Goldman Sachs has initiated coverage on Avidity Biosciences Inc RNA based on multi-blockbuster, first-to-market commercial opportunities for its investigational candidates.

高盛（Goldman Sachs）基于多部大片开始报道Avidity Biosciences Inc RNA，首次为其研究候选人提供商业机会。

Avidity Biosciences is a clinical-stage biotechnology company that develops RNA therapeutics (antibody oligonucleotide conjugates) for muscular dystrophies.

Avidity Biosciences是一家临床阶段生物技术公司，开发用于肌肉营养不良的RNA治疗剂（抗体寡核苷酸偶联物）。

The company’s lead asset, del-desiran (AOC 1001), is currently in the Phase 3 HARBOR trial in patients with myotonic dystrophy type 1 (DM1).

该公司的主要资产del desiran（AOC 1001）目前正在进行1型强直性肌营养不良（DM1）患者的3期HARBOR试验。

The company’s pipeline includes Del-brax in the Phase 1/2 Fortitude trial for facioscapulohumeral muscular dystrophy (FSHD) and del-zota in a Phase 1/2 Explore44 trial in Duchenne muscular dystrophy (DMD) for patients amenable to exon 44 skipping.

该公司的管道包括面肩肱型肌营养不良症（FSHD）的1/2期强韧试验中的Del brax和Duchenne型肌营养不良症（DMD）的1/2期Explore44试验中的Del zota，适用于适合外显子44跳跃的患者。

Goldman Sachs has initiated with a Buy rating and a price target of $59.

高盛（Goldman Sachs）最初的评级为买入，目标价格为59美元。

The analyst sees a blockbuster commercial opportunity for del-brax in FSHD and a positive view of the clinical outlook and registrational path, with a 60% probability of success.

该分析师认为del brax在FSHD中有一个巨大的商业机会，对临床前景和注册路径持积极态度，成功概率为60%。

Del-brax is well positioned to be first-to-market, and the analyst highlights significant revenue opportunity, with an estimated peak sales of $2.7 billion.

Del brax处于领先市场的良好位置，该分析师强调了巨大的收入机会，预计销售额峰值为27亿美元。

Goldman Sachs analyst also says Del-desiran’s Phase 1/2 data positions the company to potentially be a first-to-market drug in myotonic dystrophy. The analyst projects unadjusted peak sales of $4 billion and is optimistic about the trial’s outcome, with a probability of success of 75%.

高盛（Goldman Sachs）分析师还表示，Del desiran的1/2期数据表明，该公司有可能成为首个上市治疗强直性肌营养不良的药物。分析师预测未经调整的峰值销售额为40亿美元，对试验结果持乐观态度，成功概率为75%。

In August, Avidity Biosciences released Del-zota (AOC 1044) 5 mg/kg data that demonstrated a statistically significant increase of 25% of normal dystrophin production and 37% in exon 44 skipping in people living with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the Phase 1/2 EXPLORE44 trial..

8月，Avidity Biosciences发布了Del zota（AOC 1044）5 mg/kg的数据，该数据显示，在1/2期EXPLORE44试验中，患有Duchenne肌营养不良突变的人中，正常肌营养不良蛋白产生的统计学显着增加了25%，外显子44跳跃的统计学显着增加了37%。。

In addition, AOC 1044 5 mg/kg reduced creatine kinase levels to near normal with greater than 80% reduction compared to baseline.

此外，AOC 1044 5 mg/kg将肌酸激酶水平降低至接近正常水平，与基线相比降低了80%以上。

Price Action: RNA stock is down 0.07% at $43.47 at last check Tuesday.

价格走势：周二最后一次检查时，RNA股价下跌0.07%，至43.47美元。

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