Arcturus Therapeutics, a leading RNA medicines company pursuing orphan diseases, announced it will present a poster at the 10th Annual Meeting of the Oligonucleotide Therapeutics Society on “Exploring Allele-selective siRNAs for Human Transthyretin” (poster number 130) being held October 12-15, 2014 in San Diego, California.

“Arcturus is developing best-in-class RNA medicines using its therapeutic delivery platform Lunar and UNA chemistry.  Data demonstrates this promising approach for treating TTR-mediated diseases including allele-selective silencing of the V30M variant,” said Dr. Chivukula. “We believe that the Arcturus platform has broad applications for RNA medicines including breakthrough advances in autosomal dominant disorders.”

Key data presented includes, Lunar-101, an UNA oligomer therapeutic targeting transthyretin for the treatment of TTR-mediated cardiomyopathy, demonstrated a 90 per cent reduction in TTR at day 10 and continued reduction at day 20 of 91 per cent in non-human primates (n = 3, up to 94 per cent after a single low dose of only 0.3 mg/kg, The clinical dosing for Lunar-101 will be investigated with once-monthly intravenous administration coinciding with the patient’s normal physician visit.

Lunar-102, an UNA oligomer therapeutic targeting transthyretin for the treatment of TTR-mediated polyneuropathy, demonstrated in vitro allelic silencing greater than 100-fold for the V30M variant over wild-type allele. Lunar delivery technology continues to be safe and well-tolerated; a single high dose of 50 mg/kg in rodents shows no adverse events. Lunar delivery and UNA oligomer chemistry provide an exciting new approach to treat autosomal dominant diseases where targeting the mutant variant, but not the wild-type allele, is critical for optimal clinical outcomes.

Transthyretin (TTR)-mediated amyloidosis is a genetically mediated fatal disease caused by mutations in the TTR gene. Mutated TTR, which is mainly synthesised in the liver, causes errant amyloid proteins to aggregate and deposit, destroying body organs and tissue, such as the peripheral nerves and heart.  TTR-mediated familial amyloid cardiomyopathy (TTR-FAC) affects approximately 40,000 people with the mean survival of 2.5 years. TTR-mediated familial amyloid polyneuropathy (TTR-FAP) affects at least 10,000 people.  There is a substantial unmet need for effective medicines for patients suffering from TTR-mediated diseases.