BioOra and Cincinnati Children's unite to evaluate Atla-cel in paediatric relapsed or refractory B-ALL, building on emerging adult clinical data demonstrating markedly reduced neurotoxicity with this next-generation CAR-T therapy.

Key Takeaways:

• BioOra and Cincinnati Children's are partnering to advance Atla-cel, a paediatric CAR‑T therapy for relapsed/refractory B‑ALL.
  
  
• Atla‑cel shows markedly reduced neurotoxicity in adult trials, supporting its evaluation in children who could benefit from this therapy not previously available to them.
  
  
• Cincinnati Children's will lead a global clinical program to evaluate Atla-cel in children.

WELLINGTON, New Zealand and CINCINNATI, April 13, 2026 /PRNewswire/ -- BioOra Limited and Cincinnati Children's today announced a strategic partnership to advance Atla-cel, (atlacabtagene autoleucel, pINN List 134), a third-generation CD19-directed CAR-T cell therapy, into clinical development for children and adolescents with relapsed or refractory B-cell acute lymphoblastic leukaemia (B-ALL).

A differentiated safety profile — and what it means for children

Despite significant advances in frontline therapy, children with relapsed or refractory B-ALL face poor long-term outcomes and limited treatment options. First-generation approved CAR-T therapies have demonstrated meaningful efficacy. However, cytokine release syndrome (CRS) and, in particular, immune effector cell-associated neurotoxicity syndrome (ICANS), remain barriers to broader adoption, especially in paediatric patients, where neurotoxicity carries heightened developmental risks and typically requires intensive inpatient monitoring.

Atla-cel is a third-generation CD19-directed CAR-T therapy incorporating design advances intended to enhance anti-tumour activity and improve the tolerability profile compared with earlier-generation approaches. Results from the Phase 1 ENABLE-1 study in adults with relapsed or refractory lymphoma, presented at the 2024 American Society of Hematology Annual Meeting, demonstrated promising complete response rates alongside a safety profile notable for low rates of serious CRS and, critically, markedly reduced ICANS. These safety signals position Atla-cel as a compelling candidate for paediatric investigation.

In children, ICANS is not a side effect to be managed, it is an unacceptable risk. The developing brain is uniquely vulnerable to immune-mediated neurotoxicity, and this has kept CAR-T therapy from reaching many of the patients who need it most. The markedly low ICANS rates observed in adult studies provide the strongest rationale yet for paediatric evaluation and raise the prospect of outpatient delivery that would free children and families from the prolonged inpatient stays that currently approved therapies require.

The partnership with Cincinnati Children's, one of the world's leading paediatric academic medical centres with deep expertise in haematology, oncology, and cellular therapies, leverages clinical data from the ENABLE trial programme, conducted in collaboration between BioOra and the Malaghan Institute of Medical Research in New Zealand. Atla-cel incorporates design advances aimed at enhancing anti-tumour activity while improving tolerability compared with earlier-generation CAR-T therapies.

As part of the collaboration, Steve Davis, MD, President and Chief Executive Officer of Cincinnati Children's, joins the BioOra Board of Directors.

Clinical programme: trial sites and leadership

The proposed paediatric B-ALL clinical programme would enrol patients across the United States, New Zealand, and potentially Australia, with trial management from Cincinnati Children's.

The trial will be led by Stella M. Davies, MBBS, PhD, MRCP, Institute Co-Executive Director of the Cancer and Blood Diseases Institute and Director of the Division of Bone Marrow Transplantation and Immune Deficiency at Cincinnati Children's, ranked the #1 cancer care provider in the United States by US News and World Report. Dr. Davies is a recognised leader in paediatric haematology-oncology and haematopoietic cell transplantation and currently serves as President-Elect of the American Society for Transplantation and Cellular Therapy.

Leadership perspectives

Laurence Cooper, MD, PhD, paediatric oncologist and Board Member of BioOra, said: "Children with relapsed B-ALL deserve effective, safer, and more accessible therapies. The ENABLE programme has given us confidence in Atla-cel's adult safety profile; 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."

Stella M. Davies, MBBS, PhD, MRCP, Institute Co-Executive Director, Cancer and Blood Diseases Institute, Cincinnati Children's, and Principal Investigator, said: "Relapsed B-ALL remains one of the toughest problems in childhood cancer. 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."

John Robson, Chief Executive Officer of BioOra, said: "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. Cincinnati Children's brings the scientific depth, paediatric expertise, and global credibility this programme demands, and we are proud to partner with them."

Steve Davis, MD, President and Chief Executive Officer of Cincinnati Children's, said: "Cincinnati Children's is committed to delivering the safest and most advanced therapies to children with cancer. 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 for children with relapsed B-ALL in the United States and globally."

About BioOra Limited

BioOra is a commercial-stage cell therapy company headquartered in Wellington, New Zealand, focused on developing and commercialising next-generation cell therapies for cancer patients across New Zealand, Australia, and beyond. Its lead programme asset, Atla-cel, is a CD19-directed CAR-T therapy currently in a Phase 2 ENABLE-2 registration trial for adults with relapsed or refractory B-cell lymphoma and is now being advanced into paediatric B-ALL in partnership with Cincinnati Children's Hospital Medical Center. BioOra's Health & Technology Centre in Christchurch is a purpose-built GMP manufacturing facility designed to operate the Cocoon® platform at scale to support regional and international demand.

About Cincinnati Children's

Cincinnati Children's is ranked among the best pediatric health systems in the nation by U.S. News & World Report, including No. 1 in three specialties: Cancer care, Gastroenterology & GI surgery, and Diabetes & Endocrinology. Cincinnati Children's has been named repeatedly one of America's Most Innovative Companies by Fortune – and is the top-rated pediatric health system in the national ranking of organizations across all industries. Forbes ranks Cincinnati Children's among the Top 10 Best Employers for Tech Workers. Established in 1883, Cincinnati Children's is a nonprofit academic health system that leads the way in healthcare education and pediatric research, where discoveries become innovative treatments and cures that change the outcome for children. All of this is backed by an extraordinary culture with over 19,400 team members. Cincinnati Children's ranks No. 1 in Ohio and No. 5 in the nation on the Forbes list of America's Best Employers for Healthcare Professionals. More information: CincinnatiChildrens.org

Media contacts:

BioOra Limited
Mrs Sofie Parker, Head of Patient Access
Sofie.Parker@bioora.com

Cincinnati Children's
Barrett Brunsman
Barrett.brunsman@cchmc.org

View original content to download multimedia:https://www.prnewswire.com/news-releases/bioora-and-cincinnati-childrens-hospital-medical-center-partner-to-advance-next-generation-car-t-therapy-for-children-with-leukaemia-302740800.html

SOURCE BioOra