Meet the Newest Player in Hemophilia Pharma: Alnylam

For those with the chronic condition hemophilia, more medication options should be on the way. Meet Alynlam: Alnylam’s genetic medicine programs are RNAi therapeutics directed toward genetically defined targets for the treatment of serious, life-threatening diseases with limited treatment options for patients and their caregivers.

“Hemophilia and other rare bleeding disorders are characterized by deficiencies in specific clotting factors that ultimately lead to inadequate thrombin generation and a bleeding diathesis. ALN-AT3 is aimed at correcting these bleeding disorders by knockdown of AT – an endogenous anticoagulant – thus, increasing thrombin generation and improving hemostasis,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam.

According to the Wall Street Journal, Alnylam recently announced that it expects to exceed its original “Alnylam 5×15” guidance with six to seven genetic medicine programs in clinical development by the end of 2015, including at least two programs in Phase 3 and five to six programs that will have achieved human proof-of-concept results supporting further development.

“The unmet need for new therapeutic options to treat people with hemophilia remains very high, particularly in those patients that develop inhibitory antibodies to their replacement factor. Indeed, availability of a safe and effective subcutaneously administered therapeutic with a long duration of action would represent a marked improvement over currently available approaches for prophylaxis,” said Claude Negrier, M.D., head of the Hematology Department and director of the Haemophilia Comprehensive Care Centre at Edouard Herriot University Hospital in Lyon.

To read a prior article about Alnylam from December 2013, click here.

Photo credit-Alnylam Pharmaceuticals, Inc.

webleed.org – your source for bleeding news!