Pursuing bold technology is in our DNA.

BrainChild Bio is leveraging years of experience in cell engineering and drug discovery to become a first mover in the CNS-oncology immunotherapy sector. We are integrating advanced-stage synthetic technologies into our therapeutic platform to overcome the challenges of solid tumor targeting within the central nervous system.

Targeted, potent and persistent.

We are pioneering repetitive CNS cerebroventricular cell product dosing via an implantable shunt. This allows for augmented tumor access and compartmentalization of therapeutic cells for enhanced safety and efficacy, eliminating the need for systemic lymphodepleting chemotherapy.

Our recursive, fractionated dosing of cells enhances and sustains potency, ensuring the tumor bed is continuously populated with bioactive, anti-tumor effector cells in controlled amounts and durations.

Engineered functionality. Integrated by design.

Multiplexed Targeting

To overcome tumor target gene expression heterogeneity, we have a portfolio of high-value CARs targeting primary brain tumors and brain metastases, each of which have been tested in Phase I trials with excellent safety and tolerability outcomes. These targets include oncogenic EGFR, HER2, B7-H3 and IL13Ralpha2. Our portfolio is actively being investigated clinically as multiplexed targeting products as a strategy to diminish the incidence of therapeutic failures arising from the outgrowth of antigen escape tumor variants.

Potency Enhancement

Tumors that arise in or metastasize to the CNS deploy immuno-evasive mechanisms that diminish the activity of immune effector cells. We have developed a library of synthetic transgenes that, when expressed alongside CARs, augment the potency of the manufactured cell product and counteract the immunosuppressive impact of the tumor microenvironment. Our lead candidate PotencyPlus transgene will be integrated into BCB-214 for near-term Phase I testing.

DrugON Control

To date, CAR-T products rely on cell autonomous regulation and once administered, can mediate immune-mediated toxicities which degrade their therapeutic index. BrainChild Bio has developed an extensive portfolio of synthetic genetic constructs that allow for conditional activation, as well as potency enhancement. These include synthetic promoters that mediate transcription of transgenes conditional on CAR activation signaling and receptors (including CARs) that are conditionally quiescent unless a small molecule drug is present. To maximize the impact of CNS locoregional CAR-T therapy, we will deploy these technologies in BCB-513 for clinician control of potency enhanced next generation products.

Our initial clinical focus is on diffuse intrinsic pontine glioma (DIPG), an incurable type of childhood cancer that forms in the brainstem — with plans to expand to other pediatric and adult brain tumors, including glioblastoma and brain metastases.

Brain tumors are the most common solid cancer in children and the leading cause of childhood cancer-related deaths.

Glioblastoma affects nearly

15,000

Americans every year and is incurable.

Metastasis to the brain will occur in approximately

1in5

cancer patients.