Beacon Therapeutics reported positive pivotal Phase II/III results for laruparetigene zovaparvovec (laru-zova) in X-linked retinitis pigmentosa (XLRP), setting up a potential regulatory path. The Vista trial met its FDA-endorsed primary endpoint, improving low luminance visual acuity (LLVA), and the company reported that both the high- and low-dose groups showed substantial letter gains at 12 months versus no response in the untreated control. In parallel, Intrabio won FDA approval for Aqneursa (levacetylleucine) as the first treatment for ataxia-telangiectasia, following the drug’s earlier FDA nod for Niemann-Pick disease type C. The approval expands the use of a chemically modified amino acid approach, targeting a rare neurodegenerative disorder with limited treatment options. Together, the updates highlight continued momentum in gene and pathway-modulation therapies for rare diseases—areas where endpoints and regulatory scrutiny often hinge on disease-specific functional measures and durability of effect. For investors and clinicians, the two moves also emphasize how quickly rare-disease franchises can expand when pivotal or label-setting evidence clears major thresholds.