A new patient-led drug discovery community aims to study a rare neurodevelopmental disorder called Aicardi-Goutières syndrome (AGS) and support the development of therapeutic treatments for this devastating disease.

Luna, in partnership with Aicardi-Goutières Syndrome Americas Association (AGSAA) and Genetic Alliance, is working to assemble the program in collaboration with Biogen, Inc.

Learn about the AGS Patient Registry.

The program will engage patients and their families, patient advocates, and pharmaceutical partners to create a patient-driven trial design— with patients and their parents as the stewards of their data—with the goal of incorporating the optimal clinical and behavioral features and the most desired trial endpoints in the drug discovery process. Luna’s proprietary Community-Driven Innovation technology will align families and researchers on their key priorities and needs.

Using Community-Driven Innovation technology

“Luna is excited for Biogen and AGSAA to use our Community-Driven Innovation (CDI) methodology to create an actionable, living data set that supports communities that use it,” said Dawn Barry, Luna president and co-founder. “By pairing how people think and live with empirical data, CDI can guide where to spend energy to answer people’s priorities, surface new research opportunities to develop interventions, and discover where existing products can help individuals.”

Luna’s proprietary CDI technology will align families and researchers on their key priorities and needs. The AGSAA will administer the patient registry, with input from partners in academia, the clinic, and industry.

With committed community engagement and thoughtful stewardship of our data, we will finally have what we need to tell our story and begin to write a different ending.”


“Over my 26 years in advocacy, I have worked with hundreds of advocacy organizations, maybe thousands. Working with the passionate parents at AGSAA is an exciting experience for me,” said Sharon Terry, Genetic Alliance CEO. “We showed them a rocket ship, and they jumped in and took the controls. Their capacity to advance their cause faciley and quickly has both astounded and moved me. I love their passion, their savviness, and clarity of purpose. These parents will make a tremendous difference.”

New program provides power to share research

The program gives the AGS community the power to shape clinical studies that have the potential to answer their needs more quickly and efficiently. This framework, constructed from deep dives into AGS’s digital world and real-world interviews with parents and patients, helps guide researchers toward the most accurate observations and endpoints that will be built into the study design to increase participant engagement and retention, to ensure interventions meet the goals and needs of patients, to capture real-world and patient-reported insights, and to facilitate comprehensive, longitudinal study.

The framework also enables children and their parents to participate in studies from the comfort and safety of their homes, thereby reducing the hardship on families, increasing participation, and expediting research.

“This partnership represents an incredible opportunity to not only document and validate the lived experiences of families grappling with Aicardi-Goutières syndrome, but actually use these hard-won insights to shape the future of research and treatment options,” said Devon Cordova, AGSAA vice president. “With committed community engagement and thoughtful stewardship of our data, we will finally have what we need to tell our story and begin to write a different ending.”

Learn more about how to determine eligibility to participate and join the AGS study.

What is Aicardi-Goutières syndrome?

AGS primarily affects the developing brain and immune system of infants and toddlers, most often resulting in profound developmental delays, lifelong physical impairments, and persistent neurological changes. Most newborns with AGS do not display any signs or symptoms at birth but go on to develop severe brain dysfunction within the first two years of life, often after months of typical development and good health. In AGS, the body’s immune system turns on itself in a destructive way, targeting myelin, or white matter, in the brain and significantly impacting the nervous system.

Additionally, immune dysfunction associated with AGS can affect many other organs of the body, sometimes in a life-threatening manner. This can include the lungs, the liver, the heart, the skin, blood cells, and the kidneys. Because the signs and symptoms of the disorder are similar to those of a congenital viral infection, AGS is extremely difficult to diagnose.

Although rare, increasing awareness of AGS has revealed higher prevalence than previously ascertained. In order to manage severe progression and improve quality of life for affected individuals and their families, both early identification and timely access to emerging treatments are essential interventions.

About Luna

Luna’s suite of tools and services connects communities with researchers to accelerate health discoveries. With participation from more than 180 countries and communities advancing causes including disease-specific, public health, environmental, and emerging interests, Luna empowers these collectives to gather a wide range of data—health records, lived experience, disease history, genomics, and more—for research.

Luna gives academia and industry everything they need from engagement with study participants to data analysis across multiple modalities using a common data model. The platform is compliant with clinical regulatory requirements and international consumer data privacy laws.

By providing privacy-protected individuals a way to continually engage, Luna transforms the traditional patient-disconnected database into a dynamic, longitudinal discovery environment where researchers, industry, and community leaders can leverage a range of tools to surface insights and trends, study disease natural history and biomarkers, and enroll in clinical studies and trials.