Solid Biosciences Outpaces Competitors In Duchenne Gene Therapy Development
Solid Biosciences Outpaces Competitors In Duchenne Gene Therapy Development
On Thursday, Truist initiated coverage on Solid Biosciences Inc. (NASDAQ:SLDB), a clinical-stage biotech developing gene therapies for diseases of the skeletal and cardiac muscles.
在周四，Truist开始覆盖Solid Biosciences Inc.（纳斯达克：SLDB），这是一家开发针对骨骼和心脏肌肉疾病基因疗法的临床阶段生物技术公司。
Truist initiated with a Buy rating and a price target of $16.
Truist给予买入评级，目标价为16美元。
The new unified platform combines Solid Biosciences' expertise in designing microdystrophin (uDys) transgenes with AavantiBio's advanced AAV manufacturing process. Analyst Joon Lee suggests this could create a top-tier gene therapy for Duchenne muscular dystrophy, SGT-003.
新的统一平台结合了Solid Biosciences在设计微型肌营养不良蛋白（uDys）转基因方面的专业知识与AavantiBio先进的AAV制造过程。分析师Joon Lee建议，这可能创造出顶级的杜氏肌营养不良基因疗法SGt-003。
Also Read: Solid Biosciences Positioned For Next-Gen Duchenne Muscular Dystrophy Treatment Success: Analyst
另请阅读：分析师指出Solid Biosciences有望在下一代杜氏肌营养不良治疗成功中占据重要位置。
The analyst highlights that an interim review of SGT-003 human trial data, expected in the first quarter of 2025, could reduce risks for Solid Biosciences' lead Duchenne muscular dystrophy program and its broader musculoskeletal gene therapy platform.
分析师强调，预计在2025年第一季度进行的SGt-003人类试验数据的中期评审，可能会降低Solid Biosciences在杜氏肌营养不良项目及其更广泛的肌肉骨骼基因疗法平台中的风险。
Additionally, the company plans to submit a second investigational new drug application for Catecholaminergic polymorphic ventricular tachycardia in the first half of 2025.
此外，公司计划于2025年上半年提交第二个针对儿茶酚胺多态性心室心动过速的研究新药申请。
The Truist analyst adds that Solid Biosciences is ahead of Kate Therapeutics and Novartis AG (NYSE:NVS) in developing DMD treatments, as their programs are still in preclinical stages. Solid Biosciences' SGT-003 has the potential to stand out thanks to its unique uDys design, which includes a nitric oxide synthase domain that could offer additional benefits.
Truist的分析师补充说，Solid Biosciences在开发DMD治疗方面领先于Kate Therapeutics和诺华制药（纽交所：NVS），因为他们的项目仍处于临床前阶段。Solid Biosciences的SGt-003有潜力脱颖而出，因其独特的uDys设计，包括一个可提供额外益处的一氧化氮合酶结构域。
Analyst Lee highlights that the field of DMD gene therapy is highly competitive, with approved treatments and several others in clinical trials. However, Solid Biosciences stands out with its unique approach, combining an advanced payload (uDys+nNOS) and a specialized delivery method (AAV-SLB101).
分析师Lee强调，DMD基因疗法领域竞争激烈，已有批准的治疗方案以及多个处于临床试验中的其他选项。然而，Solid Biosciences凭借其独特的方法脱颖而出，结合了先进的负载（uDys+nNOS）和专门的递送方法（AAV-SLB101）。
During a recent FDA-hosted meeting, parents of DMD patients voiced concerns that existing treatments, like Sarepta Therapeutics Inc's (NASDAQ:SRPT) Elevidys—the only approved gene therapy for DMD—are not enough.
在最近一次FDA主办的会议上，DMD患者的父母表达了对现有治疗方法的担忧，如Sarepta Therapeutics Inc的（纳斯达克: SRPT）Elevidys——DMD唯一批准的基因疗法——并不足够。
"Based on the recent FDA hosted meeting where parents of DMD patients expressed the need for better options, we think there's not only room but a need for better gene therapies," the analyst said.
分析师表示："基于最近的FDA主办会议，在那里DMD患者的父母表达了对更好治疗选择的需求，我们认为不仅有改进的空间，而且有对更好基因疗法的需求。"
On Wednesday, the FDA cleared the company's Investigational New Drug application for SGT-212 for Friedreich's ataxia, a degenerative disease caused by insufficient frataxin protein levels.
周三，FDA批准了该公司针对弗里德里希共济失调的SGt-212的新药研究申请，这是一种由于弗拉塔克欣蛋白水平不足而导致的退行性疾病。
Phase 1b trial initiation is expected in the second half 2025.
预计10亿阶段试验将在2025年下半年启动。
Price Action: SLDB stock is up 4.7% at $4.10 at last check Wednesday.
价格动态：SLDb股票在周三最后检查时上涨了4.7%，达到了4.10美元。
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Photo: Shutterstock
Photo: shutterstock
