科罗拉多大学安舒茨盖茨研究所获得美国首个FDA批准的校园研发CAR T细胞疗法 clearance

Novel treatment moves into first clinical trial for adults with hard-to-treat acute myeloid leukemia

新型治疗进入针对难治性急性髓系白血病成人的首次临床试验

AURORA, Colo.

科罗拉多州奥罗拉市

,

，

April 2, 2026

2026年4月2日

/PRNewswire/ -- The

/PRNewswire/ -- The

University of Colorado Anschutz Gates Institute

科罗拉多大学安舒茨盖茨研究所

has received Investigational New Drug clearance from the U.S. Food and Drug Administration, marking the first chimeric antigen receptor (CAR) T-cell therapy authorized for clinical testing in the United States that was developed entirely on the

已获得美国食品和药物管理局的新药临床试验许可，这标志着首个完全在美国本土开发的嵌合抗原受体（CAR）T细胞疗法获准进行临床测试。

CU Anschutz

CU 安舒茨

campus. The therapy, engineered to attack a key protein called CD64 found on aggressive leukemia cells, represents a novel treatment approach for patients with relapsed or refractory disease.

校园。这种疗法经过设计，可以攻击在侵袭性白血病细胞上发现的一种名为CD64的关键蛋白，为复发或难治性疾病的患者提供了一种新颖的治疗途径。

Researchers at The University of Colorado Anschutz Gates Institute

科罗拉多大学安舒茨盖茨研究所的研究人员

Researchers working in the lab at The University of Colorado Anschutz Gates Institute

在科罗拉多大学安舒茨盖茨研究所实验室工作的研究人员

'This work was built on the seminal findings of

“这项工作建立在具有开创性意义的发现基础之上，

Craig Jordan's

克雷格·乔丹的

laboratory, identifying CD64 as a marker of leukemia cells that escape modern therapies,' said the inventor of the therapy,

实验室，将CD64鉴定为逃逸现代疗法的白血病细胞的标志物，”该疗法的发明者表示，

M. Eric Kohler, MD, PhD

M. 艾瑞克·科勒，医学博士，哲学博士

, an assistant professor of pediatrics in hematology, oncology, and bone marrow transplantation at

，儿科血液学、肿瘤学和骨髓移植助理教授

CU Anschutz School of Medicine

科罗拉多大学安舒茨医学院

and

和

Children's Hospital Colorado

科罗拉多儿童医院

. Spearheaded by Haley Simpson, MD, PhD, a clinical fellow working in the Kohler lab, the CD64-directed CAR underwent years of testing, engineering, and optimization in the laboratory before making it to this point.

在科勒实验室工作的临床研究员海莉·辛普森医学博士、哲学博士的带领下，这款靶向CD64的CAR在实验室中经历了多年的测试、工程设计和优化才达到目前的阶段。

'Advancing this therapy from an idea to a first-in-human clinical trial reflects the collaborative nature and diverse expertise on campus,' said Kohler. 'From the basic science departments to the translational teams of Gates Institute, to the clinical expertise in cellular therapy, bringing new therapies forward is what the Gates Institute is built to do.'.

“将这种疗法从一个想法推进到首次人体临床试验，体现了校园内协作的性质和多样化的专业能力，”科勒说。“从基础科学部门到盖茨研究所的转化团队，再到细胞治疗的临床专业知识，推动新疗法的发展正是盖茨研究所的使命所在。”

The first-in-human Phase 1 clinical trial, starting this summer, will be available to adults with relapsed or refractory acute myeloid leukemia (AML).

今年夏天开始的首次人体一期临床试验，将面向复发或难治性急性髓系白血病（AML）的成人患者。

'This FDA clearance represents years of work by a team committed to moving cellular therapies from discovery into meaningful clinical impact,' said

“这一FDA的批准代表了团队多年的工作成果，他们致力于将细胞疗法从发现阶段推进到产生有意义的临床影响，”

Terry Fry, MD

特里·弗莱，医学博士

, executive director of the Gates Institute. 'Launching this clinical trial is an important first step toward a different approach for this very aggressive disease, one we hope will ultimately change how myeloid leukemias are treated.'

，盖茨研究所的执行董事。“启动这项临床试验是针对这种非常侵袭性疾病的不同方法的重要第一步，我们希望这最终将改变骨髓性白血病的治疗方式。”

The Phase 1 clinical trial will evaluate the safety, tolerability and optimal dosing of CD64 CAR T cells in adults with relapsed or refractory AML. Enrollment is anticipated to begin in June 2026, with patients treated at

第一阶段临床试验将评估CD64 CAR T细胞在复发或难治性急性髓系白血病（AML）成年患者中的安全性、耐受性和最佳剂量。预计将于2026年6月开始招募患者，并在以下地点进行治疗：

UCHealth University of Colorado Hospital

UCHealth科罗拉多大学医院

.

。

A pediatric clinical trial, led by

由以下人员领导的儿科临床试验，

Sanam Shahid, MD

Sanam Shahid，医学博士

, at Children's Hospital Colorado, is expected to begin later this year expanding potential treatment options for children and adolescents.

，科罗拉多州儿童医院预计将在今年晚些时候开始，为儿童和青少年扩展潜在的治疗选择。

For patients facing relapsed or refractory AML, treatment options remain extremely limited and outcomes are poor. This trial offers a potential new pathway for patients with this aggressive blood cancer.

对于面临复发或难治性急性髓系白血病（AML）的患者，治疗选择仍然非常有限，预后也很差。这项试验为患有这种侵袭性血癌的患者提供了一条潜在的新途径。

'This marks a major milestone for The University of Colorado Anschutz Gates Institute,' said

“这标志着科罗拉多大学安舒茨盖茨研究所的一个重要里程碑，”

Mathew Angelos, MD, PhD

马修·安杰洛斯，医学博士，哲学博士

, assistant professor of medicine in hematology at the CU Anschutz School of Medicine and principal investigator of the adult clinical trial. 'This achievement reflects tremendous dedication and collaboration across our scientific, clinical and regulatory teams. I'm excited to lead this study and believe it has the potential to meaningfully impact patients with difficult-to-treat myeloid blood cancers, where new treatment strategies are urgently needed.'.

，科罗拉多大学安舒茨医学院血液学医学助理教授，成人临床试验的首席研究员。“这一成就反映了我们科学、临床和监管团队的巨大奉献精神和协作。我非常高兴能领导这项研究，并相信它有潜力对难治性髓系血液癌症患者产生有意义的影响，因为这些患者迫切需要新的治疗策略。”

Fry agreed.

弗莱同意了。

'This achievement highlights the strength of our growing cell and gene therapy ecosystem and the collaborative expertise required to advance a novel therapy from discovery to clinical testing,' he said. 'It's a powerful example of how teams across campus are working together to translate science into meaningful impact for patients.'.

“这一成就突显了我们不断发展的细胞和基因治疗生态系统的实力，以及将一种新型疗法从发现推进到临床测试所需的协作专长，”他表示。“这是校园内各团队如何共同努力将科学转化为对患者有意义的影响的有力例证。”

Manufacturing of the CAR T-cell product for the trial will take place at the

试验用CAR T细胞产品的生产将在

Gates Biomanufacturing Facility

盖茨生物制造设施

, supporting a seamless transition from laboratory research to clinical application.

，支持从实验室研究到临床应用的无缝过渡。

About the University of Colorado Anschutz

关于科罗拉多大学安舒茨分校

The University of Colorado Anschutz is a world-class academic medical campus leading transformative advances in science, medicine, education and patient care. The campus includes the University of Colorado's health professional schools, more than 60 centers and institutes, and two nationally ranked independent hospitals - .

科罗拉多大学安舒茨校区是一个世界级的学术医学园区，在科学、医学、教育和患者护理方面引领变革性进步。该校区包括科罗拉多大学的健康专业学院、60多个中心和研究所，以及两家全国排名独立医院。

UCHealth University of Colorado Hospital

UCHealth科罗拉多大学医院

and

和

Children's Hospital Colorado

科罗拉多儿童医院

- which see nearly three million adult and pediatric patient visits each year. Innovative, interconnected and highly collaborative, CU Anschutz delivers life-changing treatments, exceptional patient care and top-tier professional training. The campus conducts world-renowned research supported by $890 million in funding, including $762 million in sponsored awards and $128 million in philanthropic gifts for research..

- 每年接待近三百万名成人和儿童患者。创新、互联且高度协作的CU安舒茨校区提供改变生命的治疗、卓越的患者护理以及顶级的专业培训。该校区开展世界知名的研究，获得8.9亿美元的资金支持，其中包括7.62亿美元的赞助奖项和1.28亿美元用于研究的慈善捐赠。

About Gates Institute

关于盖茨研究所

Gates Institute is a premier cell therapy translational research institute with biomanufacturing capabilities delivering first-in-human therapies. Based at the University of Colorado Anschutz, we are part of a rich biomedical ecosystem. We bring together and support researchers and clinicians who specialize in regenerative, cell and gene therapies, accelerating their discoveries from concepts to cures..

盖茨研究所是一家顶级的细胞治疗转化研究机构，具备生物制造能力，可提供首次应用于人体的疗法。我们位于科罗拉多大学安舒茨分校，是丰富的生物医学生态系统的一部分。我们汇集并支持专注于再生医学、细胞和基因治疗的研究人员和临床医生，加速他们的发现从概念到治愈的进程。

Find the latest CU Anschutz news

查找最新的CU安舒茨新闻

here

这里

.

。

Contact:

联系人：

Laura Kelley, CU Anschutz

劳拉·凯利，科罗拉多大学安舒茨分校

(303) 704-5222,

(303) 704-5222,

[email protected]

电子邮件地址

SOURCE University of Colorado Anschutz

来源：科罗拉多大学安舒茨分校

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