BrainChild Bio’s CAR T-cell Therapy for DIPG Receives RMAT Designation

SEATTLE, WA and Cambridge, MA – May 15, 2025 – BrainChild Bio, Inc., a clinical-stage biotechnology company focused on developing CAR T-cell therapies for central nervous system (CNS) tumors, today announced that its investigational B7-H3 targeting autologous CAR T-cell therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor.

BrainChild Bio’s CAR T-cell approach aims to overcome the challenges of treating DIPG, including the tumor’s location in the brainstem, its infiltrative growth, and the blood-brain barrier, by delivering the targeted CAR T-cells directly into the cerebrospinal fluid via a specialized reservoir-catheter device. This locoregional delivery method showed encouraging overall survival benefits in patients with brain tumors in their Phase 1 trial conducted with Seattle Children’s Research Institute. The RMAT designation from the FDA will provide enhanced interaction and the potential for accelerated approval pathways. BrainChild Bio is focused on initiating a pivotal Phase 2 trial for BCB-276 by the end of this year, with the goal of bringing a new CAR T-cell treatment to children and young adults with this devastating CNS tumor. DIPG, affecting approximately 300 children annually in the U.S., has a dismal prognosis with current treatments. CAR T-cell therapy offers a novel strategy by directly targeting the tumor cells within the central nervous system, potentially improving efficacy while minimizing systemic side effects.