BrainChild Bio has received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its investigational B7-H3 CAR T-cell therapy.

BrainChild Bio公司已获得美国食品药品监督管理局（FDA）授予的再生医学先进疗法（RMAT）称号，用于其正在研究的B7-H3 CAR T细胞疗法。

BCB-276 is an autologous CAR T-cell therapy designed to target the B7-H3 protein, which is commonly found in brain tumours. Unlike conventional therapies that struggle to penetrate the brainstem and overcome the blood-brain barrier, BCB-276 is administered directly into the cerebrospinal fluid using a reservoir-catheter system.

BCB-276 是一种自体 CAR T 细胞疗法，旨在靶向常见于脑肿瘤的 B7-H3 蛋白。与传统疗法难以穿透脑干和突破血脑屏障不同，BCB-276 通过储液器-导管系统直接注入脑脊液中。

This method enables the therapy to reach the tumour site more effectively and allows for repeated dosing..

这种方法能使治疗更有效地到达肿瘤部位，并允许重复给药。

The treatment, known as BCB-276, is being developed for diffuse intrinsic pontine glioma (DIPG), a rare and aggressive paediatric brain cancer with limited treatment options.

这种被称为BCB-276的疗法正在被开发用于治疗弥漫性内生性桥脑胶质瘤（DIPG），这是一种罕见且侵袭性的儿童脑癌，治疗选择有限。

The RMAT designation is granted to regenerative medicine therapies, such as cell or gene therapies, that show preliminary clinical evidence of addressing unmet medical needs in serious or life-threatening conditions. It provides sponsors with increased support and interaction with the FDA during development, including opportunities for rolling review and priority evaluation of marketing applications..

再生医学先进疗法（RMAT）资格被授予那些在临床前研究中已显示出能够满足严重或危及生命疾病未满足医疗需求的再生医学疗法，例如细胞或基因疗法。该资格在开发过程中为申办方提供与FDA更多的支持和互动，包括滚动审查和对上市申请进行优先评估的机会。

BrainChild Bio plans to begin a multi-centre, pivotal Phase 2 trial of BCB-276 later this year. This trial will form the basis of a future Biologics License Application (BLA) submission to the FDA, targeting the treatment of children and young adults with DIPG.

今年晚些时候，BrainChild Bio计划开始BCB-276的多中心、关键性二期试验。该试验将为未来向FDA提交生物制品许可申请（BLA）奠定基础，目标是治疗患有弥漫性内生型脑桥胶质瘤（DIPG）的儿童和年轻人。

DIPG is a high-grade glioma located in the brainstem, primarily affecting children between five and ten years of age. It is considered uniformly fatal, with standard treatments offering a median survival of approximately 11 months from diagnosis. The disease’s location in the pons and the intact blood-brain barrier make conventional therapies largely ineffective..

DIPG是一种位于脑干的高级别胶质瘤，主要影响5至10岁的儿童。它被认为是一种普遍致命的疾病，标准治疗提供的中位生存期约为诊断后11个月。该疾病位于脑桥，且血脑屏障完整，使得常规疗法大多无效。

The company’s locoregional approach aims to overcome these challenges by delivering CAR T-cells directly into the cerebrospinal fluid. This allows for better exposure of the tumour to the treatment while limiting potential systemic side effects.

该公司的地方性方法旨在通过将CAR T细胞直接递送到脑脊液中来克服这些挑战。这样可以在更好地让肿瘤暴露于治疗的同时，限制潜在的系统性副作用。

The strategy may offer a new and promising avenue for more durable responses in paediatric brain tumor therapy.

该策略可能为儿童脑肿瘤治疗提供一条新的、有前景的途径，以实现更持久的疗效。

Source: brainchildbio.com

来源：brainchildbio.com