CA17103 - Delivery of Antisense RNA Therapeutics (DARTER)

Antisense oligonucleotides (ASOs) are a new class of drugs that, through very specific targeting, could correct genetic defects for rare inherited diseases, modulate autoimmune or neurodegenerative diseases or target tumors or viruses. However, only a few of such drugs are currently in the market and they have been less effective as expected. The main hurdle for their efficacy seems to be their deficient delivery to target tissues but, while translational research on ASO is surging, very little is known about the mechanisms by which ASOs are taken up by different tissues and specific cells.

Regarding delivery, the ASO field is fragmented, with researchers in academia and industry working in isolation on specific diseases, generally focusing on therapeutic effects in target tissues. The main aim of the Delivery of Antisense RNA ThERapeutics (DARTER) Action is to use networking and capacity building in the field of nucleic acid therapy delivery to allow RNA-targeting nucleic acid drugs to reach their full potential and become a mainstream therapeutic option.

DARTER will act through  3 Working Groups with research objectives  (delivery strategies, model systems, safety and toxicology) and one capacity building group (stakeholder communication) with the objective of achieving consensus on protocols and assessment of ASO delivery and toxicology and training new researchers within a cooperative research framework. DARTER COST network will contain participants from COST countries and several international partners, including academics, industrial partners, patient representatives and clinicians and it is open to other interested stakeholders.