Alnylam reveals RNAi therapeutics goals for 2013-14

RNAi specialist Alnylam Pharmaceuticals has outlined its research and development plans for the next 12 months.

Alnylam Pharmaceuticals, a leader in the field of RNA interference (RNAi) therapeutics, has revealed its main goals for the coming year.

Dr John Maraganore, the company's chief executive officer, revealed that the company made "tremendous" progress on its '5X15' pipeline in 2012.

This pipeline focuses on the development and commercialisation of novel RNAi therapeutics that address genetically defined diseases with a high degree of unmet medical need, such as haemophilia and severe hypercholesterolemia.

"The next couple of years promise to be transformative for Alnylam as we aim to advance our pipeline into phase-III, report on key clinical data in multiple programmes and expand the breadth of our '5x15' pipeline with additional clinical and pre-clinical programmes," Dr Maraganore revealed.

Giving further detail on the company's plans, he said that it is introducing a new development programme involving a product called ALN-AS1 for the treatment of an ultra-rare genetic disease called acute intermittent porphyria.

Dr Maraganore concluded that the company's ambitious goals for the coming year should help to advance innovative RNAi therapeutics and create value for its shareholders.

Barry Greene, Alnylam's president and chief operating officer, added that the company intends to directly sell its core '5x15' products in North and South America and Europe, while partnering to achieve market access in Japan and other Asian markets.