nL-PRPH2-001

nL-PRPH2-001 is a personalized antisense oligonucleotide (ASO) therapy developed by the n-Lorem Foundation for the treatment of rare forms of inherited retinal dystrophy caused by mutations in the *PRPH2* (peripherin-2) gene. PRPH2 is a transmembrane protein essential for the structural stability of the light-sensing outer segments of photoreceptor cells; mutations in this gene lead to progressive vision loss conditions such as retinitis pigmentosa, pattern dystrophy, and macular degeneration. As an "n-of-1" or personalized medicine, nL-PRPH2-001 is specifically engineered to target a particular genetic variant—notably the PRPH2 c.623G>A (p.Gly208Asp) mutation—to modulate gene expression. The ASO works by binding to the *PRPH2* mRNA transcript to either degrade the mutant allele via RNase H-mediated knockdown (reducing toxic gain-of-function effects) or to correct aberrant splicing. It is administered through intravitreal injection and is currently being evaluated in early-phase clinical trials to assess safety and efficacy in preserving retinal function in individual patients with these specific genetic profiles.