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Webinar Series: Industry Partner Update from Avidity

Presentation and Discussion on initial data from the EXPLORE44 ^TM Clinical Trial: Evaluation of del-zota(formerly AOC 1044)for the treatment of individuals with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44)

Presented by:

• Elizabeth Ackermann, Senior Vice President Clinical Development, Avidity Bioscience
• Hussam Younis, Global Program Head, Avidity Bioscience

More About AOC 1044

Data from the Phase 1/2 EXPLORE44 trial showed that AOC 1044 delivered unprecedented concentrations of PMO in skeletal muscle with up to 50-times greater concentrations of PMO in skeletal muscle following a single dose compared to peptide conjugated PMOs in healthy volunteers. AOC 1044 was well tolerated, demonstrated statistically significant exon 44 skipping compared to placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg AOC 1044 and increased exon skipping in all participants. Avidity plans to provide a first look at AOC 1044 data in people living with DMD44 in 2H 2024.