Shots:

• The P-II/III XIRIUS study involves assessing subretinal injection of cotoretigene toliparvovec in males with a genetically confirmed diagnosis of XLRP. Part 1 of the study was a 24mos. dose-escalation study while part II was a 12mos. dose expansion study with a high & low dose selected from part I based on a benefit/risk assessment
• The study failed to meet its 1EPs demonstrating improvement in the proportion of treated study eyes with ≥7 dB improvement from baseline at ≥5 of the 16 central loci of the 10-2 grid assessed by MAIA microperimetry
• BIIB112 is an investigational AAV8 vector-based gene therapy to provide full-length functioning RPGR protein in patients with XLRP

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