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Sunday, January 13, 2008

New Factor IX Products in Development

What's going on with hemophilia B? A lot! I've read recently about four new studies about new products for hemophilia B patients. In a nutshell:

A. Inspiration Biopharmaceuticals (www.inspirationbio.com) has signed an agreement with Cook Pharmacia to develop an injectable recombinant factor IX. So, what's new about that? I mean, we already have that. Well, Inspiration is focusing on revolutionizing hemophilia treatment in two ways: 1) developing lower-cost versions of existing intravenous recombinant therapies; and 2) developing non-invasive administration of therapies. Like inhaling? (Hence the name, Inspiration? Just a wild guess)

B. Baxter International (www.hemophiliagalaxy.com) has begun preclinical work on a genetically engineered product for bleeding in patients with hemophilia B. Again, we already have that, so what's new? With its partner Nektar Therapeutics of San Carlos, Calif., Baxter wants a more long-lasting treatment for hemophilia B. Imagine infusions lasting all week instead of a day, or maybe two weeks!

C. Nastech Pharmaceutical (www.nastech.com) has begun a feasibility study with an undisclosed global leader in the production of plasma products. The study will examine the possibility of developing an alternative delivery method of factor IX without an injection. Not much said about this one: who's the company? What's the method? More to come, hopefully!

D. GTC Biotherapeutics, Inc., of Framingham, Massachusetts, has purchased a license to develop a new recombinant factor IX product with ProGenetics LLC of Blacksburg, Virginia.

Currently, the only existing recombinant factor IX product is BeneFIX, made by Wyeth Pharmaceuticals of King of Prussia, Pennsylvania. But there sure is a lot of commercial interest in this hemophilia population, which is a small. Of the approximately 17,000 people with hemophilia in the US, only about 15% are estimated to have factor IX deficiency.

If this is all interesting to you, then see about attending The Coalition for Hemophilia B one-day consumer symposium on Saturday, March 8, 2008, from 9:30 am--5 pm, at the Millennium Broadway Hotel in New York City. For information, call Kim at 212-520-8272 or email her hemob@ix.netcom.com

Great Book I am Reading: Dreams from My Father: A Story of Race and Inheritance, by Barack Obama. Kevin bought this for me for Christmas. I thought, oh great, a book ghostwritten for a politician seeking election. But I was wrong. He wrote this in 1995, when he was a senator-elect. I am captivated already: it is honest, intimate, frank and eloquent. This is a guaranteed great read (and not a political endorsement), and I always appreciate a well-written book. I am only on Chapter 3 but it is insightful and informative. Obama has led an amazing life, and in this book, he traces his roots, from his grandparents to his childhood, and the unusual path his genealogy has taken and why. Eventually he returns to Kenya to learn more about the father he barely recalls. This is a look at race relations in America, one man's quest to make a difference for his race, and a quest for the meaning of family for a man who was raised in several diverse cultures. (For those of you fearing I am making a "statement," I also have it on my list to read John McCain's book, Faith of Our Fathers, also about the impact his grandfather and father had on his life. Father's Day gift for Kevin?). Three stars out of four.

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