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Saturday, March 05, 2011

Delivering RNAi

I would be remiss if I were to not revisit the Cargo Cult of RNAi.

Last week we had a conference on the Cargo Cults of Seattle. RNAi was represented by PhaseRx. Since their inception they have had very little to report besides their 19 million dollar series A financing. They do however make very bold claims on their website.

RNAi therapeutics, also referred to as short interfering RNA or siRNA, have demonstrated broad applicability to many therapeutic areas, including cancer, inflammatory and autoimmune diseases, infectious disease and metabolic disorders.

These promising therapeutics are being held largely in check today by the lack of broadly applicable delivery technology. RNAi therapeutics generally cannot, on their own, penetrate the cell membrane and gain access to the cytoplasm where they can reach the drug targets.

The PhaseRx polymer system overcomes the central stumbling block in the field of RNAi therapeutics by delivering RNAi molecules into the cytoplasm where they can reach and inhibit the desired target of interest.

How do they have evidence of efficacy for the diseases if they've been held in check by delivery. Does their delivery include a ride straight to the cells of interest, avoiding a depletion of drug by being absorbed by other cells?

The problem is scalability. If you work as an engineer in a biopharmaceutical manufacturing facility you start small and scale up. There are certain relationships involved in scaling up that have been addressed by the industry, resulting in predictable outcomes. But with RNAi, they have predicted the outcomes without doing the research. The outcomes exist mostly on company websites. What do they now know that makes everything work where it failed in the past? As predicted by the CCS, the RNAi industry is not evolving.

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