Ion-ARPA’s focus and objective is the identification and acceleration of disruptive next-wave innovations with the potential to significantly advance the development of novel therapeutic technologies. Ion-ARPA programs will be funded (up to $1M per laboratory) based on high-risk/high-reward breakthrough scientific concepts. The program is designed to support the Ionis Innovation long-term strategy to create cutting-edge technologies capable of pioneering new markets and radically improve the standards of care for disease treatment.
The Ion-ARPA program has funded investigators at nearly 20 universities across the world including:
Harvard University/MGH
Johns Hopkins School of Medicine
UC San Diego (2 projects)
UC Santa Barbara
Queen Mary University of London
UC Riverside
University of Waterloo
University of Pennsylvania
University of Washington
Northwestern University
University of Massachusetts Amherst
UMass Chan Medical School
University of Delaware
University of Queensland
UC Santa Barbara
Technical University of Munich
University of Buckingham
“The Ion-ARPA program supports the dialogue to frame big problems as solvable, apply key resources, and encourages teams to meet biotechnology’s greatest challenges.”
- David Ecker
FUNDING ANNOUNCEMENTS
New Opportunity for 2026
Strategies for Increasing ASO On-Rate In Vivo
The therapeutic performance of antisense oligonucleotides (ASOs) in vivo is potentially limited not by equilibrium binding affinity, but by the kinetics of productive target engagement (on rate). Within cells, ASOs encounter a highly crowded and heterogeneous environment characterized by low effective target concentrations, extensive RNA secondary and tertiary structure, competition from abundant non-target RNAs, and restricted temporal access to relevant subcellular compartments. Despite its importance, ASO on-rate optimization remains underexplored relative to affinity and stability engineering, representing a potentially significant opportunity for innovation in antisense therapeutics. This request for proposals seeks novel ideas to increase the on-rate of ASOs.
Funding and Project Period Award Size: Up to $500,000 total direct and indirect costs per year for up to 2 years, renewable with successful progress.
Funding Structure: Awards are structured with defined milestones and continuation criteria. Second year funding may be contingent on satisfactory progress toward performance objectives.
Key Dates:
Program announcement: April 1, 2026
White papers due: July 1, 2026
Full proposals due: September 1, 2026
Award announcements: November 15, 2026
Funding start: Immediately following award announcement and finalizing contract
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 [email protected]. 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.
Intellectual Property
The awardees will retain ownership of intellectual property created during performance of the program. In exchange for its funding, Ionis will receive a paid-up non-exclusive license and first option to negotiate for an exclusive license.
2025 AREAS OF CONTINUED INTEREST
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 proteins similar to what is in the literature already.
We are only interested in funding completely novel approaches.
Previous Opportunities
Novel Ways to Regulate Gene Expression (June 2022) Investigators funded across 6 institutions. To be announced.
Payload Delivery (March 2022) – Investigators funded across 11 Institutions including: UC San Diego, Northwestern University, Scripps Research Institute, Massachusetts General Hospital, Harvard University, University of Massachusetts Amherst, UMass Chan Medical School, Johns Hopkins School of Medicine, University of Delaware, University of Queensland, Queen Mary University of London.
Quantum Biology (January 2021) – Investigators funded across 6 Institutions including: UC Riverside, UC Santa Barbara, University of Waterloo, Technical University of Munich, University of Pennsylvania, University of Buckingham.