BrainChild Bio Receives FDA RMAT Designation for B7-H3 CAR T-cell Therapy Targeting Paediatric Brain Tumours
Friday, May 16, 2025
BrainChild Bio has received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its investigational B7-H3 CAR T-cell therapy.
BCB-276 is an autologous CAR T-cell therapy designed to target the B7-H3 protein, which is commonly found in brain tumours. Unlike conventional therapies that struggle to penetrate the brainstem and overcome the blood-brain barrier, BCB-276 is administered directly into the cerebrospinal fluid using a reservoir-catheter system. This method enables the therapy to reach the tumour site more effectively and allows for repeated dosing.
The treatment, known as BCB-276, is being developed for diffuse intrinsic pontine glioma (DIPG), a rare and aggressive paediatric brain cancer with limited treatment options.
The RMAT designation is granted to regenerative medicine therapies, such as cell or gene therapies, that show preliminary clinical evidence of addressing unmet medical needs in serious or life-threatening conditions. It provides sponsors with increased support and interaction with the FDA during development, including opportunities for rolling review and priority evaluation of marketing applications.
BrainChild Bio plans to begin a multi-centre, pivotal Phase 2 trial of BCB-276 later this year. This trial will form the basis of a future Biologics License Application (BLA) submission to the FDA, targeting the treatment of children and young adults with DIPG.
DIPG is a high-grade glioma located in the brainstem, primarily affecting children between five and ten years of age. It is considered uniformly fatal, with standard treatments offering a median survival of approximately 11 months from diagnosis. The disease’s location in the pons and the intact blood-brain barrier make conventional therapies largely ineffective.
The company’s locoregional approach aims to overcome these challenges by delivering CAR T-cells directly into the cerebrospinal fluid. This allows for better exposure of the tumour to the treatment while limiting potential systemic side effects.
The strategy may offer a new and promising avenue for more durable responses in paediatric brain tumor therapy.
Source: brainchildbio.com
