LONDON--(BUSINESS WIRE)--MiNA Therapeutics Limited, the pioneer in small activating RNA (RNAa) therapeutics, announced today that it will present new pre-clinical data for its lead RNA activation program targeting fetal hemoglobin (HbF) at the Keystone Symposia’s Delivery of Nucleic Acid Therapeutics Conference in Banff, Alberta, Canada.

伦敦--（商业新闻短讯）--MiNA Therapeutics Limited是小激活RNA（RNAa）治疗学的先驱，今天宣布，它将在加拿大阿尔伯塔省班夫举行的Keystone Symposia核酸治疗学会议上，为其针对胎儿血红蛋白（HbF）的主要RNA激活计划提供新的临床前数据。

Pre-clinical studies confirmed in vivo delivery of MiNA’s RNAa therapeutics to erythroid progenitor cells in bone marrow at levels that are clinically meaningful for the treatment of beta-hemoglobinopathies. The data will be presented on January 24, 2024, at 7:30 pm MST during a poster presentation..

临床前研究证实，MiNA的RNAa治疗剂体内递送至骨髓中的红系祖细胞，其水平对治疗β血红蛋白病具有临床意义。数据将于2024年1月24日下午7:30在MST的海报展示中呈现。。

MiNA’s HbF program is designed to increase transcription of the gamma globin (HBG) gene, enabling patients with beta-hemoglobinopathies to produce enhanced levels of HbF. HbF is a compensatory form of hemoglobin which has the potential to achieve a functional cure in patients with severe inherited blood disorders such as sickle cell disease and beta thalassemia..

MiNA的HbF计划旨在增加γ-珠蛋白（HBG）基因的转录，使β-血红蛋白病患者产生更高水平的HbF。HbF是血红蛋白的一种代偿形式，有可能在患有严重遗传性血液疾病（如镰状细胞病和β地中海贫血）的患者中实现功能性治愈。。

“These results demonstrate for the first time efficient in vivo delivery of RNAa therapeutics to erythroid progenitor cells with a delivery technology that is well-suited for safe and effective treatment in the clinic,” said Robert Habib, CEO of MiNA Therapeutics. “We are excited by this compelling evidence, which strongly supports advancement of our lead program for the treatment of sickle cell disease and beta thalassemia, which together affect more than five million people around the world.”.

MiNA therapeutics首席执行官罗伯特·哈比卜（RobertHabib）表示：“这些结果首次证明了RNAa治疗剂通过一种非常适合临床安全有效治疗的递送技术在体内有效地传递给红系祖细胞。”。“我们对这一令人信服的证据感到兴奋，这有力地支持了我们治疗镰状细胞病和β地中海贫血的领先项目的进展，这些疾病共同影响着全世界500多万人。”。

MiNA’s HbF program uses a liposomal delivery technology, NOV340, which efficiently delivers RNAa therapeutics in vivo without the need for harmful pre-conditioning or complex cell engineering. NOV340 formulations have established safety and pharmacodynamic activity in clinical trials involving more than 290 patients, including 130 patients treated with MiNA’s first RNAa development candidate, MTL-CEBPA.

MiNA的HbF计划使用脂质体递送技术NOV340，该技术在体内有效递送RNAa治疗剂，而不需要有害的预处理或复杂的细胞工程。NOV340制剂已在涉及290多名患者的临床试验中建立了安全性和药效学活性，其中包括130名接受MiNA第一个RNAa开发候选药物MTL-CEBPA治疗的患者。

MiNA anticipates advancing its HbF program, the first program to emerge from its genetic medicine portfolio, into pre-clinical development in 2024..

MiNA预计将在2024年将其HbF计划（第一个从其遗传医学组合中产生的计划）推进临床前开发。。

In the presentation, entitled “NOV340 Liposome Encapsulating Nucleic Acid Payload Achieves Efficient Biodistribution to Erythroid Progenitor Cells,” data from animal models confirmed in vivo delivery to erythroid progenitor cells at levels that met accepted benchmarks for impactful treatment of beta-hemoglobinopathies.

在题为“封装核酸有效载荷的NOV340脂质体实现了对红系祖细胞的有效生物分布”的演讲中，来自动物模型的数据证实了体内向红系祖细胞的递送水平符合有效治疗β血红蛋白病的公认基准。

In non-human primates, intravenous administration of encapsulated liposomes resulted in delivery of MiNA’s RNAa compound to over 60% of committed colony-forming unit erythroid (CFU-E) cells and pro-erythroblasts (Pro-E) in bone marrow. Equivalent levels of delivery efficiency were observed in peripheral blood monocytes, a cell type in which pharmacodynamic activity of NOV340-formulated RNAa therapeutics has previously been shown in clinical studies..

在非人灵长类动物中，静脉内施用包封的脂质体导致MiNA的RNAa化合物递送至骨髓中超过60%的定型集落形成单位红细胞（CFU-E）细胞和促红细胞（pro-E）。在外周血单核细胞中观察到等效水平的递送效率，该细胞类型先前已在临床研究中显示了NOV340配制的RNAa治疗剂的药效学活性。。

Following the symposia, the poster detailing full study results will be available on the MiNA website: www.minatx.com.

研讨会结束后，详细介绍完整研究结果的海报将在MiNA网站www.minax.com上发布。

MiNA has previously achieved clinical proof of concept for its RNAa therapeutics platform with MTL-CEBPA. MiNA is exploring out-licensing opportunities for MTL-CEBPA and its immuno-oncology portfolio, which uniquely combines the capability to specifically restore or boost any dysregulated gene target with clinically validated in vivo delivery to myeloid immune cells..

MiNA之前已经通过MTL-CEBPA实现了其RNAa治疗平台的临床概念验证。MiNA正在探索MTL-CEBPA及其免疫肿瘤学组合的许可机会，该组合将特异性恢复或增强任何失调基因靶标的能力与临床验证的体内递送至髓系免疫细胞的能力独特地结合起来。。

About MiNA Therapeutics

关于MiNA Therapeutics

MiNA Therapeutics is the global leader in small activating RNA therapeutics or RNAa. Harnessing innate mechanisms of gene activation, RNAa therapeutics are a revolutionary new class of medicines that can restore or boost normal function of genes and thereby protein-modulated pathways in cells. We are advancing a proprietary pipeline of new medicines with an initial focus on genetic medicine, while collaborating with leading pharmaceutical companies to apply our technology platform across a broad range of other therapeutic areas.

MiNA Therapeutics是小激活RNA疗法或RNAa的全球领导者。利用基因激活的先天机制，RNAa疗法是一类革命性的新型药物，可以恢复或增强基因的正常功能，从而恢复或增强细胞中蛋白质调节的途径。我们正在推进新药的专有渠道，最初专注于遗传医学，同时与领先的制药公司合作，将我们的技术平台应用于广泛的其他治疗领域。

Based on our unique know-how in RNA activation, we are expanding the possibilities of RNA-based medicine. www.minatx.com..

基于我们在RNA激活方面的独特技能，我们正在扩大基于RNA的医学的可能性。http://www.minatx.com。。