Accelerating Rare Disease Research Through Collaboration: Global Genes and Notre Dame Launch the Research Acceleration Program

Global Genes and University of Notre Dame have partnered to launch the Research Acceleration Program, a collaborative initiative designed to accelerate progress in rare disease research and clinical trial readiness. This new effort strengthens Global Genes’ commitment to empowering patient advocacy groups (PAGs) with the knowledge, tools, and connections needed to advance treatments for rare diseases.

Advancing Research Readiness Across the Rare Disease Ecosystem

The Research Acceleration Program was created to help rare disease communities identify and strategically address the barriers that often slow progress toward therapies. By applying Global Genes’ Research Acceleration Framework, the program supports communities in evaluating the current state of  research and clinical trial preparation across four key domains:

1. Disease Biology Characterization: Defining the genetic and biological basis of disease, including gene function and variant effects, and developing relevant laboratory models.
2. Therapeutic Potential:Identifying druggable targets, assessing safety, and evaluating potential therapeutic modalities such as gene therapy or antisense oligonucleotides.
3. Clinical Trial Readiness: Establishing biomarkers, defining meaningful endpoints, and building clinical networks capable of supporting high-quality, multicenter trials.
4. Patient Population & Engagement: Strengthening registries, understanding the diagnostic journey, and integrating patient priorities and quality-of-life data into research design.

By systematically assessing these areas, the program helps patient organizations and research teams pinpoint what’s needed to move from discovery to development and ultimately, to meaningful, well-designed clinical trials.

Bridging Science, Strategy, and Advocacy

Through this collaboration, Global Genes and Notre Dame are bringing together the rare disease community, academic experts, and industry partners to close critical knowledge and infrastructure gaps. The program facilitates the exchange of insights between researchers and patient groups, ensuring that scientific priorities are grounded in community needs and that patient voices are integrated from the earliest stages of research planning.

Students enrolled in Notre Dame’s Minor in Science and Patient Advocacy program contribute by helping to aggregate the scientific literature and map readiness levels across research domains.. Their work strengthens the bridge between academic training and rare disease communities.

The Global Genes team utilizes both internal expertise and external scientific guidance to take the initial readiness assessment from the published literature and extend it to include current and planned research projects. Then, the team works with a group of highly knowledgeable clinicians, laboratory researchers, advocacy leaders, and industry representatives to align and prioritize the resulting gaps with a list of projects that enable each patient advocacy organization to develop a strategic research plan. 

Empowering Communities to Accelerate Progress

At its core, the Research Acceleration Program is about empowering communities to take actionable steps to accelerate therapeutic research, reducing the time to launch  well-designed clinical trials. Many PAGs are eager to advance scientific discovery but lack access to the tools, frameworks, or expert networks needed to translate ambition into progress. By providing structured guidance and fostering collaboration among stakeholders, the Research Acceleration Program pushes for faster, more equitable access to innovation for rare disease patients. By combining Global Genes’ leadership in patient advocacy and patient-driven research with Notre Dame’s academic excellence, the partnership aims to shorten the path from diagnosis to discovery to treatment.