I respect your knowledge when it comes down to the intricacies of science. What is your general feelings regarding the other platforms (aside from DMD) as to the future value of this company?. Obviously, the short term binary catalyst is currently DMD, however, Chris's exuberant statement of new and exciting developments to come has kept me thinking an "out of left field" development. Curious as to your general company wide view. TIA
Rich, The sky is the limit. Once Eteplirsen is granted AA and then completes a PhaseIII trial all the PMO naysayers will have to reassess their opinions. One would believe that these naysayers should have already changed their opinions after the PhaseII data. At every escalating stage the PMO has outperformed the S-DNA by a wide margin. Still the naysayers come out. I heard someone state that the PMO will never perform as well as Drisa because of Oligo length. Can you imagine that. No mention of the inherent toxicities associated with these phosphorothioates. No mention of the anionic charge load that these oligo's have. It's the PMO length that's the problem. The genesis of these ridiculous claims is Stanley Crooke. His only success is liver targets. The one organ that sucks up oligos like a sponge. They are also going after SMA but they are bypassing the BBB by administering via spinal canal. Many people don't remember but Isis tried to go after viral targets and they couldn't get the 2'MOE into their target cells. The PMO+ oligo in marburg easily enters epithelial cells to the tune of a 7LOG10 reduction. Don't forget…The PMO is too long.
Sentiment: Strong Buy
Thanks Jon and Bionerd. Sounds like the launching pad for the other platforms is largely predicated and linked to "E" getting to market. In some ways this is good and in others not so good. I would like to see the other platforms advance independently (though I understand the proof of concept and validity issues). Thanks for your feed back.
There are many, many potential targets. The Gene Tools publication database is loaded with examples of using Morpholinos to alter gene expression. If there is RNA in a creature a Morpholino can bind it -- in animals, protists, plants, bacteria, viruses and about a year ago I found a German dissertation using a Morpholino for a knockdown in a fungus. Delivery into the cytosol can still be an issue for therapeutic applications but the technologies are improving. I have no doubt that once Sarepta has an approved drug they will have no problem finding good targets for the next 1000 drugs.