, the FDA approved an IND application enabling Myrio's lead product (PHOX2B PC-CAR T) to enter human trials. Myrio, in collaboration the leading children's hospital in

，美国食品药品监督管理局（FDA）批准了Myrio公司的IND申请，使其主导产品（PHOX2B PC-CAR T）可以进入人体试验。Myrio与领先的儿童医院合作，

Philadelphia

费城

have co-developed a Chimeric Antigen Receptor (CAR-T) therapy for the treatment of neuroblastoma. Under the leadership of Prof.

共同开发了一种用于治疗神经母细胞瘤的嵌合抗原受体（CAR-T）疗法。在教授的领导下。

John Maris

约翰·马里斯

, a functionally relevant and highly specific protein, called PHOX2B, was identified in neuroblastoma cells and the team elucidated that a peptide from PHOX2B could serve as an excellent target for an immunotherapy to treat this devastating disease.

在神经母细胞瘤细胞中发现了一种功能相关且高度特异的蛋白质，称为PHOX2B，团队阐明了来自PHOX2B的肽可以作为治疗这种破坏性疾病的免疫疗法的绝佳靶点。

Myrio developed a highly specific binder to the PHOX2B peptide-major histocompatibility complex (p-HLA) target using its unique ReD

Myrio利用其独特的ReD技术开发了一种针对PHOX2B肽-主要组织相容性复合体（p-HLA）靶标的高特异性结合剂。

TM

商标

technology. In collaboration with the hospital, the Myrio binder has been engineered into a CAR-T product. Importantly, unlike other binders which target a single HLA-allotype, Myrio's binder is capable of recognizing the peptide in multiple HLA-allotypes, a phenomenon referred to as breaking HLA restriction, which offers the opportunity to treat a broader population of patients using the same immunotherapy..

技术。与医院合作，Myrio的结合剂已被设计成CAR-T产品。重要的是，不同于其他靶向单一HLA型别的结合剂，Myrio的结合剂能够识别多HLA型别中的肽段，这一现象被称为突破HLA限制，这为使用同一种免疫疗法治疗更广泛的患者群体提供了机会。

This groundbreaking approach was detailed in a publication by Myrio, in the journal

这一开创性的方法在 Myrio 发表于期刊的出版物中得到了详细说明

Nature

自然

titled 'Targeting of intracellular oncoproteins with peptide-centric CARs.'

标题为“使用以肽为中心的CAR靶向细胞内癌蛋白”。

in

在

November 2023

2023年11月

This is the first time a binder developed by Myrio will enter a human trial and is a major validation of the company's technology, building on the significant insights shared in their joint research.

这是Myrio开发的粘合剂首次进入人体试验，是对该公司技术的重大验证，建立在他们联合研究中分享的重要见解之上。

'Neuroblastoma is the most common tumor of the sympathetic nervous system, the most common malignancy of infancy and accounts for 15% of pediatric cancer-related deaths', said Prof. Maris. The current treatment options for patients with high-risk neuroblastoma are associated with low response rates and significant toxicities and the development of new treatment options is desperately needed.

马里斯教授说：“神经母细胞瘤是交感神经系统最常见的肿瘤，是婴儿时期最常见的恶性肿瘤，占儿科癌症相关死亡的15%。” 目前针对高风险神经母细胞瘤患者的治疗选择反应率低且毒性显著，因此迫切需要开发新的治疗方案。

This investigational immunotherapy has the potential to be a major advance for patients suffering from this devastating disease'..

这种研究性免疫疗法有望为遭受这种毁灭性疾病困扰的患者带来重大突破。

The Phase 1 clinical trial entitled 'PHOX2B Peptide-Centric Chimeric Antigen Receptor Autologous T cells (PHOX2B PC-CAR T) for Relapsed Neuroblastoma will be conducted under the leadership of Prof. Maris and it is anticipated that the first patient will be enrolled mid-year 2025.

题为“PHOX2B肽为中心的嵌合抗原受体自体T细胞（PHOX2B PC-CAR T）治疗复发性神经母细胞瘤”的I期临床试验将在Maris教授的领导下进行，预计第一名患者将于2025年年中入组。

, CEO of Myrio Therapeutics commented 'that this is a major step forward for Myrio. It is the culmination of many years of work at Myrio in developing bispecific binders to Human Leukocyte Antigens for the treatment of solid tumours.'

Myrio Therapeutics首席执行官评论道：“这对Myrio来说是一个重大的进步。这是Myrio多年来致力于开发针对人类白细胞抗原的双特异性结合剂以治疗实体瘤的成果结晶。”

About Neuroblastoma

关于神经母细胞瘤

Neuroblastoma is the most common tumor of the sympathetic nervous system (97%) and the most common malignancy of infancy with a median age of diagnosis of 17 months.

神经母细胞瘤是交感神经系统最常见的肿瘤（97%），也是婴儿期最常见的恶性肿瘤，诊断时的中位年龄为17个月。

The annual incidence of neuroblastoma in

神经母细胞瘤的年发病率

the United States

美国

is approximately 800 cases, and it accounts for 15% of pediatric cancer-related deaths.

大约有800例，占儿科癌症相关死亡的15%。

Due to the high variability in its presentation, clinical signs and symptoms at presentation can range from a benign palpable mass with distension to major illness from substantial tumor spread. Although overall increases in five-year event-free survival have been reported, this has come at the cost of significant life threating treatment-related side effects, and subgroup-specific analysis of mortality has revealed discordance between the high cure rates for the more benign low-risk forms and little improvement in the high-risk groups..

由于其表现的高变异性，临床症状和体征可从伴有膨胀的良性可触及肿块到因肿瘤广泛扩散导致的重大疾病不等。尽管据报道五年无事件生存率总体有所提高，但这是以显著威胁生命的治疗相关副作用为代价的，且对死亡率的亚组分析显示，较低风险的良性形式治愈率较高，而高风险组的改善甚微，两者之间存在不一致。