Visit Capella to learn more about our "Alnylam 5x15" Strategy and the programs we are developing addressing genetically defined diseases with significant unmet medical need.
Alnylam 5x15 Programs
In January 2011, we launched our "Alnylam 5x15™" product strategy focused on the development of innovative RNAi therapeutics for genetically defined diseases addressing major unmet medical needs.
The "Alnylam 5x15" strategy, launched in January 2011, establishes a path for development and commercialization of novel RNAi therapeutics toward genetically defined targets for the treatment of diseases with high unmet medical need.
Products arising from this initiative share several critical characteristics including: a genetically defined target and disease; the potential to have a major impact in a high unmet need population; the ability to leverage the existing Alnylam delivery platform; the opportunity to monitor an early biomarker in Phase I trials for human proof of concept; and the existence of clinically relevant endpoints for the filing of a new drug application (NDA) with a focused patient database and possible accelerated path for commercialization.
By the end of 2015, we expect to have five such RNAi therapeutic programs in clinical development, including programs in advanced stages, on its own or with a partner. The "Alnylam 5x15" programs include: ALN-TTR02, an intravenously delivered RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR) in patients with familial amyloidotic polyneuropathy (FAP); ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting TTR for the treatment of ATTR in patients with familial amyloidotic cardiomyopathy (FAC); ALN-AT3 for the treatment of hemophilia and rare bleeding disorders (RBD); ALN-PCS for the treatment of hypercholesterolemia; ALN-AS1 for the treatment of acute intermittent porphyria (AIP); ALN-TMP for the treatment of treatment of beta-thalassemia and iron-overload disorders; ALN-CC5 for the treatment of complement-mediated diseases, and other programs.
Alnylam intends to focus on developing and commercializing certain programs from this product strategy itself in North and South America, Europe, and other parts of the world; these include ALN-TTR, ALN-AT3, ALN-AS1, and ALN-CC5.