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Arcturus Adds Second ATTR Program to Pipeline


NEW YORK (GenomeWeb) – Arcturus Therapeutics has announced that it has added a second drug candidate to its transthyretin-mediated amyloidosis (ATTR) program.

ATTR disease is caused by mutations in the transthyretin (TTR) gene, which triggers the accumulation of abnormal amyloid proteins in the body. Previously, Arcturus announced that it is developing an ATTR drug designed to silence both the wild-type and mutant forms of the gene, which would be used for patients with a form of ATTR that primarily damages heart tissue called familial amyloidotic cardiomyopathy (FAC).

This week, the company announced new preclinical data showing that its RNAi molecules, which incorporate proprietary unlocked nucleobase analog modifications, are capable of silencing mutant TTR while leaving the wild-type form intact.

Based on these data, Arcturus has added a mutant allele-specific ATTR candidate to its drug-development roster. According to a company official, the new compound will be developed for another form of ATTR called familial amyloidotic polyneuropathy, or FAP, which is characterized by damage to the peripheral nervous system.

Arcturus has previously stated that its FAC drug is slated to enter the clinic in early 2015.