Several biotech firms reported pivotal progress in therapeutic development targeting rare diseases and cancers. Ionis Pharmaceuticals announced positive top-line results in a phase 3 trial for Alexander disease using their antisense oligonucleotide candidate zilganersen, demonstrating improved mobility and planning an FDA submission. MBX Biosciences revealed phase 2 data showing statistical improvements in chronic hypoparathyroidism patients treated with canvuparatide, poised to initiate phase 3 studies. Dana-Farber Cancer Institute introduced MARLIN, an AI-powered diagnostic tool combining DNA methylation patterns for acute leukemia subtype inference, promising enhanced diagnostic accuracy and clinical decision support. Meanwhile, Capsida Bio suspended its gene therapy trial for STXBP1 encephalopathy following a patient death, underscoring challenges in gene therapy safety.