CAMBRIDGE, Mass. — BrainChild Bio has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration for its investigational CAR T-cell therapy, BCB-276, aimed at treating diffuse intrinsic pontine glioma (DIPG), a devastating and currently incurable pediatric brain tumor.
The designation, which follows a Breakthrough Therapy designation granted less than a month ago, underscores the FDA’s recognition of the therapy’s potential to address a critical unmet medical need. DIPG is an aggressive brainstem tumor that primarily affects children and is notoriously resistant to traditional therapies due to its location, infiltrative growth, and the presence of an intact blood-brain barrier.
BCB-276 is an autologous CAR T-cell therapy targeting the B7-H3 antigen. Unlike traditional systemic treatments, BrainChild Bio has engineered the therapy for locoregional delivery via the cerebrospinal fluid, using an indwelling catheter system to bypass the blood-brain barrier and deliver immune cells directly to the tumor site. This approach showed encouraging results in a Phase 1 trial (BrainChild-03), conducted at Seattle Children’s Research Institute and published in Nature Medicine.
“We are very pleased to now receive RMAT designation, which, along with the Breakthrough Therapy designation, validates the strength of our clinical data and the urgent need for a therapy to treat DIPG,” said Dr. Michael Jensen, founder and chief scientific officer of BrainChild Bio. “Our focus is now on launching a pivotal Phase 2 trial by the end of this year to accelerate our path toward regulatory approval.”
The FDA’s RMAT designation is reserved for regenerative medicines, including cell therapies, that show early clinical promise in treating serious or life-threatening conditions. The designation allows for closer collaboration with the FDA and can provide benefits such as rolling submissions and priority review for a future Biologics License Application (BLA).
Seattle Children’s CEO Dr. Jeff Sperring welcomed the designation as a milestone in the fight against pediatric brain cancer. “This recognition represents another critical step in advancing new treatments for children facing DIPG,” he said. “We are proud to see our research moving forward in ways that could soon benefit young patients.”
BrainChild Bio plans to initiate its Phase 2 multi-center registration trial in late 2025, following alignment with the FDA in a Type B meeting held in 2024. If successful, the trial could support a BLA submission for the first disease-modifying treatment for DIPG.
