Nusinersen, developed by Biogen in collaboration with Ionis Pharmaceuticals, is an antisense oligonucleotide that targets the survival motor neuron 2 (SMN2) gene.
Loncastuximab tesirine is an antibody-drug conjugate (ADC) developed by ADC Therapeutics, targeting CD19, a protein that is expressed on the surface of B cells.
Antisense oligonucleotide (ASO) drugs represent a significant branch of the small nucleic acid therapeutic field, functioning as molecules that regulate gene expression through specific binding to mRNA.