ASGCT 2025聚焦基因与细胞疗法：应对罕见病、自身免疫疾病等：-动脉网

Gene and Cell Therapies in Focus at ASGCT 2025: Tackling Rare Diseases, Autoimmune Conditions, and More Related posts:

GeneOnline 等信源发布 2025-05-14 18:21

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by Bernice Lottering

作者：贝妮丝·洛特林

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his year’s American Society of Gene and Cell Therapy (

今年的美国基因与细胞治疗学会 (

ASGCT

) Annual Meeting, held May 13–17 in New Orleans, is buzzing with new breakthroughs from the gene and cell therapy world. Biotech companies like Immusoft, Myrtelle, and Sail Biomedicines are presenting data that could reshape how we treat a range of tough-to-tackle diseases—from rare inherited disorders to autoimmune conditions.

）年度会议于 5 月 13 日至 17 日在新奥尔良举行，基因和细胞治疗领域的新突破成为热议话题。Immusoft、Myrtelle 和 Sail Biomedicines 等生物技术公司正在展示可能重塑我们治疗各种棘手疾病方式的数据——从罕见的遗传病到自身免疫性疾病。

With new approaches like engineered B cells, tunable gene therapies, and RNA-based treatments, there’s a lot of momentum behind these next-gen innovations. Here’s a look at how these companies are aiming to change the game..

随着工程化B细胞、可调节基因疗法和基于RNA的治疗等新方法的出现，这些下一代创新背后有着很大的推动力。以下是这些公司如何试图改变游戏规则的展望。

Innovative Therapies for Rare Genetic Disorders: Immusoft’s Pioneering B Cell Therapy for MPS I

罕见遗传病的创新疗法：Immusoft 的 MPS I 先锋 B 细胞疗法

Immusoft, a California-based cell therapy innovator, is

Immusoft，一家位于加利福尼亚的细胞治疗创新公司，是

presenting

呈现

the world’s first clinical trial data for engineered B cells targeting

世界上首个针对工程化B细胞的临床试验数据

mucopolysaccharidosis type I

黏多糖病I型

(

MPS I

)

, a rare genetic disorder caused by a deficiency in the enzyme iduronidase. This condition triggers a harmful buildup of substances, leading to severe developmental and physical challenges. At ASGCT 2025, Paul J. Orchard, MD, from the University of Minnesota, will share results from a first-in-human trial, highlighting the safety and early efficacy of autologous B cells genetically modified to produce iduronidase.

，一种由艾杜糖醛酸酶缺乏引起的罕见遗传病。该病症会引发有害物质的积累，导致严重的发育和身体障碍。在2025年ASGCT会议上，来自明尼苏达大学的保罗·J·奥查德博士将分享一项首次人体试验的结果，重点介绍经过基因改造以生产艾杜糖醛酸酶的自体B细胞的安全性和早期疗效。

This novel approach could transform therapeutic protein delivery and offers a promising path for MPS I patients..

这种新颖的方法可以改变治疗性蛋白质的递送方式，并为MPS I患者提供了充满希望的途径。

Myrtelle’s Gene Therapy for Canavan Disease

Myrtelle的Canavan病基因疗法

In a related area of research,

在相关研究领域，

Myrtelle

is working on a potential treatment for Canavan disease—a rare and fatal genetic disorder that appears in early childhood. The disease is caused by mutations in the

正在研究一种潜在的 Canavan 病治疗方法——这是一种在幼儿期出现的罕见且致命的遗传疾病。该疾病由以下基因突变引起：

ASPA

gene, which prevents the brain from developing properly and disrupts the formation of myelin, the protective coating around nerve fibers. Without enough myelin, the nervous system cannot function correctly. Myrtelle’s experimental gene therapy, called rAAV-Olig001-ASPA, uses a specially designed viral vector to deliver a healthy version of the .

基因，它阻止大脑正常发育，并破坏神经纤维周围保护性髓鞘的形成。没有足够的髓鞘，神经系统就无法正常运作。Myrtelle 的实验性基因疗法名为 rAAV-Olig001-ASPA，使用一种特别设计的病毒载体来传递健康的。

ASPA

gene directly to brain cells, with the goal of restoring normal function. At ASGCT, the company will also highlight its participation in the FDA’s START Pilot Program, which offers faster and more flexible regulatory guidance for developing new treatments.

将基因直接递送到脑细胞，目标是恢复其正常功能。在ASGCT会议上，该公司还将强调其参与FDA的START试点计划，该计划为开发新疗法提供更快、更灵活的监管指导。

CEO Adrian Stecyk

首席执行官阿德里安·斯特奇克

emphasized how this program is helping move their therapy forward more quickly for families affected by this devastating condition. With Orphan Drug and Fast Track designations—intended to support treatments for serious and rare diseases—this approach could help address a significant gap in available options for Canavan disease..

强调了这个项目如何帮助那些受这种毁灭性疾病影响的家庭更快地推进治疗。凭借孤儿药和快速通道资格——旨在支持针对严重和罕见疾病的治疗——这种方法可能有助于填补Canavan病可用治疗选项中的重要空白。

Revolutionizing Autoimmune Disease Treatment—Sail Biomedicines’ In Vivo CAR-T Therapy

革新自身免疫疾病治疗——Sail生物医药公司的体内CAR-T疗法

Sail Biomedicines is

帆生物医学是

presenting preclinical data

呈现临床前数据

on SAIL-0804, a groundbreaking in vivo CAR-T therapy for autoimmune diseases. CAR-T therapy, traditionally used in cancer treatment, involves harvesting a patient’s T cells, genetically modifying them, and reintroducing them to target specific cells in the body. However, this process is complex, requiring cell harvesting and laboratory-based modifications.

在SAIL-0804上，这是一种突破性的用于治疗自身免疫性疾病的体内CAR-T疗法。CAR-T疗法传统上用于癌症治疗，涉及采集患者的T细胞，对其进行基因改造，然后重新注入体内以靶向特定细胞。然而，这一过程复杂，需要进行细胞采集和基于实验室的改造。

In contrast, SAIL-0804 uses targeted lipid nanoparticles to deliver circular RNA (eRNA) directly into the body, which temporarily reprograms T cells to deplete B cells. B cells are a type of immune cell that can contribute to autoimmune diseases, where the body’s immune system mistakenly attacks its own tissues..

相比之下，SAIL-0804 使用靶向脂质纳米颗粒将环状 RNA（eRNA）直接递送到体内，暂时重新编程 T 细胞以消耗 B 细胞。B 细胞是一种免疫细胞，可能参与自身免疫疾病，在这类疾病中，身体的免疫系统会错误地攻击自身的组织。

In humanized mouse models, SAIL-0804 successfully achieved complete depletion of B cells across various sites, including the blood, spleen, lymph nodes, and bone marrow. B cells that repopulated after treatment displayed an immature phenotype, suggesting the immune system resets to a more balanced, less harmful state.

在人源化小鼠模型中，SAIL-0804成功实现了在包括血液、脾脏、淋巴结和骨髓等多个部位的B细胞完全耗竭。治疗后重新填充的B细胞表现出不成熟的表型，提示免疫系统重置为更平衡、危害较小的状态。

This is important because it could potentially help in conditions where the immune system is overactive, such as lupus or rheumatoid arthritis..

这很重要，因为它可能有助于免疫系统过度活跃的病症，如狼疮或类风湿性关节炎。

SAIL-0804 simplifies treatment compared to traditional CAR-T therapies, avoiding complex cell-based procedures and lymphodepletion, and enabling outpatient use. This makes it a more accessible and cost-effective option for patients. Sail is now advancing SAIL-0804 into IND-enabling studies, the final step before clinical trials, which could bring this innovative therapy closer to helping patients with autoimmune diseases..

SAIL-0804相较于传统的CAR-T疗法简化了治疗过程，避免了复杂的细胞操作和淋巴清除步骤，并能够实现门诊治疗。这使其成为对患者更易获得且更具成本效益的选择。Sail公司目前正推进SAIL-0804进入IND申报所需的研究，这是临床试验前的最后一步，有望使这一创新疗法更接近于帮助自身免疫疾病患者。

Changing the Game in Rare and Autoimmune Disease Treatments with Gene and Cell Therapies

改变罕见病和自身免疫性疾病治疗的游戏规则：基因与细胞疗法

The landscape of gene and cell therapies is rapidly evolving, with significant strides made in treating rare genetic disorders, autoimmune diseases, and neurological conditions. At the ASGCT 2025 Annual Meeting, companies like Immusoft, Myrtelle, and Sail Biomedicines are presenting groundbreaking approaches, such as engineered B cell therapies, tunable gene therapies, and RNA-based treatments.

基因和细胞治疗的领域正在迅速发展，在治疗罕见遗传疾病、自身免疫疾病和神经系统疾病方面取得了显著进展。在2025年ASGCT年会上，Immusoft、Myrtelle和Sail Biomedicines等公司展示了突破性的方法，如工程化B细胞疗法、可调基因疗法和基于RNA的治疗方法。

These innovations hold promise for transforming the treatment paradigms of challenging diseases..

这些创新有望转变具有挑战性疾病的治疗模式。

The momentum in the field is reflected in recent industry developments. In 2024, the FDA

该领域的势头在最近的行业发展中得到体现。2024年，FDA

approved

已批准

several new gene and cell therapies, including Tecelra, the first T cell receptor gene therapy for synovial sarcoma, and

几种新的基因和细胞疗法，包括Tecelra，这是首个用于治疗滑膜肉瘤的T细胞受体基因疗法，以及

afamitresgene autoleucel

阿法米雷斯基因自体白细胞

, a TCR gene therapy for solid tumors. Additionally, the gene therapy pipeline continues to expand, with over 500 therapies in development and expectations for 10–20 approvals annually by 2025.

，一种针对实体瘤的TCR基因疗法。此外，基因治疗管道持续扩展，目前有超过500种疗法正在开发中，并预计到2025年每年将有10至20个批准。

Looking ahead, the integration of artificial intelligence in gene therapy development is anticipated to

展望未来，人工智能在基因治疗开发中的整合预计将

enhance

增强

decision-making processes, potentially accelerating the approval of treatments for inherited disorders . As these therapies progress from the lab to clinical settings, they offer renewed hope for patients and families affected by previously untreatable conditions.

决策过程，可能加快对遗传病治疗的审批。随着这些疗法从实验室走向临床环境，它们为受以前无法治疗的疾病影响的患者和家庭带来了新的希望。

The advancements presented at

所展示的进展

ASGCT

美国基因与细胞治疗学会

2025 underscore a pivotal moment in the field of gene and cell therapies, signaling a future where personalized, effective treatments for complex diseases are increasingly within reach.

2025年标志着基因和细胞治疗领域的一个关键转折点，预示着针对复杂疾病的个性化、有效治疗将日益触手可及。

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掌握基因与细胞治疗：最大化利用ASGCT内容的蓝图

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2025年ASGH和香港国际医疗展将改变医疗保健格局

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