Orna Therapeutics在美国基因与细胞治疗学会年会上展示了支持其体内CAR疗法在自身免疫疾病中的新临床前数据-动脉网

Orna Therapeutics Presents New Preclinical Data Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases at the American Society of Gene and Cell Therapy Annual Meeting

CISION 等信源发布 2025-05-15 21:00

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WATERTOWN, Mass.

马萨诸塞州沃特敦

，

May 15, 2025

2025年5月15日

/PRNewswire/ -- Orna Therapeutics, a leading biotechnology company developing a proprietary pipeline of

/PRNewswire/ -- Orna Therapeutics，一家开发专有管道的领先生物技术公司

in vivo

体内

therapies across a broad range of autoimmune and oncology indications, today announced the presentation of new preclinical data supporting its

跨越广泛的自身免疫和肿瘤学适应症的疗法，今天宣布了支持其的新临床前数据的展示

in vivo

体内

CAR therapy approach in autoimmune diseases during an oral session at the 28

在第28届会议的口头报告中，关于自身免疫疾病中的CAR疗法方法

American Society of Gene & Cell Therapy (ASGCT) Annual Meeting being held

美国基因与细胞治疗学会（ASGCT）年会正在举行。

May 13-17, 2025

2025年5月13日至17日

, in

，在

New Orleans, Louisiana

路易斯安那州新奥尔良

。

'The preclinical data presented today at ASGCT highlight our potential to deliver on the promise of

“今天在ASGCT上展示的临床前数据突显了我们实现承诺的潜力

in vivo

体内

CAR T therapy,' said

“CAR T细胞疗法，”他说

Joseph Bolen

约瑟夫·博伦

, Ph.D., Chief Executive Officer of Orna Therapeutics. 'Our CD19 panCAR program has demonstrated not only successful delivery of our lead panCAR LNP to disease-relevant immune cell types, but also robust and sustained B cell depletion at low doses in both peripheral blood and lymphoid tissues in non-human primates (NHPs).

Orna Therapeutics首席执行官博士表示：“我们的CD19 panCAR项目不仅成功地将我们的主要panCAR LNP递送到了与疾病相关的免疫细胞类型，还在非人灵长类动物（NHP）的外周血和淋巴组织中以低剂量实现了强劲且持续的B细胞清除。”

These compelling results continue to reinforce our commitment to translating our promising science into meaningful therapies for patients and we look forward to advancing our CD19 panCAR program towards the clinic in 2026.'.

这些令人信服的结果继续坚定我们致力于将我们充满前景的科学转化为对患者有意义的疗法，我们期待在2026年推进我们的CD19泛CAR项目进入临床。

Presentation Details:

演示详情：

Title

标题

：

In Vivo

体内

panCAR™ Therapy Using Circular RNA for the Treatment of Autoimmune Disease

使用环状RNA治疗自身免疫性疾病的panCAR™疗法

Speaker

扬声器

：

Megan Hoban

梅根·霍班

, Ph.D., panCAR Program Lead, Orna Therapeutics

博士，panCAR项目负责人，Orna Therapeutics公司

Date/Time

日期/时间

：

Thursday, May 15, 2025

2025年5月15日，星期四

，

8:00 AM

早上8点

–

9:45 AM CDT

上午9点45分（中部夏令时间）

Session Name

会话名称

: Cellular and Gene Therapies for Autoimmune Disease

：自身免疫性疾病的细胞和基因疗法

Location

位置

: Room 388-390

房间388-390

In today's presentation, Orna will showcase preclinical data demonstrating the potential of its

在今天的演讲中，奥尔娜将展示预临床数据，证明其潜力

in vivo

体内

panCAR therapy, enabled by its proprietary circular (oRNA®) technology and best-in-class lipid nanoparticle (LNP) delivery system to achieve robust and sustained B cell depletion in both humanized mouse models and non-human primates across multiple doses.

panCAR疗法，依托其专有的环状RNA（oRNA®）技术以及一流的脂质纳米颗粒（LNP）递送系统，能够在人源化小鼠模型和非人灵长类动物中实现强效且持久的B细胞耗竭，并在多剂量下发挥效果。

Key findings from the study include:

研究的主要发现包括：

Validated extra-hepatic delivery

验证了肝外递送

to disease-relevant immune cell types, including T cells, in mice and NHPs without requiring targeting ligands.

与疾病相关的免疫细胞类型，包括小鼠和非人灵长类动物中的T细胞，而无需靶向配体。

Lead panCAR LNP achieved over 60% delivery

铅 panCAR LNP 实现了超过 60% 的递送率

to peripheral blood and splenic T cells in NHPs.

外周血和脾脏T细胞在非人灵长类动物中。

CD19 panCAR

CD19泛CAR

doses as low as 0.03mpk led robust B cell depletion

剂量低至0.03mpk即可导致显著的B细胞耗竭

, with multi-dosing achieving increased B cell depletion in humanized mice.

，多剂量给药在人源化小鼠中实现了更显著的B细胞耗竭。

In a humanized lupus mouse model,

在人源化狼疮小鼠模型中，

CD19 panCAR

CD19 全CAR

showed strong B cell depletion and a meaningful and differentiated reduction in dsDNA titers

显示了强烈的B细胞耗竭，并且dsDNA滴度有显著且差异化的降低

compared to rituximab.

与利妥昔单抗相比。

CD19 panCAR induced full depletion of B cells across peripheral blood, spleen, lymph nodes, and bone marrow in NHPs

CD19 panCAR在NHP中诱导了外周血、脾脏、淋巴结和骨髓中B细胞的完全耗竭。

, with peripheral B cells beginning to reconstitute after three weeks.

，四周后外周B细胞开始重建。

About Orna Therapeutics

关于奥尔纳治疗学

Orna Therapeutics is dedicated to designing and delivering a new class of fully engineered circular RNA (oRNA

奥尔纳治疗公司致力于设计和提供一类全新的完全工程化的环状RNA（oRNA）。

) therapeutics to unlock the potential of RNA medicine to treat diseases anywhere in the body. Orna's oRNA transcripts have advantages over traditional mRNA approaches, including simplified production, improved formulation into lipid nanoparticles, and superior protein expression. Its industry-leading LNP-based delivery systems, comprehensive editing programs and validating large pharma partnerships position Orna to advance novel RNA medicines with vast potential to transform patient care.

）治疗学，以释放RNA药物在身体任何部位治疗疾病的潜力。Orna的oRNA转录本相比传统的mRNA方法具有多项优势，包括简化的生产流程、改进的脂质纳米粒制剂以及更优越的蛋白质表达。其行业领先的基于LNP的递送系统、全面的编辑项目以及与大型制药公司的合作验证，使Orna能够推进具有巨大潜力的新型RNA药物，从而彻底改变患者护理。

To learn more, visit .

要了解更多信息，请访问。

www.ornatx.com

and follow Orna Therapeutics on

并关注奥尔纳治疗公司

and

和

领英

。

Orna Therapeutics Investor Contact:

奥尔纳治疗公司投资者联系方式：

Alex Lobo