cells, making them sensitive to light after photoreceptor loss. The BLA is supported by positive results from the Phase 2b/3 RESTORE trial, which met its primary and key secondary endpoints for visual acuity at weeks 52 and 76, alongside longer-term data from the REMAIN follow-up study. – Bree Foster