Replies to post #25281 on Biotech Values

[drbio45]

I note ALNY has not done well thus far with delivery. Merck...dropped their Macular Degeneration JV last year when ALNY was unable to get their med across the single barrier of one cell membrane. I believe some patients suffered some injuries.”
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I had heard the same thing but didn't feel it appropriate to start rumors based on heresay. I had heard that the rnai products couldn't get passed the cell membrane but had not heard about injuries.

[isolution]

RNAi and Anlylam


Dew,you agree that delivery into the cells is a key issue for RNAi (same for antisense or rybozymes).

To solve the problem of cell penetration and also to enhance half life, iRNA companies have several strategies such as complex the RNA with a lipid or modify the RNA's phosphate backbone...etc.

The most experienced company for these is SIRNA for it comes form their Ribozyme Pharma decade of experience.

I didn't check the modification strategies for each Acuity, Alnylam and Sirna, but so far, only Acuity and Sirna are in the clinic.

I haven't heard about pateint injuries, this could depend on the potency/toxicity ratio of the construct itself, but you see that on animals before to do a dose ranging on patients. That must be a rumor.

PS: none of the above cited Cos are posting at ARVO this year.