Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California that specialises in discovering and developing RNA-targeted therapeutics.

It’s aim is to identify next-wave technologies with the potential to accelerate the creation of therapeutic technologies through disruptive innovation. The programme is modeled on the U.S. Department of Defense programme known as DARPA (Defense Advanced Research Projects Agency). Ion-ARPA programmes will be funded based on high-risk ideas with big payoff potential.

Programmes are intended to run for two years to test bold ideas, with the potential to continue if encouraging results are obtained.

Proposers are encouraged to solicit a white paper in advance of submitting a full proposal. White papers that propose programmes outside the scope of RFP's will be considered on a rolling basis.

Area of interest #1: New delivery strategies that enable delivery of large nucleic acid payloads to the CNS

There are many opportunities to treat diseases in the CNS, but methods to deliver large nucleic acids to appropriate cells in the CNS are limiting. We are seeking proposals for unconventional approaches (excluding AAV or other live viruses, and beyond the ideas currently in the literature) to deliver large therapeutic payloads to cells of the CNS.

Area of interest #2: New strategies for regulating the epigenome for therapeutics

Epigenome editing has emerged as a therapeutic strategy to regulate gene expression without creating nicks or breaks in the DNA. We are interested in funding novel strategies to provide long lasting epigenome modulation to increase or decrease expression of gene products. We are not interested in using replicating viruses (e.g. AAV), editing RNA or ideas based on dead Cas similar to what is in the literature already. We are only interested in funding completely novel approaches.

If you are interested in submitting a proposal, please submit a white paper (5 pages maximum) that succinctly describes your idea. Use the standard white paper template and submit to Ion-ARPA(ELIMINAR)@ionisph.com. If you would like to discuss your concept in advance of submitting a white paper, please reach out to us for a pre-white paper discussion at the same address.

In case of interest, please click on "m'interessa" and we will contact you.