PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) announced today that it has dosed the first subjects in a Phase 1/2a clinical trial (NCT06209177) of ARO-CFB, the company’s investigational RNA interference (RNAi) therapeutic, in up to 66 healthy volunteers and patients with complement mediated kidney disease..

加利福尼亚州帕萨迪纳市（商业新闻短讯）--箭头制药公司（NASDAQ:ARWR）今天宣布，它已经在ARO-CFB（该公司的研究性RNA干扰（RNAi）治疗剂）的1/2a期临床试验（NCT06209177）中为多达66名健康志愿者和补体介导的肾脏疾病患者服用了第一批受试者。。

James Hamilton, M.D., MBA, Chief of Discovery and Translational Medicine at Arrowhead, said: “In preclinical studies, ARO-CFB achieved deep and durable reductions in liver production of complement factor B, which is involved in alternative complement pathway activation and associated with pathogenesis of diseases involving complement activation.

Arrowhead的发现和转化医学主管詹姆斯·汉密尔顿（JamesHamilton）医学博士说：“在临床前研究中，ARO-CFB实现了补体因子B肝脏产生的深度和持久减少，补体因子B参与替代补体途径激活，并与涉及补体激活的疾病的发病机制有关。

ARO-CFB is our second clinical program designed to address diseases associated with activation of the complement pathway, the first being ARO-C3, our Phase 1 program targeting complement component 3. We look forward to further progressing both programs to investigate whether these candidates can address the substantial unmet medical need that remains in the treatment of multiple complement mediated diseases.”.

ARO-CFB是我们的第二个临床计划，旨在解决与补体途径激活相关的疾病，第一个是ARO-C3，我们针对补体成分3的第一阶段计划。我们期待着进一步推进这两个项目，以调查这些候选人是否能够解决治疗多种补体介导的疾病仍然存在的大量未满足的医疗需求。”。

ARO-CFB is designed to reduce hepatic expression of complement factor B (CFB), which plays an important regulatory role in amplifying complement alternative pathway activation and has been identified as a promising therapeutic target. ARO-CFB is being developed as a potential treatment for complement mediated kidney diseases such as immunoglobulin A nephropathy (IgAN), which is the most common glomerular disease worldwide and carries a high lifetime risk of progression to end-stage renal disease.

ARO-CFB旨在降低补体因子B（CFB）的肝脏表达，补体因子B在扩增补体替代途径激活中起着重要的调节作用，已被确定为一种有前途的治疗靶点。ARO-CFB正在被开发为补体介导的肾脏疾病的潜在治疗方法，例如免疫球蛋白a肾病（IgAN），这是世界上最常见的肾小球疾病，终生罹患终末期肾病的风险很高。

Additionally, ARO-CFB may have clinical applications in non-renal diseases involving complement activation..

此外，ARO-CFB可能在涉及补体激活的非肾脏疾病中具有临床应用。。

About Arrowhead Pharmaceuticals

关于箭头制药

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes.

箭头制药公司通过沉默导致顽固性疾病的基因来开发治疗顽固性疾病的药物。使用广泛的RNA化学组合和有效的递送方式，箭头疗法触发RNA干扰机制，以诱导靶基因的快速，深入和持久的敲低。

RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing..

RNA干扰（RNAi）是活细胞中存在的一种机制，可抑制特定基因的表达，从而影响特定蛋白质的产生。Arrowhead基于RNAi的疗法利用了这种基因沉默的自然途径。。