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

Disease Info

TTR amyloidosis is a hereditary, systemic disease caused by a mutation in the transthyretin (TTR) gene. TTR protein is produced primarily in the liver and is normally a carrier for thyroid hormones and retinol binding proteins. The mutation causes abnormal amyloid proteins to accumulate in and damage body organs and tissue such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and cardiomyopathy. In its severest form, TTR amyloidosis presents a very large unmet medical need with significant morbidity and mortality as an orphan disease; FAP (familial amyloidotic polyneuropathy) affects approximately 10,000 people worldwide with additional patients affected by FAC (familial cardiac amyloidosis). TTR amyloidosis patients with FAP have a mean life expectancy of nine to eleven years from symptom onset and the only treatment option is liver transplantation; as a result there is a significant need for novel therapeutics to treat patients who have a mutation in the TTR gene.