Ionis Pharmaceuticals: The Ion-ARPA initiative- Novel Therapeutic Payloads (White papers)

The program seeks proposals that detail strategies to develop non-conventional technologies to modulate the expression of mammalian genes to overcome a disease state. The strategy could leverage inherent or synthetic molecular mechanisms. Approaches could be tunable, autoregulatory (e.g., respond to the context of the specific cells being treated), or controlled with a physical method (e.g., ultrasound or magnetic fields). General areas of interest include, but are not limited to, novel strategies for silencing or activating genes, targeted RNA modification, and gene editing beyond existing CRISPR/Cas9- or deaminasebased approaches. The scale of the approach can range from the modulation of a single gene to that of an entire chromosome.

Funding: Annual funding could range from seedlings of $300K to full programs of $1M per laboratory, with appropriate justification.

https://www.ionispharma.com/ionis-innovation/ion-arpa-initiative/