Newswise — On June 18, more than 400 researchers, clinicians, educators, and community supporters gathered for Science Day 2025—The Saban Research Institute’s (TSRI) annual symposium and poster session.

Attendees heard from experts across the scientific landscape, celebrating the groundbreaking pediatric research that translates scientific insight into real-world treatments for children at Children’s Hospital Los Angeles and beyond.

This year’s event focused on highlighting advances in cell and gene therapy. 

“Research has been part of the fabric of CHLA for well over a century,” said Paul Viviano, Chief Executive Officer. “It has laid the foundation for today’s remarkable achievements.” 

“This community is not only building on the past but working toward the future,” added Lara Khouri, Chief Operating Officer. “There’s no doubt that this is a difficult time for research—and pediatric research in particular. That means that our resolve needs to be stronger. The clarity that these challenges offer us can be a treasure.”

Every department across CHLA was represented—whether through symposium presentations, scientific posters, or data visualization. 

“Today is about inspiration, it’s about passion, and it exemplifies the very best of team science,” said Pat Levitt, PhD, Chief Scientific Officer and Senior Vice President of TSRI.

Key highlights from the day:Cell and gene therapy symposium: Changing the landscape from discovery to care

The symposium invited four gene therapy experts from research institutions across the country to share their latest discoveries around approaches that seek to treat some of the world’s most devastating genetic disorders. “It’s an extremely exciting time in the field,” said Alan S. Wayne, MD, the hospital’s Pediatrician in Chief, noting that 10 FDA-approved therapies are available to children at CHLA, the most of any pediatric health center in the West. “These therapies are paradigm-changing for children born with previously incurable diseases.”

Common threads among each presentation:

  • Gene therapies’ effects are strongest when administered early, even before patients exhibit symptoms.
  • Understanding the full phenotype of each disease and its biological mechanisms is critical to developing highly effective treatments with minimal off-target effects.
  • Next-generation tools like AI, used alongside classical research methods, will help accelerate development and bring treatments to kids faster.
  • Treatment breakthroughs for one disease may benefit many; most gene therapy approaches are reproducible in multiple contexts.
  • Gene therapies are still in the early stages. The treatments of tomorrow will utilize more sophisticated mechanisms of action to produce safer, more effective drugs. 

Carsten Bönnemann, MD, from the National Institute of Neurological Disorder and Stroke at the National Institutes of Health presented his perspective on opportunities and challenges in using AAV-mediated gene therapy—where an adeno-associated virus is used as a vector to deliver therapeutic genetic material into cells—for neuromuscular disorders like Duchenne muscular dystrophy, spinal muscular atrophy, and giant axonal neuropathy. Existing and developing therapies have demonstrated the ability to not only stop the progression of disease but also create a regenerative effect.

Alex Marson, MD, PhD, from the University of California, San Francisco, illustrated how his team is programming immunotherapies with CRISPR—one of the most precise forms of gene editing technologies in use today. His research explores the potential to refine CRISPR technologies to turn genes on and off, delete genes, make multiple epigenetic edits at once, and program a person’s pattern of gene expression for a more effective therapy with fewer side effects. 

Catherine Argyriou, PhD, MSc, a new member of CHLA’s retinal gene therapy team, presented her latest progress and lessons learned while developing retinal gene therapy for PEX1-mediated Zellweger spectrum disorder, a highly complex, multi-system disease. Using a clinically translatable design for an AAV vector that successfully delivered gene replacement therapy and improved functional vision and retinal structure in mouse models, she plans to continue to fine-tune the research for clinical validation. 

To close out the symposium, Audrone Lapinaite, PhD, from the University of California, Irvine shared how her team is empowering the development of safer, more precise genome editing therapies by investigating molecular mechanisms of disease. She pointed out that almost 60% of genetic diseases are caused by point mutations—a single letter out of place in the genetic code. This is where CRISPR technologies can be engineered to precisely target these differences with greater potency and fewer off-target effects. 

Poster session: From basic science to conversations at the bedside

Researchers presented a record-breaking 129 scientific posters. Topics in the poster session spanned basic science, clinical research, community research, medical education research, and quality improvement research. 

Before judges unveiled this year’s winners, 11 selected finalists presented bite-sized versions of their research findings in a data-blitz program. 

Data-blitz winners: 

  • For Basic Laboratory Science: Chandra Kaladhar Vemula (Mentor: JinSeok Park)
  • For Clinical Research, and People’s Choice Winner: Joanne Yip (Mentor: Nhu Tran)
  • For Quality Improvement: Andy Chan (Mentor: Vivian Lee)
  • For Community Research: Sydney Jacobs (Mentor: Charlotte DiStefano)

Celebrating the power of visual science

For the first time, organizers hosted a graphical abstract contest, where participants were invited to submit images created using the visualization tool Biorender. 

Graphical abstract contest winners:

  • Rana M. Shoaib, MD (BioRender Winner)
  • Katerin Joachin, MS (People’s Choice Winner)

Organizers also presented the winners of the Cellular Imaging Core’s annual image contest. 

Cellular Imaging Core contest winners:

  • 1st Place: Leila Bahmani, PhD, Postdoctoral Research Fellow, Aaron Nagiel lab, The Vision Center, Department of Surgery
  • 2nd Place: Claire Baldauf, MD, Neonatology Attending, Department of Pediatrics, Alexandre Bonnin Lab, Department of Pathology/FRIEND Lab, Department of Pediatrics
  • 3rd Place: Brianna Mozo, Undergraduate Student Researcher, Mark Frey lab, Department of Pediatrics

What’s next?

Dr. Levitt closed out Science Day by reiterating the importance of continued conversations around the value of science, encouraging attendees to share with their peers, communities, and lawmakers how science translates from the lab bench to lifesaving treatments at the bedside.

“What we heard this morning is truly remarkable progress,” shared Dr. Levitt. “It’s not hyperbolic to say that we have opportunities to cure diseases that we thought were completely uncurable.” 

“As advanced as we can get in our technology and our approaches to cures, they don’t matter unless we can get them to patients,” he continued. “We often underestimate that as scientists, and even sometimes as clinicians. But it’s something we need to pay attention to and put more and more effort towards.”

Thanks to the organizers

Pat Levitt, PhD, Chief Scientific Officer, Senior Vice President and Director, TSRI

Co-organizers: Bridget Fernandez, MD, MS, FRCPC, FCCMG, FACMG; Rohit Kohli, MBBS, MS

Symposium Committee: Kimberly A Chapman, MD, PhD; Babak Moghimi, MD; Aaron Nagiel, MD, PhD; Leigh Maria Ramos-Platt, 

Poster Session Award Committee: Shana Adise, PhD; Christopher Kuo, MD; Michael A. Schumacher, PhD

TSRI’s space team and The Office of Training, Education, Career Planning and Development (TECPAD)

Learn more about The Saban Research Institute of Children’s Hospital Los Angeles.