/PRNewswire/ -- YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for

/PRNewswire/--YolTech Therapeutics是一家致力于提供终身治疗的临床阶段基因编辑公司，宣布启动针对

YOLT-204

YOLT-204

, an investigational therapy for the treatment of transfusion-dependent beta-thalassemia (TDT). TDT is a severe genetic blood disorder where mutations in the beta-globin gene leads to reduced or absence of hemoglobin, a condition where regular blood transfusion is required to manage anemia and prevent other complications..

，一种治疗输血依赖性β地中海贫血（TDT）的研究性疗法。TDT是一种严重的遗传性血液疾病，β-珠蛋白基因突变导致血红蛋白减少或缺乏，这种情况需要定期输血来控制贫血和预防其他并发症。。

YOLT-204

YOLT-204

is a first-in-class

是一流的

in vivo

体内

gene editing therapy leveraging YolTech's proprietary lipid nanoparticles (LNP). The therapy edits the regulatory region of hemoglobin to induce expression of fetal hemoglobin, potentially alleviating the imbalance of hemoglobin production and normalizing the number of red blood cells in TDT patients.

利用YolTech专有脂质纳米颗粒（LNP）的基因编辑疗法。该疗法编辑血红蛋白的调节区以诱导胎儿血红蛋白的表达，有可能缓解TDT患者血红蛋白产生的不平衡并使红细胞数量正常化。

In pre-clinical models, .

在临床前模型中。

YOLT-204

YOLT-204

showed effective and sustained induction of fetal hemoglobin, suggesting therapeutic potential for TDT.

显示出有效且持续的胎儿血红蛋白诱导，表明TDT具有治疗潜力。

YOLT-204

YOLT-204

may also be an effective treatment for patients with sickle cell disease (SCD), as increased expression of fetal hemoglobin in these patients has been associated with less polymerization of sickle hemoglobin as well as reduced complications and mortality.

对于镰状细胞病（SCD）患者也可能是一种有效的治疗方法，因为这些患者胎儿血红蛋白表达的增加与镰状血红蛋白聚合的减少以及并发症和死亡率的降低有关。

The clinical trial for

临床试验

YOLT-204

YOLT-204

is a dose-escalation study to preliminarily examine the safety and efficacy of a single-dose regimen with

是一项剂量递增研究，旨在初步检查单剂量方案的安全性和有效性

YOLT-204

YOLT-204

in TDT. If successful,

在TDT中。如果成功，

YOLT-204

YOLT-204

may eventually provide an off-the-shelf curative treatment for TDT patients without conditioning chemotherapy and HSCT (Hematopoietic Stem Cell Transplantation).

可能最终为TDT患者提供现成的治疗方法，而无需调理化疗和HSCT（造血干细胞移植）。

'The initiation of clinical trial for

“开始临床试验

YOLT-204

YOLT-204

represents a significant milestone of gene editing therapy development for TDT and SCD,' said Dr.

代表了TDT和SCD基因编辑疗法发展的一个重要里程碑。

Yuxuan Wu

吴 玉

, founder and Chief Executive Officer of YolTech Therapeutics. 'We are excited to collaborate with our clinical investigators to bring this innovative therapy to patients.'

，YolTech Therapeutics的创始人兼首席执行官我们很高兴与我们的临床研究人员合作，将这种创新疗法带给患者。”

gene editing therapy developed for TDT without conditioning chemotherapy and HSCT. YOLT-204 delivers a gene editor to hematopoietic stem cells through YolTech's proprietary lipid nanoparticles. The editor together with a guide RNA targets the hemoglobin regulatory region to induce expression of fetal hemoglobin.

基因编辑疗法是为TDT开发的，无需调节化疗和HSCT。YOLT-204通过YolTech专有的脂质纳米颗粒为造血干细胞提供基因编辑器。编辑器与指导RNA一起靶向血红蛋白调节区以诱导胎儿血红蛋白的表达。

The expression of fetal hemoglobin has the potential to normalize hemoglobin composition and the red blood cell counts in patients with transfusion-dependent beta-thalassemia, eventually making them transfusion independent..

胎儿血红蛋白的表达有可能使输血依赖性β地中海贫血患者的血红蛋白组成和红细胞计数正常化，最终使他们独立于输血。。