Arcturus Names TTR Amyloidosis as First Pipeline Program | GenomeWeb

RNAi startup Arcturus Therapeutics this week named transthyretin-mediated amyloidosis as the first indication to be added to its pipeline.

The disease is caused by mutations in the TTR gene, which triggers accumulation of abnormal amyloid proteins in the body. Notably, Alnylam Pharmaceuticals has been developing its own drugs for the disease, with an intravenously delivered one in phase III testing and a subcutaneously administered one in phase II.

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