Biotech Values

[acgood]

A few more thoughts in response as promised.

I reiterate that ISIS does a great job of presenting the science. You can also find on their website (and scattered in fragments around my website) a series of posters from last year's annual meeting that expand on various topics.

CRP - I agree with your summary here. Big risk, big rewards. Years away from knowing the answer though.

Do you know what size royalties they typically mean when they say 'double digit'?

Not sure what specifically you are referring to. Mipomersen is 30-50%, GSK deal starts in single digits and goes up to double digits.

Do you know of any background in anti-sense cancer risks? (it is the obvious question when it comes to playing with nucleotides)

I am not familiar with any studies about this, or it being raised as a concern. Warrants further research though

Do you know the kind/size of injection used for their drugs?

All are SC as of now. Started as weekly, generally 200 mg, now exploring 3x/week or daily as alternative.

How the heck are they planning to make an oral anti-sense?

They claim this is possible with the even more potent generation 2.5 chemistry (virtually all current pipeline drugs are generation 2.0)...again we are no where close to seeing this yet.

How are they using antisense to fix a splicing problem?

It is for the SMA program - they basically take a second gene (SMN-2) and alter it's splicing to produce the missing SMN-1 protein. I put up a slide here (have more info somewhere but can't find right now)
http://www.biotechduediligence.com/severe-rare-neurodegenerative-diseases.html