Murdoch Children’s Research Institute Support the future of child health. For all. Forever.
Our purpose
To give all children the opportunity to live a healthy and fulfilled life. About MCRI Our story began in 1986 when visionary philanthropist Dame Elisabeth Murdoch AC DBE and paediatrician and genetics pioneer Professor David Danks AO imagined a better future for children. Now, Murdoch Children’s Research Institute (MCRI) is Australia’s largest medical research institute and a global leader in child health research.
Our global impact at-a-glance: • Ranked in the top three child health research institutes globally for research quality and impact • Over 1,900 world-leading researchers working across more than 150 diseases and in collaboration with over 120 countries • Publications cited six times more than the global average • Philanthropic investment leveraged ten-fold across all programs • Located at the epicentre of a world-renowned biomedical precinct, including more than 48,000 employees across over 40 organisations, in Melbourne, Australia 2
Murdoch Children’s Research Institute
• Part of the International Precision Child Health Partnership (IPCHiP) to advance genomics in precision care across Australia, Canada, the United Kingdom (UK) and the United States (US) • Host of four World Health Organization Collaborating Centres • More than 7,000 publications with US collaborators between 2020-2025 • Over 1,500 US media mentions reaching a US audience of 11.4 billion people in 2024-2025.
Complexities of child health in Australia and the US Despite extraordinary advances in medicine, children continue to face significant health challenges.
One in three are overweight or obese.
One in four start school developmentally vulnerable.
One in five experience high psychological distress.
One in six live in poverty.
One in 10 live with chronic conditions such as asthma and allergies.
3
Transforming lives At MCRI, our goal is to solve the challenges facing children and the adults they will become – in our hospitals, in our community and as global citizens. To do this, our expertise spans five core themes:
Genomic Medicine
Stem Cell Medicine
Clinical Sciences
Harnessing genomic technologies for families affected by genetic disorders.
Developing a new generation of transformative stem cellbased therapies.
Accelerating discoveries into clinical care.
Population Health
Infection, Immunity and Global Health
Advancing policy and practice for equitable healthy populations.
4
Murdoch Children’s Research Institute
Addressing childhood infections and inequality through disease prevention, strong health systems and health education.
Our world-leading research breakthroughs MCRI has combined scientific excellence, clinical care and global collaboration to tackle some of the most significant challenges in child health. Across four decades, our research has helped: • Uncover a life-changing dwarfism treatment • Pioneer growing mini kidneys from stem cells • Discover human rotavirus, leading to vaccine development • Engineer world-first blood stem cells closely resembling those in the human body • Spearhead concussion research and national guidelines • Introduce peanut allergy oral immunotherapy as standard care for pre-schoolers
• Develop an AI tool to detect tiny brain lesions that cause severe epilepsy • Reduce scabies prevalence on a national scale • Shape policy and clinical care nationally and internationally • Recruit up to 200,000 participants to support over 40 studies spanning birth to adulthood. Now, MCRI is poised to lead the next generation of breakthroughs in child health research.
“No matter where a child is born, Murdoch Children’s Research Institute can change the course of their life for the positive. Through funding their work, together we can fundamentally change the lives of children everywhere.” MCRI Co-chair and Global Advisory Board Chair Sarah Murdoch 5
MCRI – US partnerships for impact Our partnerships across the US connect worldleading Australian child health research with global expertise, technology and philanthropic support. Together with leading hospitals, universities, research institutes, biotechnology companies and community partners, we are accelerating discoveries to improve child health worldwide. These are just some of our high-impact collaborations.
6
Murdoch Children’s Research Institute
State
Partner
Research lead
Collaboration
California
Anthropic
Professor Enzo Porrello
Accelerating discoveries into genetic and childhood heart diseases
California
G’Day USA Arts Gala
Professor Enzo Porrello
A celebration of Australian excellence across the arts and sciences
California
Gladstone Institutes
Professor Enzo Porrello
Decoding Broken Hearts
California
Retro Biosciences
Associate Professor Elizabeth Ng
New US$35m partnership to advance blood disorder therapies
Massachusetts
Boston Children’s Hospital
Professor Kathryn North AC
International Precision Child Health Partnerships
Massachusetts
Boston Children’s Hospital
Professor John Christodoulou KIF1A-Associated Neurological Disorder (KAND) research program
Massachusetts
Boston Children’s Hospital
Associate Professor Katherine Howell
Rapid genome sequencing highly effective at diagnosing babies with epilepsy
Massachusetts
Broad Institute
Professor Daniel MacArthur
Genome Aggregation Database (gnomAD) development and extension
Massachusetts
Broad Institute and Microsoft Research
Professor Zornitza Stark
Genomic tool highly effective at detecting rare diseases
New York
American Australian Association
Professor Sharon Goldfeld
2025 President’s Lunch hosted by AAA
New York
American Australian Association
Professor Enzo Porrello
Old mates come together for MCRI
New York
Goldman Sachs
Professor Enzo Porrello
Friends of MCRI
New York
KIFA1A.org and FOX Nation
Dr Simranpreet Kaur
Hope Starts Here documentary
New York
Mount Sinai
Professor Kirsten Perrett
National Allergy Centre of Excellence
Ohio
Cincinnati Children’s Hospital Professor Andrew Steer
Strep A vaccine development
Ohio
Cincinnati Children’s Hospital Professor Ravi Savarirayan
Oral treatment for dwarfism on cusp of replacing injections
Pennsylvania
Children’s Hospital of Philadelphia
Dr Nicole Van Bergen
Vitamin B3 treatment may halt severity of rare genetic disease
Texas
Texas A&M University
Dr Jessica Kaufman
Antenatal vaccine communication model 7
Hear from some of our US partners “Goldman Sachs is incredibly proud of our relationship with Murdoch Children’s Research Institute and to support the important work they are doing to improve the health and wellbeing of children and families. We’re proud to help MCRI build its presence and community here in the US and look forward to continuing to support their global impact.” Goldman Sachs Head of Infrastructure, Global Banking & Markets Private and MCRI Global Advisory Board member Jodie Relton
“The American Australian “Australian science has a history Association is proud to partner of being at the leading edge with MCRI, connecting worldof genetics, bioinformatics and leading Australian research with domains where emerging AI new opportunities across the capabilities are poised for realUnited States. Together, we world impact. It’s incredibly can accelerate discoveries that exciting to see this expansion improve children’s health and of our AI for Science program transform lives around the world.” to accelerate fundamental and clinical discoveries.” American Australian Association President Steven Marshall AO Anthropic Head of Life Sciences Jonah Cool “For decades, it’s been a dream of the stem cell field to convert pluripotent stem cells into blood stem cells that engraft permanently and produce all the needed blood lineages to maintain a person’s health. The principal investigators at MCRI finally cracked this code, and we immediately saw the potential for sustaining a healthy blood system into late life. Our team at Retro is incredibly excited to be the exclusive licensee for this new generation of autologous therapies.” Retro Biosciences Co-founder and CEO Joe Betts-LaCroix
Left: MCRI Stem Cell Medicine Theme Director Enzo Porello (centre) with MCRI Global Advisory Board members Nicholas Stone (left) and David Calvert-Jones (right) at Friends of MCRI 2025 supported by Goldman Sachs, Formstack and Asensa Advisors at Old Mates, New York. Picture: Jeff Neira 8
Murdoch Children’s Research Institute
"Working alongside Professor Andrew Steer and MCRI, together with our colleagues at the Uganda Heart Institute, has allowed the GOAL studies to generate evidence that is directly changing the trajectory of rheumatic heart disease for children in Uganda and beyond. This partnership shows what's possible when institutions commit to long-term, locallygrounded research together.” Cincinnati Children’s Hospital Pediatric Cardiologist Professor Andrea Beaton
“Our collaboration with MCRI is bringing us closer to earlier diagnoses, better treatments and healthier futures for children and families facing these conditions. I'm truly grateful for your partnership.” Gladstone Institutes Assistant Professor Christina Theodoris
Right: MCRI Director Professor Kathryn North AC and Retro Biosciences Co-founder and CEO Joe Betts-LaCroix. 9
Hear from some of our researchers “Boston Children’s Hospital is a key collaborator with MCRI through the International Precision Child Health Partnership - the first major global initiative focused on advancing genomics in paediatric care. As a key IPCHiP partner, BCH adds invaluable capacity in terms of patient cohorts and expertise.” MCRI Director Professor Kathryn North AC
Above: MCRI Director Professor Kathryn North AC. Opposite top: MCRI Infection, Immunity and Global Health Theme Director Professor Andrew Steer. 10
Murdoch Children’s Research Institute
“The Decoding Broken Heart research program, as a collaboration between MCRI and Gladstone Institutes, continues to break new ground. Together, we are moving closer to new approaches that have the potential to change how we treat heart failure in children today, and for generations to come.” MCRI Stem Cell Medicine Theme Director and Decoding Broken Hearts Research Lead Professor Enzo Porrello
“To better understand the rare genetic condition NAXD deficiency, we partnered with experts from the Children’s Hospital of Philadelphia. Dr Rebecca Ganetzky’s expertise in rare mitochondrial disease helped us connect patient care with scientific discovery, strengthening the study and helping pave the way toward developing future treatments for affected children and families.” MCRI Brain and Mitochondrial Group Team Leader Dr Nicole Van Bergen
“Our partnership with Professor Andrea Beaton and the team at Cincinnati Children’s, alongside the Uganda Heart Institute, has been one of the most rewarding collaborations of my career — together through the GOAL studies we’ve helped transform how rheumatic heart disease is understood and managed in children. It’s a genuine threeway partnership, and a model for how global child health research should be done.” MCRI Infection, Immunity and Global Health Theme Director Professor Andrew Steer
“Professor Hugh Sampson, one of the world’s leading food allergy researchers, provides invaluable insight and strategic direction on our clinical studies and research programs as Chair of the National Allergy Centre of Excellence (NACE) International Scientific Advisory Board.” MCRI Population Allergy Group Lead and National Allergy Centre of Excellence Director Professor Kirsten Perrett “Translating MCRI’s KIF1AAssociated Neurological Disorder (KAND) research program through a collaboration with FOX Nation, world-renowned journalist Benjamin Hall and KIF1A.ORG Foundation for the Hope Starts Here documentary had an incredible impact on the international community living with this disorder.” MCRI Brain and Mitochondrial Research Group Team Leader Dr Simranpreet Kaur
11
For more information Please contact Arielle Tigel Murdoch Children’s Research Institute US Engagement & Development Lead arielle.tigel@mcri.edu.au +1 929 991 6441 Make a tax deductible donation mcri.edu.au/global
Mini kidneys: a colourful cellular ecosystem. Picture: MCRI Kidney Regeneration Team Research Assistant Hannah Baric and Research Officer Dr Allara Zylberberg
mcri.edu.au Printed October 2026