Biotech Values

[ghmm]

RNA/GSK / SRPT:

Skipping Multiple Exons of Dystrophin Transcripts
Using Cocktail Antisense Oligonucleotides
h/t @AndyBiotech

http://online.liebertpub.com/doi/pdf/10.1089/nat.2013.0451
On possible reasons for RNA Phase 3 failing

Finally, skipping ef?ciency via subcutaneous
administration is 40%–80% lower than that achieved via
intravenous administration, at least in the mouse model
(Heemskerk et al., 2010). Taken together, exon skipping
therapy that targets different exons, or with different AO
chemistry and intravenous administration might be more
effective in terms of recovery of motor function in DMD
patients.