Transgenic animals

Hatching a New Factor Therapy

Here’s a new post I read in the news wires: biotech start-up company Genavia Therapeutics wants to produce a blood clotting protein from chicken eggs.

Just when you think you’ve heard everything.

You may have read in my newsletter PEN about pigs that may be able to express factor through their milk glands. These are called transgenic animals. Another company here in Massachusetts wants to do this with goats.

Well, Genavia will do it with chicken eggs. It plans to use technology developed by Californian company Origen Therapeutics to produce human factor VIII. The technology involves injecting a human gene into chickens and extracting the protein from the whites of their eggs. Omelets, anyone?

Genavia chief operating officer Peter Bradley has said that this “avian transgenic technology” could cut the cost of treatment to as little as 20% of the current price. “We plan to not only take market share from the current players but to actually grow the market,” Bradley said as reported in the Waikato Times.* Now that would be something to crow about.

Stay tuned as we watch developments in this research and production; we hope to provide more information as it becomes available.

*”Start-up gets boost” by ANDREW JANES – Waikato Times, 10/03/2009

Great Book I Just Read
Beautiful Boy by David Sheff.
Heartbreaking, gutsy, honest, scary and hopeful, Sheff recounts the harrowing and bleak downward spiral of his “beautiful boy,” his teen son Nic, into a nightmarish world of drugs and addiction. This was a child who seemed to have it all: intelligence, charm, opportunities, and two caring parents. Sheff’s account is a damning testament to the devastating threat of meth to our youth, the lure drugs have to teen boys, the helplessness and anguish of families, and the sliver of hope that some teens can survive addiction. ALL parents of teens and preteens MUST read this book. Teens should read it too; it should be required reading in all grades. All of us can see ourselves on its pages: the denial (“My son wouldn’t…”), the suspicion (“Could he possibly…”) and the blame (“Where did we go wrong?”). Sheff’s skilled storytelling allows all parents to journey with him through hell and back. It’s the best book you will read all year, and the most important. Four stars.

Factor Through Goats?

A persistent question we are always asked is “What ever happened to gene therapy?” We used to write extensively about this subject when gene therapy was hot, but since the deaths of two young people in gene therapy trials unrelated to hemophilia, all gene therapy seems to have taken a slow and extremely cautious route. There instead seems to be great interest in creating new and less expensive ways to manufacture factor. We recently heard of a new biotech company in California, founded by a parent of a child with hemophilia, that is developing all therapies, which hints that these products one day will be lower in cost.

And the newswires announced this last week: GTC Biotherapeutics, a Massachusetts-based biotech company, held a webcast this morning from Monaco; the webcast will be available tomorrow through the company’s website. This company “develops, produces, and commercializes therapeutic proteins through transgenic animal technology.” This means literally milking animals for the desired protein that is developed through gene therapy and then expressed through the animal’s milk.

Like many, I feel a bit skeptical but according to GTC, in August 2006 its recombinant form of human antithrombin was approved by the European Commission for use in patients with hereditary antithrombin deficiency undergoing surgical procedures. This was the first approval anywhere in the world of a therapeutic protein produced from a transgenic animal. GTC has developed goats that have the human antithrombin gene linked to a milk-protein promoting gene so that they express this protein in their milk.

In 2006, GTC was granted a patent in the United States through 2021 for the production of any therapeutic protein in the milk of any transgenic mammal. And GTC has established a strategic collaboration with LFB Biotechnologies of France to jointly develop recombinant human factor VIIa as a potential treatment for hemophilia inhibitor hemophilia patients. This would be a direct competitor to NovoSeven, apparently. Although the article doesn’t directly state this, it implies that this recombinant FVIIa would be expressed by animals?

It bears watching, and we hope to bring more information about this through PEN and through HemaBlog. Watch the webcast yourself tomorrow at the GTC web site, www.gtc-bio.com.

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