🧬 Rare Disease & Gene Therapy

AAV Vector Capsid EngineeringNewAdeno-associated virus vectors are re-engineered at the capsid surface to redirect tissue… ASO Splice ModulationNewAntisense oligonucleotide (ASO) modulation of alternative splicing involves the use of ASOs… Base & Prime Editing PrecisionNewBase editing is a precise genome editing technique that allows for the conversion of one… N-of-1 Ultra-Rare Disease Drug PipelineNewDevelopment of personalized antisense drugs for patients with unique mutations is a… Lysosomal Enzyme Replacement TherapyNewEnzyme replacement therapy for lysosomal storage diseases involves the administration of…