Developing Antisense Oligonucleotide Medicines for Rare Genetic Diseases

Michelle Hastings

University of Michigan, Hastings Lab, Medical Science Bldg, Ann Arbor MI 48109, USA, hastingm@umich.edu

Antisense oligonucleotides (ASOs) are programmable, RNA-targeted therapeutics with particular promise for rare genetic diseases underserved by conventional drug development. We are developing splice-switching ASOs (SSOs) for CLN3 Batten disease, a progressive neurodegenerative disorder affecting the brain and retina. Our first SSO medicine, Zebronkysen, is being used clinically to treat children with an ultra-rare CLN3 mutation. Treatment has been well tolerated, and early clinical observations are encouraging. Building on this experience, we are developing additional CLN3-targeted SSOs, including a therapy for the most common disease-causing mutation. I will discuss the design, preclinical validation, and clinical translation of these ASOs for CLN3 Batten disease.

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