Replies to post #4647 on GTC Biotherapeutics (fka GTCB)

[exwannabe]

>> companies with interesting new technologies should not compound their risk by applying their technology to difficult or intractable targets


And perhapse that's exactly why GTCB choose not to lead with the DIC indication?

First establish ATryn as a valid biologic via the antithrombin hereditary deficiency trials, then expand to DIC; seams like the exact stratagy you would like :-)

[HobGlobulin]

From "The Demon in the Freezer" by Richard Preston (Barbara Birke was a German nursing student who contracted hemorrhagic smallpox from a patient at St. Walberga Hospital in 1970):

>> With flat hemorrhagic smallpox, the immune system goes into shock and cannot produce pus, while the virus amplifies with incredible speed and appears to sweep through the major organs of the body. Barbara Birke went into a condition known as disseminated intravascular coagulation (DIC), in which the blood begins to clot inside small vessels that leak blood at the same time. ... <<

I believe the U.S. government has spent around half a billion dollars to provide smallpox vaccine for everyone old enough to receive it. I also believe they are ready to spend a similar sum on a new vaccine for the 10% of the population who are too immune-compromised to be candidates for the old vaccine.

From "The Demon in the Freezer" I learned scientists are able to infect macaque monkeys with hemorrhagic smallpox. If ATryn is efficacious in treating DIC, the news may become known sooner than expected.

Hob

>> Mother Nature is an extremely powerful force and she has ways of wreaking havoc in a manner which you never imagined. <<

Isn't that the point Jeff Goldblum kept making in "Jurassic Park?"

[vinmantoo]

<<In regards to DNA or viral contamination, the bottom line is that the FDA is convinced that it's not a problem for CHO cells. The verdict is still out on GTCB's expression format.

To the 2nd point, I still submit that there is a far, far, far greater concern about heterogeneity and contamination when dealing with a herd of animals than a fermentation culture of CHO cells. Mother Nature is an extremely powerful force and she has ways of wreaking havoc in a manner which you never imagined. However, as I stated before, I believe that GTCB's scientists have overcome this risk, and in this case, it's a good thing, because this risk is the single most important reason that we all have been able to purchase this stock at a very nice discount.>>

First, you don't really think DNA is an issue with either CHO or milk derived proteins, do you?

Second, the opinion of the FDA isn't going to be the same as the uninformed public and there is no reason to think they won't considered a contolled set of animals held in very clean and isolated conditions as being safe. The EMEA has already approved rATIII. I repeat, the EMEA has already approved rATIII. It is now a validated technology. Why do you think the FDA will be so negative?

Third, you are comparing CHO derived proteins to those from goats milk. That isn't a valid comparison for rATIII. You need to compared milk from clean and well isolated goats that derived from pooled human blood taken from a variety of people. Such blood can contain many pathogens and viruses known and unknown. How do you think HIV and Hep atici B and C have spread so widely? There is no comparison and the milk derived proteins are far safer.

[DewDiligence]

>The problem with sepsis is that basically the body is in full blown nuclear meltdown. It's a very risky indication where many compounds have failed. There are a lot of unknowns.<

Plasma-derived antithrombin is already approved for DIC/sepsis in Japan and some European countries, but I guess that’s a detail that wasn’t consistent with your message.