/PRNewswire/ -- Rona Therapeutics Inc., a global leader in RNA-targeted therapeutics, announced the clearance of an Investigational New Drug (IND) by the U.S. Food and Drug Administration (FDA) for RN0361-an APOC3 targeted siRN

/PRNewswire/ -- 全球RNA靶向治疗领域的领导者罗纳治疗公司（Rona Therapeutics Inc.）宣布，其针对APOC3的siRNA药物RN0361已获得美国食品药品监督管理局（FDA）的新药临床试验（IND）批准。

small interfering RNA) therapeutic for severe hypertriglyceridemia (SHTG), mixed dyslipidemia, and familial chylomicronemia syndrome (FCS) treatment. The company plans to advance RN0361 development in HTG patients based on a favorable safety profile and sustained triglyceride (TG) reduction in a 6-month single dose phase 1 clinical trial.

小干扰RNA）用于治疗严重高甘油三酯血症（SHTG）、混合型血脂异常和家族性乳糜微粒血症综合征（FCS）。公司计划基于在6个月单剂量1期临床试验中表现出的良好安全性及持续的甘油三酯（TG）降低效果，推进RN0361在高甘油三酯血症患者中的开发。

The upcoming randomized, double-blind, placebo controlled, multicenter global Ph2 study will evaluate RN0361 efficacy, safety and duration in hypertriglyceridemia patient population for nine-month follow-up period..

即将进行的随机、双盲、安慰剂对照、多中心全球二期研究将评估RN0361在高甘油三酯血症患者群体中的疗效、安全性和持续时间，随访期为九个月。

RN0361 is a potential best-in-clas

RN0361 是一款潜在的同类最佳

POC3 target siRNA utilizes Rona's proprietary siRNA conjugate and oligo chemistry platform GAIA to enable robust and durable hepatocyte-specific silencing via subcutaneous administration. The therapy achieves durable gene silencing through optimized chemical modifications, significantly silencing APOC3 mRNA and reducing the expression of APOC3 protein..

POC3靶向siRNA利用Rona专有的siRNA偶联和寡核苷酸化学平台GAIA，通过皮下注射实现强大且持久的肝细胞特异性沉默。该疗法通过优化的化学修饰实现持久的基因沉默，显著抑制APOC3 mRNA并降低APOC3蛋白的表达。

'This IND submission underscores our commitment to advance transformative RNAi therapies for patients with severe lipid disorders,' said

“此次IND提交表明了我们致力于为严重脂质紊乱患者推进变革性RNAi疗法的承诺，”

Stella Shi

史黛拉 Shi

, CEO and Founder of Rona Therapeutics

，Rona Therapeutics首席执行官兼创始人

. 'RN0361's Phase I data validate its potential as a best-in-class siRNA with durable efficacy and patient-friendly dosing. We look forward to collaborating with global investigators and agency to accelerate its development.'

‘RN0361’的I期数据验证了其作为同类最佳siRNA的潜力，具有持久的疗效和患者友好的给药方式。我们期待与全球研究者和机构合作，加速其开发进程。

, CMO and EVP of translational medicine

，首席医学官兼转化医学执行副总裁

, was excited to share: 'This Phase 2 study of RN0361 in dysmetabolic patients with hypertriglyceridemia will built on the evidence for RN0361 as an important therapeutic for multiple populations with significant hypertriglyceridemia and its pathologic consequences.' He added, 'The remarkable potency and durability of triglyceride reduction already seen in the First in Human single dose study offers patients a potential powerful tool in the management of a substantial unmet need.'.

，兴奋地分享道：‘这项关于RN0361在患有高甘油三酯血症的代谢紊乱患者中的二期研究，将为RN0361作为治疗多种人群显著高甘油三酯血症及其病理后果的重要疗法提供证据。’他补充道：‘在首次人体单剂量研究中已经观察到的甘油三酯显著降低的效力和持久性，为患者提供了一个潜在的强大工具，以应对一项重要的未满足需求。’

About Rona Therapeutics

关于罗纳治疗学

Rona Therapeutics is a global leader in nucleic acid innovative drug platform company, specializing in the treatment of metabolic and neurological diseases. Rona Therapeutics is always committed to developing the best and first-in-class siRNA drugs with differentiation and innovation to address unmet needs and improve outcome in cardiovascular diseases, obesity, and MASH.

朗润药业是全球领先的核酸创新药物平台公司，专注于代谢和神经类疾病治疗。朗润药业始终致力于开发具有差异化和创新性的最佳同类小干扰RNA（siRNA）药物，以满足未竟需求并改善心血管疾病、肥胖症和MASH的治疗效果。

In addition, Rona Therapeutics is unlocking the potential of extra-hepatic delivery for neurological disorders and adipose/muscle delivery or various metabolic syndromes..

此外，Rona Therapeutics 还在挖掘针对神经系统疾病的肝外递送潜力，以及针对多种代谢综合征的脂肪/肌肉递送潜力。