About the Advanced Therapeutics Initiative
Closing the Pediatric Therapeutics Gap at Lurie Children’s
At Ann & Robert H. Lurie Children’s Hospital of Chicago, advancing children’s health means not only delivering world-class care, but also ensuring that children have access to new therapies they might need.
The Advanced Therapeutics Initiative (ATI) is an institution-wide effort designed to accelerate the development, testing, and translation of innovative therapies for children. ATI was created to address a persistent gap in pediatric medicine: children often gain access to novel therapies years, sometimes decades, after adults. Too many promising discoveries never reach children, and too few therapies are developed specifically for pediatric populations. Children are not simply small adults. They have distinct disease biology, unique developmental considerations, and pharmacologic needs that demand pediatric-specific investigation.
By integrating therapeutic discovery, regulatory strategy, precision diagnostics, and early-phase clinical trial execution within a single, coordinated model, ATI enables Lurie Children’s to lead at the earliest stages of pediatric drug development, where scientific insight, regulatory engagement, and clinical impact begin. Building on Lurie Children’s established clinical volume, deep subspecialty expertise, and mature research infrastructure, ATI represents a strategic acceleration rather than a greenfield build, positioning the institution to meet the moment created by the United States RACE for Children Act and a rapidly expanding pediatric therapeutic landscape.
Scientific advances in genomics, cell and gene therapy, tissue engineering, and precision medicine are rapidly expanding what is possible for children with serious and rare diseases. Realizing the promise of these innovations requires specialized infrastructure, multidisciplinary expertise, and the ability to translate discoveries efficiently into clinical care.
Lurie Children’s brings these capabilities together in a single pediatric-focused ecosystem. With nationally recognized clinical programs and significant subspecialty expertise, established early-stage clinical trials and first-in-child trial activity, advanced genomic and molecular diagnostic capabilities, mature regulatory and clinical research unit infrastructure, a commitment to innovation, and a national referral network, we are positioned to accelerate development of the next generation of pediatric therapeutics.
ATI serves as an institutional engine that transforms clinical insight and scientific discovery into first-in-child and early-phase pediatric clinical trials. We accelerate this work through two complementary pathways: serving as a national site for sponsored early-phase pediatric trials and advancing internally developed discoveries toward first-in-child testing. Through these efforts, ATI brings together:
- Investigators and clinician-scientists
- Regulatory and operational expertise
- Centralized infrastructure and governance
- Institutional and external partnerships
This model is designed to reduce fragmentation, accelerate timelines, and enable responsible scale, ensuring that promising therapies move efficiently from concept to clinical testing.
ATI provides investigators and partners with the infrastructure and support needed to move therapies forward faster and more effectively. Through ATI, investigators gain access to:
- Dedicated infrastructure for early-phase and first-in-child pediatric trials
- Integrated IND/IDE, IRB, and regulatory strategy support
- Centralized trial activation, finance, and operational support through the Office of Clinical and Community Trials (OCCT)
- Advanced diagnostics, imaging, and multimodal data through the ATI Data Commons
- A coordinated institutional framework aligned around pediatric therapeutic innovation
- Training and mentorship through a structured early-phase clinical trial career development program, developing the next generation of pediatric therapeutic researchers
- Pathways for industry, academic, and foundation partnerships in pediatric drug development
Together, these capabilities position Lurie Children’s as a leading site for early-stage pediatric therapeutic development.
ATI is designed to create lasting clinical, scientific, and institutional impact. Over the coming years, ATI will:
- Expand early-phase pediatric trial capacity
- Advance internally developed therapeutics toward IND submission
- Enable new models for pediatric drug development
- Build strategic partnerships across academia and industry
- Engage patients and families as partners throughout therapeutic development and trial design
- Train the next generation of pediatric therapeutic researchers through the Phase I early-phase clinical trial career development program
Through this work, Lurie Children’s is strengthening its role as a leader in pediatric therapeutic innovation, ensuring that children and families benefit from the earliest advances in science and medicine. For families facing limited options, ATI offers therapeutic pathways—and hope—when standard approaches have been exhausted.
Children's Research Fund
As the principal benefactor of Stanley Manne Children’s Research Institute, the Children’s Research Fund (CRF) is accelerating the Advanced Therapeutics Initiative through a transformative $11.5 million investment. The CRF is a volunteer fundraising board composed of 75 men and women dedicated to advancing pediatric research. Since 1951, they have invested in the expertise, infrastructure, and resources needed to develop innovative therapies for children with serious and complex conditions.
Through its investment in ATI, the CRF is helping bring together researchers and clinical experts, strengthen the pathway from scientific discovery to clinical care, expand access to early-phase clinical trials, and help bring promising new treatments to children. Manne Research Institute is grateful for the CRF’s partnership and longstanding commitment to advancing pediatric research at Ann & Robert H. Lurie Children’s Hospital of Chicago.
Philanthropy
Your support is vital in helping us continue to make a difference in the lives of patients and families. Lurie Children's relies on philanthropic funding to enhance its programs, services and research for children. To learn more, please e-mail the Ann & Robert H. Lurie Children’s Hospital of Chicago Foundation at foundation@luriechildrens.org or call 312.227.7500.
