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Jul 28, 2009

U.K. DMD Charity to Provide AVI BioPharma $1.2M to Progress Drugs

  • Action Duchenne has earmarked about $1.2 million for a collaboration focused on progressing AVI BioPharma’s exon-skipping drug candidates for the treatment of duchenne muscular dystrophy (DMD).

    The company reports that its lead candidate, AVI-4658, is designed to skip exon 51 of the dystrophin gene, restoring the reading frame of the mRNA sequence, and resulting in a truncated but potentially functional form of the dystrophin protein. A dose-finding clinical trial evaluating systemic delivery of AVI-4658 is currently under way in the U.K.

    A second, preclinical-stage candidate, AVI-5038, is based on a second generation of AVI BioPharma’s phosphorodiamidate morpholino oligomer antisense chemistry and is deisgned to skip Exon 50.

     



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