Funding, clinical progress and strategic shifts - Weekly News Round-up

BrainChild Bio raises $116 million to advance CAR-T for paediatric brain cancer

Seattle-based BrainChild Bio has raised $116 million in a Series A financing to advance its CAR-T cell therapy programmes for paediatric brain cancers. The company, spun out of Seattle Children’s Hospital in 2023, will use the funding to progress its lead candidate, BCB-276, through a pivotal Phase 2 trial in diffuse intrinsic pontine glioma (DIPG), a rare and aggressive childhood brain tumour. BCB-276 is designed to target tumours in the central nervous system, with BrainChild taking an approach that involves modifying patients’ own T cells before administering them directly into the CNS. The company is also developing a second programme targeting glioblastoma, with a Phase 1 trial planned for next year.

The financing reflects continued investor interest in cell therapies addressing significant unmet needs, while also highlighting the challenges associated with developing treatments for paediatric cancers and other difficult-to-treat solid tumours.

Bristol Myers Squibb reports positive results from pivotal CAR-T trial

Bristol Myers Squibb has announced positive topline results from the registrational Phase 2 Quintessential trial of arlocabtagene autoleucel (arlo-cel), an investigational GPRC5D-directed CAR-T therapy for patients with heavily pretreated relapsed or refractory multiple myeloma. The trial met its primary endpoint of overall response rate, as well as key secondary endpoints including complete response rate. The study focused on patients who had received at least four previous lines of treatment, including prior therapy targeting BCMA, an increasingly important population as patients develop resistance to existing treatment options.

Arlo-cel is designed to target GPRC5D, a protein expressed on plasma cells in multiple myeloma and distinct from BCMA. BMS said the results support the potential of targeting alternative antigens with CAR-T therapy, particularly for patients whose disease has progressed following BCMA-directed treatment.

ArsenalBio makes major pivot towards in vivo CAR-T

ArsenalBio is making a significant strategic shift towards in vivo CAR-T, resulting in the departure of 99 employees, representing the majority of its workforce. The company has also stopped development of its existing ex vivo clinical assets as it redirects its resources towards the emerging in vivo CAR-T field. The move follows an earlier round of restructuring in 2025, when the company reduced its workforce to focus on its lead CAR-T programme in renal cell carcinoma. ArsenalBio had previously raised $325 million and secured backing from major industry players, including Bristol Myers Squibb and Regeneron.

The pivot illustrates both the potential and the competitive pressure surrounding in vivo CAR-T. Rather than manufacturing CAR-T cells outside the body before administering them to patients, in vivo approaches aim to generate CAR-T cells directly within the patient. ArsenalBio now joins a growing field that includes major pharmaceutical companies such as Eli Lilly, Johnson & Johnson, AstraZeneca and Gilead's Kite Pharma.

Encoded Therapeutics secures $275 million to advance Dravet syndrome therapy

Encoded Therapeutics has raised $275 million in a Series F financing to support the development of its lead programme for Dravet syndrome, while also expanding its internal manufacturing capabilities. The funding will support a pivotal study of ETX101 in infants and young children with SCN1A-positive Dravet syndrome, a rare genetic form of epilepsy. The company is also planning to use the financing to scale commercial manufacturing and prepare a 2027 IND submission for ETX301, an AAV9-based gene therapy programme targeting post-amputation neuroma pain.

The financing was co-led by GV and an undisclosed healthcare fund, with participation from ARCH Venture Partners, Janus Henderson Investors, RTW Investments and SoftBank Vision Fund. The investment comes as ETX101 moves into pivotal development, following encouraging clinical data from earlier-stage studies.

This week's developments highlight several of the key forces shaping the advanced therapies landscape: substantial private investment is continuing to support programmes approaching pivotal development, while clinical validation is increasingly influencing where capital is deployed. At the same time, companies are reassessing established approaches and redirecting resources towards emerging technologies. As more programmes move from early-stage research towards clinical and commercial milestones, the ability to demonstrate meaningful clinical differentiation, build scalable manufacturing infrastructure and secure sufficient capital will remain central to the sector's progress.