Projects and pillars

Projects funded annually by the RNA Therapeutics Discovery and Translation Program include both pilot and standard awards:

March 2025 Awards

Project Name Award Type Principal Investigator(s) Adviser RNA Modality Focus Area
Validating Antisense Oligonucleotides as Potential Therapies for Progressive Supranuclear Palsy Pilot Xue Wang, Ph.D. Margot A. Cousin, Ph.D. Antisense oligonucleotides (ASO) Neurology
Development of Brain-Penetrant Biological Nanoparticles for In Vivo RNA Delivery Pilot Tsuneya Ikezu, M.D., Ph.D. Sai Yerneni, Ph.D. Messenger RNA (mRNA) Neurology
IL-1Ra-mRNA-Engineered Extracellular Vesicles for Treatment of Spinal Cord Injury With Modulation of Neuroinflammation and Neurodegeneration Pilot Wenchun Qu, M.D., Ph.D.; Ahad M. Siddiqui, Ph.D. Liewei Wang, M.D., Ph.D. mRNA Neurology
A Disease-Modifying Therapeutic for Parkinson's Disease Standard Pam J. McLean, Ph.D. Julie G. Allickson, Ph.D. Single guide RNA Neurology

May 2024 Awards

Project Name Award Type Principal Investigator(s) Adviser RNA Modality Focus Area
Aptamer Research Lab to Design and Build for Neuro-Research Targets Pilot Tsuneya Ikezu, M.D., Ph.D. Nilufer Ertekin-Taner, M.D., Ph.D. Aptamers Neurology
Building a Computational Framework to Accelerate Clinical Translation of RNA Therapeutics Pilot Daniel P. Wickland, Ph.D.; Xue Wang, Ph.D. Margot A. Cousin, Ph.D. ASO Neuroinformatics
Lipid Nanoparticles-Mediated Generation of CAR T-Cells and Silencing of Checkpoint Immune Receptors in Pancreatic Ductal Adenocarcinoma Pilot Dev Mukhopadhyay, Ph.D. Keith L. Knutson, Ph.D. Small Interfering RNA (siRNA) Cancer
Optimizing Targeted Cellular Delivery of RNA Therapeutics Standard Tushar C. Patel, M.B., Ch.B. Haidong Dong, M.D., Ph.D. Aptamers Cancer
Allogeneic APOE-Ch-Engineered Extracellular Vesicles for Treatment of Neurodegenerative Diseases Standard Abba C. Zubair, M.D., Ph.D.; Takahisa Kanekiyo, M.D., Ph.D. Nilufer Ertekin-Taner, M.D., Ph.D. mRNA Neurology
RNA Therapeutics for Cholangiocarcinoma Standard Tushar C. Patel, M.B., Ch.B. Liewei Wang, M.D., Ph.D. siRNA Cancer

Pillars

RNA strands circle in the middle, surrounded by the words 'discovery,' 'manufacturing,' 'commercialization' and 'clinical trials.' Outer circles represent talent, partnerships, knowledge and infrastructure. RNA Therapeutics Discovery and Translation Program Pillars.

The RNA Therapeutics Discovery and Translation Program moves new RNA-based treatments from early ideas to real-world impact. At the center is RNA science — the foundation of the program. Surrounding it is a continuous, circular process that shows how discoveries are developed, tested and ultimately delivered to patients.

The inner circle in the image above represents the lifecycle of an RNA therapy:

  • Discovery. Identify and design promising RNA based therapeutic ideas.
  • Manufacturing. Develop reliable ways to produce RNA therapies.
  • Commercialization. Prepare therapies to move beyond the lab and into broader use.
  • Clinical trials. Test safety and effectiveness in participants.

This cycle is ongoing, meaning insights from each step help continuously improve the next generation of RNA therapies.

Supporting this cycle are four key pillars shown around the outside of the image above:

  • Talent. Experts with the skills needed to discover, develop and advance RNA therapies.
  • Infrastructure. The technology, tools and facilities required for RNA research and manufacturing.
  • Knowledge. Data, scientific understanding and computational insights that guide smarter decisions.
  • Partnerships. Collaboration with academic, clinical and industry partners to accelerate progress.

Together, the cycle and the four pillars show how the program brings people, resources and expertise together to transform RNA discoveries into new treatments that can benefit patients.