A Seattle startup born from Seattle Children's Hospital research has raised $116 million to test a therapy for one of the deadliest childhood cancers.
BrainChild Bio announced the Series A financing on Tuesday, Sept. 8, alongside the start of a pivotal Phase 2 clinical trial for its lead drug, BCB-276, GeekWire reported. The therapy targets diffuse intrinsic pontine glioma, known as DIPG, a brainstem tumor that strikes about 300 children per year in the United States. Most are between ages 5 and 10. Median survival with the current standard treatment, palliative radiation, is about 11 months.
The round was led by an undisclosed private family fund and foundation. Seattle Children's, BrainChild Bio's original backer, and WRF Capital, the investment arm of the Seattle-based Washington Research Foundation, also participated.
Traditional venture firms largely passed. CEO Steven Brugger told Genetic Engineering & Biotechnology News that the small patient population did not fit most institutional investors' models, pushing the company toward non-traditional backers instead.
BCB-276 is a CAR T cell therapy, a treatment that reprograms a patient's own immune cells to attack cancer. BrainChild Bio's version is unusual because the engineered cells are delivered directly into cerebrospinal fluid through an implanted catheter, bypassing the blood-brain barrier that blocks most drugs from reaching brain tumors. Patients can receive repeated doses, up to 15 over roughly seven to eight months.
No FDA-approved CAR T therapy targets a solid tumor. All current approvals treat blood cancers such as leukemia and lymphoma.
The company's Phase 1 trial at Seattle Children's offered early evidence the approach could work. Among 21 patients who received the therapy, median survival from diagnosis reached 19.8 months, compared with the 11-month benchmark, according to results published in Nature Medicine. Three patients were still alive more than 44 months after diagnosis. Those results came from a small, single-center trial without a control group, so cross-trial comparisons carry limits.
"In the 50 years of clinical trials for DIPG, no drug had ever changed the 11-month overall survival mark before," founder and Chief Scientific Officer Michael Jensen told GEN.
Jensen developed the underlying CAR T technology at Seattle Children's, which licensed it exclusively to BrainChild Bio in December 2023.
He previously co-founded Juno Therapeutics, the Seattle company whose leukemia therapy eventually won FDA approval and led to Celgene's $9 billion acquisition of Juno in 2018. Brugger previously founded Affinivax, acquired by GSK for $3.3 billion in 2022.
The new Phase 2 trial, called ILLUMINATE, is recruiting at six pediatric cancer centers nationwide, including Seattle Children's, where Dr. Sarah Leary serves as principal investigator. The company has dosed the first patient. The FDA agreed to a single-arm design with no control group of children receiving only radiation. The agency had already granted BCB-276 Fast Track, Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations by May 2025.
BrainChild Bio has grown to about 50 employees since its December 2023 launch. It is headquartered in Seattle with a second office in Cambridge, Mass. The company also plans to advance a second therapy, BCB-214, into a Phase 1 glioblastoma trial in 2027.
