New Patient Registry Launches Aicardi-Goutières Syndrome Research

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.”

DEVON CORDOVA
AGSAA VICE PRESIDENT

“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.

Participant-centered Research

Digital Health Predictions: Participant-Centered Research Goes Mainstream


We’ve been hearing about patient-centered research for years. Yet while those of us in the industry have long been immersed in creating participant-centered research, the concept is still not mainstream. But that might be changing.

The challenges of the pandemic have propelled us to develop incredible advances in technology and clinical research. It has highlighted the importance of health data that reflects one’s lived experience. In addition, we’ve learned that we have to move quickly to adapt to these challenges. 

A Wave of Digital Clinical Trials

A recent survey conducted by Informa Pharma Intelligence found that 76 percent of professionals involved in clinical trials are now conducting digital, remote studies due to the pandemic. 

The most common steps researchers took? The adoption of patient-facing technologies, such as the use of wearables and mobile devices for data collection. 

Research doesn’t have to be a zero-sum game. We are now seeing a real acceleration of research efforts in which everyone has a seat at the table.”

Dawn Barry
PRESIDENT AND CO-FOUNDER OF LUNA

Trust and Transparency in Digital Health

In response to these creative solutions, we’re increasingly seeing both those in the industry and everyday people wanting transparency, trust, control, and privacy when it comes to individual health data. Study participants and researchers are asking questions and demanding that personal data be secure and controlled not by companies, but by the individual.

Even before the pandemic, patients have been concerned about their data. In June 2019, the Clinical Trials Transformation Initiative published a report about patient preferences on the use of mobile technologies in clinical research. Among the findings were that 80 percent of participants said it was important to have access to the information collected about them from the mobile technology used in a study. Respondents who were concerned about the security of their health data were, not surprisingly, less likely to join a mobile clinical trial. We believe that number would be even higher today.    

80% of study participants said it was important to have access to the information collected about them from the mobile technology used in a study.

Clinical Trials Transformation Initiative

All are Welcome to a Seat at the Table

From its start, Luna has been committed to safely and securely using health data to accelerate understanding and fuel insights to provide better health for individuals and families. We do this by transparently bringing together researchers, patient advocacy organizations, individual participants, and industry partners to answer important research questions. 

Our latest partnership helps families affected by mucopolysaccharidois type II (MPS II), an inherited disorder also known as Hunter syndrome. To make a difference in accelerating research into this rare genetic disease. families are joining the study, which is designed to reflect their needs, wants, and lifestyles.

Research doesn’t have to be a zero-sum game. We are now seeing a real acceleration of research efforts in which everyone has a seat at the table. What we’ve built at Luna can deliver better, faster, and more cost-effective research. We are amplifying the science by amplifying the opportunity for individuals. This is participant-centered research.

Digital Health Prediction for 2022

Two years of adapting to the pandemic have created opportunities for us to embrace the best of what technology has to offer and apply it to clinical research and the advancement of health. 

For 2022, Luna will bring into the mainstream the guiding principles that helped establish the organization in 2017:

  • We’ll bring participant-centered research into the mainstream, where individuals have a seat at the table and a voice in the research. They’ll control their health data. It’s secure. It’s private. It can be used for many studies that are important to them (not just collected for one research question).
  • We’ll continue to apply the best technology we have in our consumer world and apply it to health science, clinical research, genetic disorders, and rare diseases. 
  • We’ll bring greater focus on more cost-efficient, faster, and better research. 
  • We’ll unite industry and advocacy to help them accelerate and execute participant-centered research. 

This all will fuel partnerships, research, and ultimately insights to provide better health for families and individuals. 

Who’s ready to get started? 

To learn how Luna can help your organization bring together individuals, communities and researchers to produce powerful insights, contact us at collaborations@lunadna.com.


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.


Dawn Barry

Dawn Barry

PRESIDENT + CO-FOUNDER

Dawn is the president and co-founder of Luna. She is the former VP of Applied Genomics at Illumina and, for 12 years, built pioneering teams from health screening to nutritional security.


Close-up of Young Boy

Luna and Partners Work to Accelerate Therapies for Hunter Syndrome


We believe medical studies should be done in a truly collaborative way, where people living with the disease are treated as the experts, and researchers study the information streams that reflect the true lived experiences of the families.

Advocacy organizations—the National MPS Society and Genetic Alliance—partnered with Luna to achieve this goal. In early December, we started enrolling participants in a health information-sharing and medical-discovery digital community that begins in the homes of families living with Hunter syndrome. The program is called 100 Patient Project: Unlocking MPS.

As a father of two children with a rare genetic disease, my heart goes out to families living with Hunter syndrome. We are absolutely dedicated to changing the way individuals engage with research—and how research can partner with advocacy organizations.”

Joe Beery
CEO of Luna

This program demonstrates a study framework where all parties are better served while enhancing research goals. This design ensures the clinical and behavioral features and key endpoints are incorporated into the drug discovery process. This can only happen when families—those who live daily with the disease and know the lived experience best—have a seat at the table as partners.

In conjunction with Takeda Pharmaceutical Company, this partnership will advance the understanding of Hunter syndrome and help develop therapeutic interventions for patients with this rare genetic disease.

Participants will share what matters most to them about living with Hunter syndrome. The program provides an opportunity for individuals to engage with a genetic counselor and control the use of their data throughout the study. Access to these data streams, including whole-genome sequencing (WGS), medical history, and patient-generated health data, will form the basis for discovery and development.

Developing a Family-centered Study Design

“We are excited to work with Takeda in exactly our sweet spot. It is we who know these families and their affected children the best. They look to us for support and long-term solutions,” said Terri L. Klein, CNPM, president and CEO of the National MPS Society.

Klein said the organization is happy to be an integral partner in the study, which will engage and support patients with Hunter syndrome and their families throughout the process.

The National MPS Society and Genetic Alliance play a leading role in this program to ensure a family-centered study design–one that is consistent with their needs, wants and lifestyles. The study framework will allow for patients to participate virtually, which reduces the burden on families.

Dawn Barry
president and Co-Founder of Luna

“This allows greater participation from more diverse populations, expedites study recruitment, and amplifies the statistical power for discovery—creating unique benefits for both participants and researchers,” said Sharon Terry, president and CEO of Genetic Alliance. The nonprofit advocacy organization is providing engagement and regulatory expertise for the program. For more than three decades, it has developed and deployed tools that allow families to be in charge of their health to drive research.

Understanding Hunter Syndrome

An inherited disease that occurs almost exclusively in males, Hunter syndrome, also known as Mucopolysaccharidosis type II (MPS II), is a lysosomal storage disorder. It is caused by mutations in the iduronate 2-sulfatase (IDS) gene, which affects every organ of the body.

Conducting WGS will support better understanding Hunter syndrome’s natural history, disease heterogeneity, and the contribution of IDS mutations and genetic modifiers outside of IDS to disease presentation.

More than 600 IDS disease-causing mutations have been implicated in Hunter syndrome. While some mutation types are typically associated with neuronopathic disease and cognitive impairment, single-nucleotide mutations show variable association with other disease manifestations.

Although the age of onset, disease severity, and rate of progression of Hunter syndrome varies significantly, initial symptoms usually become apparent in children from two to four years of age. There is currently no cure for this condition.

“This project exemplifies the interdependency between research and the patients and families who we hope will one day benefit from potential breakthroughs that result from the program,” said Dan Curran, M.D., Head, Rare Genetics & Hematology Therapeutic Area Unit at Takeda. “We have had the privilege of working with the National MPS Society and the global Hunter syndrome community for many years as part of our ongoing commitment to delivering novel therapies, disease education and support resources to those affected by this rare lysosomal storage disorder. We are proud to partner together to create more patient-centered discovery programs.”

To learn how Luna can help your organization bring together individuals, communities and researchers to produce powerful insights, contact us at info@lunadna.com.


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.


LunaPBC Announces Joe Beery as New Chief Executive Officer


New Funding and Leadership Supports Operational and Commercial Scaling

SAN DIEGO, May 18, 2021 /PRNewswire/ — LunaPBC, manager of LunaDNA, a health data platform uniting people, communities, and researchers, has experienced rapid growth in adoption, as the demand from communities, researchers, and regulators for direct accounting and engagement of individual’s health and lived experience increases. The Company announces new executive leadership and funding to scale its platform and services.

The Company is excited to announce that Mr. Joe Beery has been named Chief Executive Officer of LunaPBC, Inc. effective today. Beery joins President and Co-founder, Dawn Barry, to lead the Company. Bob Kain, LunaPBC’s Co-founder who has served as Chief Executive Officer since Company formation in 2017, will remain active with the Company in the role of Executive Chairman of the Board.

“I am grateful to have had the role of CEO in building this Company,” said Bob Kain. “We have created a novel business model that enables our mission to provide a shared platform for patients, communities, and researchers looking for answers to improve clinical outcomes and quality of life. I am excited to move into a new role within the Company and support Joe in his transition to CEO.”

Beery brings industry-leading experience in operational and commercial scaling of innovative information technology systems, having served as the Chief Information Officer for four multibillion-dollar enterprises – America West Airlines, US Airways, Life Technologies, and Thermo Fisher Scientific. As CEO, Beery will draw on his expertise in strategic technology operations, digital and business transformations, cybersecurity, global M&A integrations, and disruptive innovation to grow the Company and scale LunaDNA technology.

“Joe has been a force in the development of scale in many industries, including at the vanguard in digitalization of the life and health sciences industries,” said Dr. Scott Kahn, LunaPBC’s Chief Information and Privacy Officer. “His deep understanding of digital transformation, scientific research, and therapy development make him an ideal choice to guide the growth of Luna as we continue to empower patients as partners in health science and keep them connected with ongoing research over time.”

With participation from more than 180 countries and communities advancing causes including disease-specific, public health, environmental, and emerging interests, LunaDNA technology empowers these collectives to gather a wide range of data –  health records, lived-experience, disease history, genomics, and more. LunaDNA technology gives academia and industry everything they need from engagement with study participants to data analysis in compliance with HIPAA, GDPR, and CCPA and across multiple modalities using a common data model. By providing privacy-protected individuals a way to continually engage, LunaDNA technology 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.

“In addition to Joe’s outstanding business experience, he brings a deeply personal and relevant life experience having had twin children born with a rare genetic disorder that was ultimately diagnosed and treated as a result of their family’s extraordinary activism for their kids,” said Dawn Barry, LunaPBC’s President. “Joe is also a champion for diversity and inclusion, drawing from his Hispanic heritage.”

Beery’s appointment coincides with the close of new funding. Key investors in this round include returning investors, ARCH Venture Partners, F-Prime Capital, and Osage University Partners.  John Tishler and Jason Jones of Sheppard Mullin Richter & Hampton LLP acted as legal counsel for LunaPBC in the financing. The funds will be used to accelerate our goal of digitally transforming health research and include expanding the software engineering and marketing teams, increasing community onboarding-to-discovery velocity, augmenting analysis and discovery tools, and enhancing user experience to optimize participant’s ability to share more health data and outcomes thus further enhancing the discovery potential.

“I am honored to be part of this exciting Company and team. Luna is at the forefront of efforts to digitally integrate individuals, communities, industry, and research,” said Joe Beery. “Luna’s platform and services are ready to scale and drive significant value to all of our customers. From both a personal and professional perspective, I see the alignment in both technology and services that Luna provides as key in accelerating the realization of discovery and patient outcomes. We are in a wonderful position to leverage and scale the unique capabilities built by the Company founders.”


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.


Bob Kain

LunaPBC’s CEO Bob Kain Named to Fast Company’s Annual List of Most Creative People in Business for 2020


We’re proud to announce that our Chief Executive Officer, Bob Kain, has been named to Fast Company’s annual list of the Most Creative People in Business for 2020.

This news comes just months after LunaPBC was named to Fast Company’s annual list of the Most Innovative Companies for 2020 in the Social Good category. Bob is recognized for his incredible achievements and significant impact in health tech, architecting Illumina’s HiSeq sequencing program, starting in 2008, and co-founding LunaDNA in 2017, the world’s first member-owned data-sharing platform for health research.

This year’s winning group features individuals working in artificial intelligence, winemaking, cybersecurity, television, underwater museum design, and more. It includes leaders from Patagonia, Amazon, Kaiser Permanente, Citi, and Google; and it spans across the globe, from China to Peru to Jordan to Burlington, Vermont.

“The Most Creative People in Business list offers a highly vetted, fully reported view of the powerful ideas and diverse leaders already shaping tomorrow,” said Jill Bernstein, Editorial Director at Fast Company.

Bob Kain
Bob Kain, CEO and Co-founder at LunaPBC

Bob Kain and His Creative Endeavors in Health Tech

Biotech executive, inventor, and entrepreneur, Bob is a renowned pioneer in genomics, dedicating most of his career building Illumina, Inc. pre-IPO from 30 employees with no revenue, to a burgeoning workforce of over 3,000 employees and $1.4B in revenue. In 2017, Bob came out of retirement following his 15-year career as the chief engineering officer at Illumina to co-found LunaPBC, the public benefit corporation behind LunaDNA. In between his career at Illumina and LunaPBC, Bob founded health and fitness business, Mesa Rim Climbing and Fitness Center, with multiple sites in San Diego, California, and Reno, Nevada.

“Innovation has always been hailed as a core value in business, and rightfully so. Every business leader will admit this powerful tool supports developing valuable technologies, designing disruptive products, and vastly improving the way products or services are delivered. But what seeds innovation? How might business leaders develop this skill to deliver truly impactful change? It starts with creativity,” Bob shares in his article, Creativity Seeds Innovation.

When applied appropriately, creativity is the secret sauce to improving organizational efficiency, decreasing costs, reducing risks, improving morale, and of course, seeding innovation. It is a crucial asset to an organization often left untapped. Fortunately, we all have the capacity to be creative in solving problems and carrying out work responsibilities.”

LunaDNA’s COVID-19 Study Program

As technology advances, so do creative health innovations, including the recent spike in biotechnology companies aiming to support biomedical research studies at scale. Amid the coronavirus pandemic, Bob believes there’s no better time to seed innovation than now. In March 2020, Bob helped launch LunaDNA’s COVID-19 Study, a program that offers people and communities the tools to share their lived experiences during this unique time, recognizing that each person, community, and geography is uniquely impacted and will likely experience different longterm health effects. LunaPBC is collaborating with various groups to support privacy-protected COVID-19 information sharing including Genetic Alliance and Disease InfoSearch, xCures, San Diego Blood Bank, and the Propionic Acidemia Foundation. Each study has a special focus, ranging from how the virus affects people with cancer, genetic disorders and co-morbidities, to capturing individual’s interest to join research studies.

Co-founders from Left to Right: CFO David Lewis, CEO Bob Kain, President Dawn Barry

LunaPBC focuses on supporting research to identify links between our biology and health, as well as surface other factors related to wellness, such as environmental and social influences. LunaDNA elevates people to partners in research with data privacy, control, and transparency, recognizing that the future of discovery requires big relationships over big data. Bob hopes that LunaDNA will bring data together at scale to fill the missing gaps in today’s databases.

We praise Bob for this incredible recognition and look forward to the additional creative innovations he’ll bring to health tech.

“I would like to thank all of my colleagues at Illumina and at LunaPBC for joining me on these journeys, and Fast Company for honoring me with 2020’s Most Creative People in Business Award. It’s been a fun ride.”


About LunaDNA and LunaPBC
LunaDNA makes discovery representative of the real world and aligned with people’s true goals by giving all individuals a role in research from right where they are. LunaDNA is a digital data-sharing community owned by its members. By sharing health information, you directly power disease research. As a member, your data is confidential. You control the information you share — only one copy of your data exists and you always control its inclusion in LunaDNA. We make it simple for all credible researchers to pursue health and quality of life discovery. LunaDNA is managed by Public Benefit Corporation, LunaPBC, founded in 2017 and headquartered in San Diego, California. The LunaPBC team, investors, and advisors are renowned in the patient-advocacy, health, and science fields, including several former chief executives of Illumina, industry academics, and financial executives.

About Fast Company
Fast Company is the only media brand fully dedicated to the vital intersection of business, innovation, and design, engaging the most influential leaders, companies and thinkers on the future of business. Since 2011, Fast Company has received some of the most prestigious editorial and design accolades, including the American Society of Magazine Editors (ASME) National Magazine Award for “Magazine Of The Year,” Adweek’s Hot List for “Hottest Business Publication,” and six gold medals and 10 silver medals from the Society of Publication Designers. The editor-in-chief is Stephanie Mehta and the publisher is Amanda Smith. Headquartered in New York City,Fast Companyis published by Mansueto Ventures LLC, along with our sister publication Inc., and can be found online at fastcompany.com.

For more information visit fastcompany.com.