New Alliance Aims to Deliver Safer Leukaemia Therapy to Children

WELLINGTON, New Zealand and CINCINNATI, OH – April 13, 2026 – A groundbreaking international partnership is set to tackle one of the toughest challenges in childhood cancer, bringing a promising new therapy from New Zealand to a global stage. BioOra Limited, a Wellington-based cell therapy company, has joined forces with Cincinnati Children's Hospital Medical Center, the top-ranked pediatric cancer center in the United States, to advance a next-generation cancer treatment for children with relapsed or refractory B-cell acute lymphoblastic leukaemia (B-ALL).

The collaboration centers on Atla-cel, a third-generation CAR-T cell therapy designed to offer a powerful-yet-safer alternative for young patients. Building on compelling clinical data from adult trials, the partnership will launch a global pediatric clinical program led by Cincinnati Children's, aiming to provide a life-saving option that has been out of reach for many due to the risks associated with existing therapies.

The Promise of a Gentler Weapon

Chimeric Antigen Receptor T-cell (CAR-T) therapy has been a revolutionary force in oncology, re-engineering a patient's own immune cells to hunt down and destroy cancer. For children with B-ALL who have exhausted other options, it has offered unprecedented hope. However, this powerful weapon comes with a significant cost: severe side effects.

First-generation CAR-T therapies are known for causing Cytokine Release Syndrome (CRS) and, more alarmingly for pediatric patients, Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS). This neurotoxicity can manifest as confusion, seizures, or brain swelling, posing an unacceptable risk to a child's developing brain. Pivotal trials for the first approved pediatric CAR-T therapy, Kymriah, reported neurotoxicity in 40% of patients, with 13% of cases being severe. This has limited its use and necessitates long, intensive inpatient hospital stays for monitoring.

Atla-cel was engineered to overcome this critical hurdle. Results from the Phase 1 ENABLE-1 study in adults with lymphoma demonstrated not only promising efficacy but, critically, a safety profile with markedly reduced rates of serious ICANS. This differentiated safety is the cornerstone of the new partnership.

"Children with relapsed B-ALL deserve effective, safer, and more accessible therapies," said Laurence Cooper, MD, PhD, a pediatric oncologist and BioOra Board Member. "Markedly reduced neurotoxicity is not a minor footnote in paediatric oncology, it is central to whether a therapy can safely be used in children. That is why this paediatric programme matters."

A Global Alliance Forging the Path

The partnership represents a powerful synergy of innovation and expertise. BioOra brings Atla-cel, its next-generation asset developed in collaboration with the Malaghan Institute of Medical Research, and its advanced manufacturing capabilities. Cincinnati Children's, ranked the #1 cancer care provider for children by U.S. News & World Report, contributes its unparalleled clinical leadership and deep expertise in pediatric cellular therapies.

This collaboration is more than a simple agreement; it's a deep strategic alignment. As a testament to this, Steve Davis, MD, President and CEO of Cincinnati Children's, has joined the BioOra Board of Directors. "Cincinnati Children's is committed to delivering the safest and most advanced therapies to children with cancer," Dr. Davis stated. "Atla-cel's emerging safety profile, combined with BioOra's manufacturing expertise and our shared clinical vision, creates a strong foundation to pursue a genuinely differentiated treatment option."

The upcoming global clinical program, set to enroll patients across the United States, New Zealand, and potentially Australia, will be managed by Cincinnati Children's. The trial will be led by Principal Investigator Stella M. Davies, MBBS, PhD, a world-renowned leader in pediatric hematology-oncology. "Relapsed B-ALL remains one of the toughest problems in childhood cancer," Dr. Davies explained. "The low neurotoxicity signals from the ENABLE programme make Atla-cel a compelling candidate for paediatric investigation, and if that profile holds in children, it could mean bringing life changing CAR-T therapy to more kids."

Redefining Treatment and Accessibility

The potential impact of a safer CAR-T therapy extends far beyond the hospital room. If Atla-cel's low neurotoxicity profile is confirmed in children, it could fundamentally shift the treatment paradigm from intensive, prolonged inpatient care to a more manageable outpatient setting. This would dramatically reduce the physical, emotional, and financial burden on young patients and their families, freeing them from the confines of a hospital and allowing for a better quality of life during treatment.

This vision for greater accessibility is also built into BioOra's operational strategy. The company is tackling the infamous bottlenecks of CAR-T therapy—high cost and complex logistics—head-on. At its new purpose-built Health & Technology Centre in Christchurch, BioOra is implementing the Cocoon® platform, a closed, automated system for cell therapy manufacturing. This technology is designed to streamline production, reduce costs, and create a scalable model to support regional and international demand.

"This collaboration reflects our conviction that next-generation CAR-T design, disciplined clinical execution, and automated manufacturing can together transform outcomes for children with ALL," said John Robson, CEO of BioOra. "Cincinnati Children's brings the scientific depth, paediatric expertise, and global credibility this programme demands, and we are proud to partner with them."

The journey ahead involves rigorous clinical evaluation to confirm that the safety and efficacy seen in adults translate to a pediatric population. For the families of children facing a devastating diagnosis, this international alliance represents a significant step forward, carrying the promise of not just another treatment, but a better, safer, and more accessible path toward a cure.