Mustang Bio, a clinical-stage biopharmaceutical company, has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) for its investigational therapy MB-101.

临床阶段的生物制药公司Mustang Bio的在研药物MB-101已获得美国食品药品监督管理局（FDA）授予的孤儿药资格。

MB-101 and MB-108 are currently being studied in Phase 1 clinical trials at City of Hope and The University of Alabama at Birmingham, respectively. Further development of the MB-109 programme will depend on securing additional funding or strategic partnerships.

MB-101 和 MB-108 分别在希望之城和阿拉巴马大学伯明翰分校进行 I 期临床试验。MB-109 项目的进一步开发将取决于获得额外的资金或战略合作伙伴关系。

MB-101 forms part of a broader therapeutic strategy under development by Mustang Bio. The company is exploring the potential of combining MB-101 with MB-108, an oncolytic virus therapy, under the combined programme MB-109. This approach is designed to enhance treatment outcomes in patients with malignant glioma by modifying the tumour microenvironment (TME).

MB-101 是 Mustang Bio 正在开发的更广泛治疗策略的一部分。该公司正在探索将 MB-101 与溶瘤病毒疗法 MB-108 联合使用的潜力，这一联合项目被称为 MB-109。该方法旨在通过改变肿瘤微环境 (TME) 来改善恶性胶质瘤患者的治疗效果。

MB-108 is administered first to stimulate immune activity within the tumour, which may in turn increase the effectiveness of MB-101..

首先施用MB-108以刺激肿瘤内的免疫活性，这可能会提高MB-101的有效性。

The treatment targets IL13Ra2 using CAR T-cell technology and is intended for use in patients with recurrent diffuse and anaplastic astrocytoma and glioblastoma (GBM).

该疗法利用CAR T细胞技术靶向IL13Ra2，用于治疗复发性弥漫性及间变性星形细胞瘤和胶质母细胞瘤（GBM）患者。

Orphan Drug Designation is awarded to therapies aimed at treating rare conditions affecting fewer than 200,000 individuals in the United States. The designation provides a range of benefits, including tax credits for clinical trials and a waiver of certain regulatory fees. It also offers seven years of market exclusivity following approval, separate from any patent protection..

孤儿药资格授予旨在治疗美国境内少于20万人影响的罕见病的疗法。该资格提供一系列好处，包括临床试验的税收抵免和某些监管费用的豁免。它还提供了在获批后七年的市场独占期，这与任何专利保护是分开的。

Preclinical and early clinical data suggest that this combination could be particularly effective in patients with “hot” tumours — those already showing signs of immune activation. Notably, earlier research showed that two patients with such tumours responded exceptionally well to MB-101 alone, achieving complete responses lasting several months to years..

临床前和早期临床数据表明，这种组合对“热”肿瘤患者可能特别有效——那些已经显示出免疫激活迹象的患者。值得注意的是，早期研究显示，两名这样的肿瘤患者对单独使用MB-101的反应非常好，实现了持续数月到数年的完全缓解。

The company continues to focus on developing innovative therapies for hard-to-treat cancers, with the aim of improving outcomes in rare and aggressive brain tumours.

公司继续专注于为难治性癌症开发创新疗法，目标是改善罕见且侵袭性的脑肿瘤的治疗效果。

Source: mustangbio.com

来源：mustangbio.com