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.
在週四，Truist開始覆蓋Solid Biosciences Inc.（納斯達克：SLDB），這是一家開發針對骨骼和心臟肌肉疾病基因療法的臨牀階段生物技術公司。
Truist initiated with a Buy rating and a price target of $16.
Truist給予買入評級，目標價爲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.
新的統一平台結合了Solid Biosciences在設計微型肌營養不良蛋白（uDys）轉基因方面的專業知識與AavantiBio先進的AAV製造過程。分析師Joon Lee建議，這可能創造出頂級的杜氏肌營養不良基因療法SGt-003。
Also Read: Solid Biosciences Positioned For Next-Gen Duchenne Muscular Dystrophy Treatment Success: Analyst
另請閱讀：分析師指出Solid Biosciences有望在下一代杜氏肌營養不良治療成功中佔據重要位置。
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.
分析師強調，預計在2025年第一季度進行的SGt-003人類試驗數據的中期評審，可能會降低Solid Biosciences在杜氏肌營養不良項目及其更廣泛的肌肉骨骼基因療法平台中的風險。
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，公司計劃於2025年上半年提交第二個針對兒茶酚胺多態性心室心動過速的研究新藥申請。
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的分析師補充說，Solid Biosciences在開發DMD治療方面領先於Kate Therapeutics和諾華製藥（紐交所：NVS），因爲他們的項目仍處於臨牀前階段。Solid Biosciences的SGt-003有潛力脫穎而出，因其獨特的uDys設計，包括一個可提供額外益處的一氧化氮合酶結構域。
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).
分析師Lee強調，DMD基因療法領域競爭激烈，已有批准的治療方案以及多個處於臨牀試驗中的其他選項。然而，Solid Biosciences憑藉其獨特的方法脫穎而出，結合了先進的負載（uDys+nNOS）和專門的遞送方法（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主辦的會議上，DMD患者的父母表達了對現有治療方法的擔憂，如Sarepta Therapeutics Inc的（納斯達克: SRPT）Elevidys——DMD唯一批准的基因療法——並不足夠。
"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.
分析師表示："基於最近的FDA主辦會議，在那裏DMD患者的父母表達了對更好治療選擇的需求，我們認爲不僅有改進的空間，而且有對更好基因療法的需求。"
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.
週三，FDA批准了該公司針對弗里德里希共濟失調的SGt-212的新藥研究申請，這是一種由於弗拉塔克欣蛋白水平不足而導致的退行性疾病。
Phase 1b trial initiation is expected in the second half 2025.
預計10億階段試驗將在2025年下半年啓動。
Price Action: SLDB stock is up 4.7% at $4.10 at last check Wednesday.
價格動態：SLDb股票在週三最後檢查時上漲了4.7%，達到了4.10美元。
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Photo: Shutterstock
Photo: Shutterstock
譯文內容由第三人軟體翻譯。
