TTR Amyloidosis
ALN-TTR: TTR Amyloidosis
We are advancing ALN-TTR, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR amyloidosis, as one of our key development programs. Data from pre-clinical studies showed the potential therapeutic benefit of an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR amyloidosis, including familial amyloidotic polyneuropathy (FAP). The new data demonstrated that highly potent RNAi therapeutics targeting TTR dramatically reduced the levels of target messenger RNA (mRNA) in the liver and TTR protein in circulation. For most patients with TTR amyloidosis, liver transplantation is the only treatment option. These data suggest that treatment of TTR amyloidosis with an RNAi therapeutic may represent a promising alternative medical strategy, including the ability to simultaneously reduce the expression of mutant as well as wild-type TTR.
ALN-TTR Clinical Timeline

