- In May 2024, Ionis Pharmaceuticals deepened its partnership with Roche in May 2024 to co-develop antisense oligonucleotides targeting neurological disorders such as Alzheimer’s and Huntington’s. Roche obtained exclusive global rights to develop, manufacture, and commercialize these early-stage RNA-targeted programs, reinforcing the alliance's long-term commitment to CNS therapeutics
- In March 2024, Sarepta Therapeutics received traditional FDA approval for ELEVIDYS (delandistrogene moxeparvovec-rokl) for ambulatory Duchenne muscular dystrophy patients and accelerated approval for non-ambulatory patients aged 4 + years
- In February 2024, Wave Life Sciences introduced a novel peptide nucleic acid (PNA) backbone antisense platform enhancing stability, binding affinity, and CNS targeting. This innovation strengthens oligonucleotide platforms for neurological applications
- In January 2024, TransCode Therapeutics entered a collaboration with Debiopharm to develop targeted nucleic acid therapeutics for oncology indications. This alliance aims to leverage TransCode’s delivery technology alongside Debiopharm’s clinical and regulatory expertise to develop antisense oligonucleotides against solid tumor targets
- In December 2023, BioMarin launched Phase 1 clinical trials for BMN 349, an antisense oligonucleotide targeting phenylketonuria (PKU), marking advancement in ASO therapy for metabolic conditions
