I simply lump all RNA drug companies together because I think targeting RNA is a mistake. I stand by that claim. Serepta, like the others, has some interesting chemistry in its product. Phosphorodiamidate morpholino oligomers (PMOs) are synthetic structures modeled after the natural nucleic acid framework of RNA. They replace the ribose ring with morpholine which is linked through phosphorodiamidate rather than anionic phosphodiester bonds. You can get some of this stuff from a company called GeneTools LLC and start your own RNA company or just do some research with it.
Gene Tools, LLC produces and markets Morpholino oligos for antisense applications and ancillary products for basic research. We are also engaged in research relating to delivery of Morpholino oligos into cells and use of Morpholino oligos as probes and in diagnostic systems.Lest people think I am against everything, I want to say that I love this kind of science. It is not unlike the work of one of my heros Gertrude Elion. Here she talks about her work with nucleic acid analogs:
Morpholino oligos and the subunits used in their assembly were invented and developed at Sarepta Therapeutics (formerly AVI BioPharma Inc. and earlier ANTIVIRALS Inc.), the pioneer antisense company founded by James Summerton in 1980. Gene Tools manufactures and markets research quantities of Morpholino oligos. In Japan, Funakoshi Co. is the distributor of Morpholino oligos and related products.
The work became fascinating almost from the very beginning. We were exploring new frontiers, since very little was known about nucleic acid biosynthesis or the enzymes involved with it. I had been assigned quite early to work on the purines and, with the exception of a few deviations into the pteridines and into some other condensed pyrimidine systems, the remainder of my work concentrated almost completely on the purines. Each series of studies was like a mystery story in that we were constantly trying to deduce what the microbiological results meant, with little biochemical information to help us.There came a time where Gertrude had to go off the beaten path and deduce what the results meant. She went from chemistry to biology to "what do these results mean?" It is at this same juncture in the research process where GeneTools LLC and Serepta parted ways. GeneTools LLC represents the science of PMOs versus RNA. Serepta represents the business of drug approval. Somewhere between the work of GeneTools LLC and Serepta we enter the Cargo Cults.
Why didn't James Summerton make that journey? The powers of his PMOs as a drug, over RNAi molecules, are touted on the Serepta website:
- The ability to target specific gene sequences without non-specific interactions
- Safe at higher doses
- Able to UP and down regulate gene expression
- Easier to deliver to specific cells
- Resistant to enzymatic degradation