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Internet Buzzes About Alnylam Pharmaceuticals And Genzyme Alliance To Develop And Commercialize For Treatment Of Amyloidosis

October 22, 2012

        Alnylam Pharmaceuticals and Genzyme announce on October 22, 2012, an alliance to develop and commercialize RNAi therapeutics targeting transthyretin (TTR) for treatment of transthyretin-mediated amyloidosis (ATTR) in Japan and other Asia-Pacific countries. 

        The Internet buzzes about this new alliance : 

1)  Alnylam Pharmaceuticals October 22, 2012 News Release : 

     –  Alnylam to receive $22.5 million in upfront payment

     –  Alnylam to receive milestone payments & royalties on product sales

     –  Alnylam maintains all rights in US, EU, and Rest of World (ROW). 

2)  FiercePharma.com October 22, 2012 article titled,”Genzyme pays $22.5M upfront to bag Asian rights to Alnylam’s hot ATTR program”  

3)  BostonGlobe.com October 22, 2012 article titled,”Alnylam, Genzyme form alliance”  

4)  RNAitherapeutics.blogspot.com October 22, 2012 article titled,”Genzyme Taps RNAi Therapeutics for TTR Amyloidosis”.   

Transthyretin-Mediated Amyloidosis (ATTR) rare disease facts : 

–      Inherited disease is caused by TTR gene mutations

–      TTR protein is produced mainly in liver & is carrier of thyroid hormones & retinol binding proteins

–       TTR mutation causes accumulation of bad amyloid proteins, which damages body organs and tissue

–       FAP (familial amyloidotic polyneuropathy) affects about 10,000 worldwide

–       FAC (familial amyloidotic cardioiomyopathy) affects > 40,000 worldwide.       

ALN-TTR02 facts are as follows :

–      FDA Orphan Drug Designation (ODD) on June 14, 2012 for treatment of familial amyloidotic polyneuropathy

–      Phase I positive clinical data is presented July 16, 2012, at Boston University School of Medicine – Alnylam Pharmaceuticals July 16, 2016 News Release.

–      Significant results show that ALN-TTR02 “leads to robust knockdown of serum TTR protein levels of up to 94%”, Alnylam Pharmaceuticals July 16, 2016 News Release

–      “Suppression of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, durable, and specific after just a single dose”, Alnylam Pharmaceuticals July 16, 2016 News Release

References 

Alnylam Pharmaceuticals Pipeline  

Amyloidosis Foundation  

Amyloidosis Support Groups

Copyright  © 2012-2013, Ann-Teresa Cusenza and Orphan Druganaut Blog.  All rights reserved.

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