February 1, 2010

Big Week for New Hemophilia Products

There was lots of news this week under the heading of “What can I look forward to when I don’t have gene therapy?”

How about extended half-life factor? So, instead of prophy three times a week, you dose once, and it lasts all week? Extended half-life factor is the next great thing in hemophilia, while we wait someday for a cure. I think this will be a reality in the next two years.

This week Biogen Idec (a company right here in Massachusetts; yes, they are freezing just like the rest of us this week) announced that they’ve dosed their first patient with extended half-life recombinant factor IX.There are about 6 companies studying long-acting factor IX. One of these is also GTC Biotherapeutics, also located in Massachusetts, which has a FIX product in preclinical studies. And this is really different: GTC is using transgenic animals, like rabbits and goats. These animals are specially bred to excrete human FVIII or IX through their milk glands. Fascinating!

Now even when a company has a new product in clinical trials, this doesn’t mean that it automatically will come to fruition. These are trials, and sometimes things don’t work out. For example, just this week Bayer cancelled its LipLong study, which seemed so promising. Bayer was exploring an extended half-life factor VIII (BAY 79-4980), but results weren’t as expected. But don’t worry; they are also working on another extended half-life FVIII in different preclinical trial.

One thing to note: you can actually voluteer to participate in one of these studies. Go to clinicaltrial.gov. You could help make history!

Great Book I Just Read
Take Yourself to the Top by Laura Fortgang

I read this book every two years at New Year’s to get motivated, to reasses and to plan. Not only for my business, but also for myself. This book has been around a while, but I love its no-nonsense approach, its friendly and cajoling tone, its practical applications for thinking bigger, laying the groundwork for success and removing all obstacles. I recommend it for anyone starting a business or starting a new passion within a business. First step, get off the caffeine. Man, it’s like she reads my mind in this book at times! Four stars.

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