Cell therapy weekly: world’s first B-cell therapy administered to pediatric patient
This week: The US Food and Drug Administration (FDA; MD, USA) will host a workshop, “Advancing Pediatric Cell and Gene Therapy Clinical Trials,” featuring a presentation on a case review of the world’s first engineered B-cell therapy in a pediatric patient. Meanwhile, researchers launch global studies on difficult-to-treat pediatric solid cancers and a cell manufacturing partnership expands to further facilitate advanced therapies in Australia and New Zealand.
The news highlights:
- World’s first B-cell therapy administered to pediatric patient
- Launch of global studies on difficult-to-treat pediatric solid cancers
- Cell manufacturing partnership expands
World’s first B-cell therapy administered to pediatric patient
On 9 April 2026, clinical-stage biotechnology company Immusoft (CA, USA) will present a clinical case review of the world’s first engineered B-cell therapy administered to a patient. The presentation will take place at the FDA’s public workshop titled, “Advancing Pediatric Cell and Gene Therapy Clinical Trials.” It will feature observations from Immusoft’s ongoing Phase I trial of its ISP-001 therapy. The workshop, scheduled from 11:00 am – 1:15 pm ET, is being co-hosted by the Alliance for Regenerative Medicine (DC, USA).
ISP-001 is an investigational modified B-cell therapy designed to treat Mucopolysaccharidosis type I, a rare pediatric genetic disorder caused by a deficiency of the enzyme α-L-iduronidase. This enzyme is essential for breaking down glycosaminoglycans, complex sugar molecules found in cells. Without sufficient α-L-iduronidase, glycosaminoglycans accumulate, leading to organ damage and potentially early death.
The therapy aims to overcome the limitations of current enzyme replacement therapy infusions and address safety concerns associated with stem cell therapy. ISP-001 utilizes the patient’s own B cells to produce and secrete therapeutic levels of α-L-iduronidase. The FDA has recognized ISP-001 with Orphan Drug, Rare Pediatric Disease, and Fast Track designations.
“While still early, these findings demonstrate signs of functional improvement and reductions in pain, providing initial evidence supporting the potential to enable sustained in vivo protein expression via engineered B cells,” commented Robert Sikorski, Chief Medical Officer of Immusoft. “We appreciate the opportunity to engage with FDA and the broader community as this new therapeutic modality continues to evolve.”
Watch the workshop via the FDA’s YouTube channel.
Launch of global studies on difficult-to-treat pediatric solid cancers
A global team of researchers from Cancer Grand Challenges (London, UK) has initiated three interconnected international early-phase studies to evaluate innovative T-cell therapies for children and young individuals with hard-to-treat solid tumors.
The team, known as NexTGene, is focusing on next-generation CAR-T therapies and tumor-associated antigen T cells engineered to overcome TGFβ-mediated immunosuppression. These trials are based on the team’s preclinical research, which identified B7-H3 as a widely and consistently expressed target across pediatric sarcomas and brain tumors.
“We’re not just testing therapies, we’re learning from every patient,” explained Catherine Bollard, NexTGen co-lead. “Our correlative studies will help us understand why therapies work and how to make them better.”
The study program will be the first of its kind and is open at three centers, each focusing on specific tumor types and therapy approaches. The sites include:
- MIGHTY trial at University College London (UK):
At University College London Hospital and Great Ormond Street Hospital (London, UK), the MIGHTY trial will focus on treating pediatric patients with sarcomas, including relapsed or refractory rhabdomyosarcoma and Ewing sarcoma. The trial will utilize B7-H3-targeting CAR-T cells, designed to precisely recognize and attack tumor cells without affecting healthy tissue.
- SABRE trial at Children’s National Hospital (DC, USA):
Pediatric patients with relapsed or refractory embryonal tumors, including rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumors, will receive a combination of B7-H3-targeting CAR-T cells and PRAME-specific T cells. This approach aims to address antigen loss and overcome the suppressive tumor microenvironment.
- Trial at the Dana-Farber Cancer Institute (MA, USA):
At this site, central nervous system tumors, including diffuse midline gliomas and other high-risk pediatric brain tumors, will be treated using the same B7-H3 CAR-T therapy. The study will also explore intracranial delivery methods to evaluate therapy uptake within the tumor.
The Cancer Grand Challenges NexTGen team is co-funded by Cancer Research UK (London), the National Cancer Institute (MD, USA) and the Mark Foundation for Cancer Research (NY, USA).
Cell manufacturing partnership expands
BioOra Limited (Wellington, New Zealand), a commercial-stage cell therapy company, and Octane Biotech Inc. (Ontario, Canada), an Octane Medical Group Company, are strengthening their partnership to advance cell therapy manufacturing. The two companies have signed a Letter of Intent to co-develop and deploy advanced therapies using Octane’s Cocoon® automated cell therapy manufacturing platform. This collaboration aims to drive regional growth for BioOra while enabling Octane to expand its capacity to support both Australian and international markets.
As part of the agreement, BioOra’s CD19-targeted CAR-T therapy (Atla-Cel), produced on Octane’s Cocoon Platform, will continue to be used in the registration trial, ENABLE-2, for patients with relapsed or refractory B-cell lymphoma in New Zealand. To meet the growing demand for advanced therapies, BioOra is also constructing the BioOra Health & Technology Centre, a cell therapy manufacturing facility in Christchurch, New Zealand, to operate Cocoon systems to meet growing regional demand for advanced cell therapies.
“BioOra has demonstrated that manufacturing using the Cocoon Platform can support the commercial delivery of CAR-T therapy in New Zealand,” said John Robson, CEO of BioOra. “Our new Christchurch facility is designed to scale this model using multiple Cocoon systems. Partnering with Octane allows us to expand into Australia and beyond while improving efficiency and increasing patient access.”
