The Foundation for Children with Atypical HUS

New drug patented that targets Factor H mutations

Has anyone heard about the new drug TT30? I just came across this link:

http://www.businesswire.com/news/home/20100823005362/en

It basically says that a company called Taligen has been granted a patent for a new drug, TT30, that targets Complement Factor H.

I think this is good news because it gives us one more line of treatment apart from Soliris. It may be a few years before it even starts to be used however.

Kamal

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Comment by Colette Ann Frysz on September 1, 2010 at 8:37am
Kamal,
Yes I ran across the drug you mentioned for the Factor H. I believe there is a company in France that is working on the factor h replacement drug. They from what I read are in the beginnings of the prcess. But it is also very promising. I ran across the site by just googling factor H.
Comment by Cheryl Biermann on August 25, 2010 at 4:02pm
Hi all.

Yes, we were in contact with Taligen as recently as maybe 2 1/2 years ago. We did some work for them regarding patient's genetic testing. They were quite excited to get the information, although we were no where near done when the news about Soliris came out. Based on that information, they decided to take a "wait and see" attitude and although there have been rumors floating around, this is the first concrete evidence we have had since that contact. They are a really reputable company and we enjoyed working with them for the short time we did.

I'm sure Bill will be looking at the new information, (he's our main research decipherer here in our house, I'm head nurse, teacher, liason and bottle washer!), and then I'll eventually get around to it, after he deciphers it first, and interprets for me!

I can't believe after all these years nothing-and seemingly over night all the advancements. It really is all exciting!
Comment by Phyllis Ann Talbot on August 25, 2010 at 2:11pm
Hi there Kamal - I know that Bill Biermann had been in contact with Taligen over the last few years regarding this - but hadn't heard anything from him lately - maybe he or Cheryl will chime in? Thanks!

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Did you know...

CFH (Serum Complement Factor H) is a regulatory protein. The secreted protein product of CFH consists of 20 repetitive units named "short consensus repeats" or SCRs (each approximately 60 amino acids). In patients with aHUS the last 5 "pearls" in the twenty pearl strand protein, SCR16 - SCR20, should bind to protect cells but do not- they are defective in one or more of the last 5 SCR locations. If they cannot bind or stick to the kidney to protect that tissue, the platelets clump into clots that affect the glomeruli of the kidney -potentially causing acute renal failure.
  
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It is estimated that there are about 300 cases of aHUS in the U.S., and it is most common with young children. The condition is life threatening and either can be chronic or can recur at intervals.
  
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