Transgenic corn yields biotech drug for rare disease

Many biologics are notoriously expensive to produce, but genetically modified corn offers a potential alternative for producing complex protein drugs. And veteran Reuters scribe Ben Hirschler has chronicled one of the latest feats in "molecular farming," with scientists growing an enzyme drug for a rare genetic disease in transgenic corn.

The use of corn as a drug factory of sorts could cost less than current bio-manufacturing methods, Hirschler reported. While the scientists, led by Simon Fraser University in Canada, have yet to prove their corn-grown drug in clinical trials, their approach led to a corn strain that could yield alpha-L-iduronidase for the lysosomal storage disorder MPS I and they theorized that humans should be able to take the med safely without harmful side effects.

Sanofi's ($SNY) Genzyme unit and Biomarin ($BMRN) charge a hefty $300,000 annually to treat children with an enzyme-replacement drug called Aldurazyme for MPS I, according to the Reuters article. And it's relatively expensive and tricky to make such protein drugs with mammalian cell cultures inside bioreactors. Whether cheaper production methods would impact the cost of such drugs isn't addressed in the article, and it could take years before industrial-scale production of biotech drugs from the scientists' corn becomes commercially viable.

But count on pharma players to evaluate approaches to reduce their production costs, including plant-based methods. As Hirschler points out, Pfizer ($PFE) and partner Protalix BioTherapeutics took a step in this direction with their drug for the rare genetic disease Gaucher, using carrot cells in culture but not entire plants to produce the drug, on which the FDA stamped an approval in May. Also, German drugmaker Bayer has made news for its investment in the use of tobacco plants to produce protein drugs. 

- check out Hirschler's article

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