Solid Biosciences (SLDB) 2025 Conference June 04, 2025 02:00 PM ET Speaker0 Everyone. My name is Maury Raycroft and I'm one of the biotech analysts at Jefferies. It's with great pleasure that I'd like to welcome the SolidBio team. We've got Bo Kumbow and Gabe Brooks joining us today. And we're going to do fireside chat format. So, for those who are new to the story, maybe give a brief intro to Solid, your key programs and platform. Speaker1 Yeah, thank you, Mari. I appreciate the invitation. Solid Bioscienc ...

Solid Biosciences Investor Contact: CHARLESTOWN, Mass., May 28, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company" or "Solid"), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, announced that Bo Cumbo, President and CEO, and Gabriel Brooks, M.D., Chief Medical Officer, will participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 4, at 2:00 pm ET. A live webcast of the fireside chat will b ...

- Duchenne (SGT-003): Participant dosing ongoing in the Phase 1/2 INSPIRE DUCHENNE trial; Solid on track to discuss accelerated pathways with U.S. FDA later in 2025 - - FA (SGT-212): Dosing of first participant anticipated in the second half of 2025 - - CPVT (SGT-501): FDA IND on track for submission first half of 2025 - - Cash: Company ended first quarter 2025 with $306.9 million in cash, cash equivalents, investments and available-for-sale securities; Solid has anticipated cash runway into the first half ...

FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 Commission File Number: 001-38360 Solid Biosciences Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 90-0943402 (Stat ...

纪要涉及的公司和行业 - **公司**：Solid Biosciences（SLDB）、Avanti Bio、Elevitus、Regenexx、Sarepta、Lexio - **行业**：基因治疗行业，专注于杜氏肌营养不良症（Duchenne）、弗里德赖希共济失调（Friedrichs Ataxia）、儿茶酚胺敏感性多形性室性心动过速（CPVT）等疾病的治疗 核心观点和论据 公司转型与技术优势 - **核心观点**：SolidBio在与Avanti Bio合并后发生重大转变，专注于下一代基因治疗技术，有望为基因治疗带来变革 [4][5] - **论据**：公司致力于改进基因治疗的递送方式，包括衣壳、启动子和制造工艺等方面，拥有针对杜氏肌营养不良症的最佳基因疗法，以及19个使用其递送工具的合作伙伴关系 [5][7] SGT003的差异化优势 - **核心观点**：SGT003在设计上与其他项目有显著差异，具有更好的治疗效果 [8] - **论据**：拥有新颖的转基因，去除中间铰链，使蛋白质更具灵活性，增加长期耐久性；是首个在人类受试者中显示酶NNOS活性的项目，可增加血管舒张、血流，减少炎症和纤维化 [8][9][10] INSPIRE试验结果 - **核心观点**：INSPIRE试验的初步数据显示SGT003具有良好的治疗潜力，有望达到临床终点 [11][12] - **论据**：SLB101衣壳具有高结合能力和转导速度，使载体基因组拷贝数达到最高水平；蛋白质表达水平高，80%的患者肌肉纤维在中期活检时呈阳性；70%的肌营养不良糖蛋白复合物聚集在一起；NOS活性较基线增加62倍，42%的肌肉纤维具有NOS活性；对肌肉损伤的急性、慢性和成熟阶段的生物标志物有显著影响，可减少胚胎型肌球蛋白重链的表达 [13][14][15][17] 药物对心脏的影响 - **核心观点**：药物对杜氏肌营养不良症患者的心脏问题有积极影响 [21] - **论据**：25%左右6岁的儿童开始出现心脏疾病的早期表现，10岁时约50%的儿童有心脏问题；两名基线时肌钙蛋白升高的男孩在用药后肌钙蛋白恢复正常并低于基线；三名男孩中有两名的射血分数有改善 [23][24][25] 试验进展与前景 - **核心观点**：试验进展顺利，有望获得加速批准，市场前景广阔 [27][32] - **论据**：将在秋季与FDA会面；Sarepta患者死亡事件使更多患者和医生希望参与试验；公司在美国继续给药以寻求加速批准，秋季将在国外开展双盲安慰剂对照试验；公司认为加速批准是满足罕见病患者需求的必要机制，其下一代药物具有优势 [27][28][30][32] 弗里德赖希共济失调项目 - **核心观点**：SGT - 212有望成为治疗弗里德赖希共济失调的唯一有效药物 [38] - **论据**：预计第四季度开始给药；采用双途径给药方式，可到达心脏、DRG、脊髓和小脑等部位，能治疗不同年龄段和不同病情的患者；该疾病在美国有5000 - 7000名患者，欧洲和国外有15000 - 20000名患者，未满足的医疗需求高 [38][40][41] 心血管疾病项目 - **核心观点**：CPVT项目是一种独特的药物，有望治疗该致命疾病 [44] - **论据**：预计在今年上半年提交IND申请，下半年开始给药；该疾病在7 - 12岁时易被诊断，20年内死亡率达40%；在小鼠实验中可恢复正常心律，有望让患者停用部分药物并减轻恐惧 [46] 其他重要但可能被忽略的内容 - SGT003的载体拷贝数和肌营养不良蛋白表达水平相对稳定，这可能与衣壳上的整合素受体有关 [18] - 公司认为即使市场上已有针对杜氏肌营养不良症的药物，仍需开发下一代疗法以满足未满足的医疗需求 [33] - 公司规模为2亿美元左右，目标是在美国发病率人群中实现高盈利，而全球大量患者群体是额外的市场机会 [36]

CHARLESTOWN, Mass., April 30, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, announced that Bo Cumbo, President and CEO, will participate in a fireside chat at The Citizens Life Sciences Conference on Wednesday, May 7, at 12:30 pm ET. A live webcast of the fireside chat will be available on the Events page of the Investors section of the Company website or by cli ...

1. Title: Initial Experience from the INSPIRE DUCHENNE Phase I/II Study of SGT-003 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy Session Title: Late-Breaking Abstracts II Date/Time: May 17, 9:15 – 9:30am CT Location/Room: Room 391-392 Presenter: Kevin Flanigan, MD, Advisor Consultant & Researcher, Nationwide Children's Hospital and investigator in the INSPIRE DUCHENNE clinical trial 2. Title: Insight into the Mechanism of Action of AAV-SLB101, a Novel Muscle-Tropic Capsid for Neuromuscular an ...

In our opinion, Solid Biosciences Inc. (NASDAQ: SLDB ) may be repositioning itself in a space that has very recently come under considerable scrutiny. Though still in early-stage trials, its lead candidate has shown encouraging results.IB's Thoughts is owned and operated by a stock market enthusiast, who has written for Seeking Alpha since 2014. IB's Thoughts is focused on identifying exciting investment opportunities, with a particular emphasis on healthcare.Analyst’s Disclosure: I/we have no stock, option ...

文章核心观点 - 治疗杜氏肌营养不良症（DMD）的唯一FDA批准基因疗法ELEVIDYS出现患者死亡事件，影响Sarepta公司销售和股价，Solid Biosciences的SGT - 003安全性表现出色，有机会进入DMD治疗市场并带来积极影响，但仍面临挑战 [2][4][8] 行业背景 - DMD是一种通常在2 - 5岁男孩中首次出现的肌肉疾病，由基因改变引起且病情会逐渐恶化 [1] ELEVIDYS情况 - Sarepta Therapeutics公司生产的ELEVIDYS是唯一FDA批准治疗DMD的基因疗法，自销售以来增长迅速，2024年Q4收入达3.84亿美元，总营收超10亿美元 [2][3] - 3月18日Sarepta宣布一名使用ELEVIDYS的年轻人因急性肝衰竭死亡，此前测试未显示该药物会导致死亡并发症，消息传出后Sarepta股价暴跌超27%，引发对药物安全性的质疑，可能影响销售和股价 [4][5] - 欧盟监管机构暂时停止ELEVIDYS三项临床试验，Sarepta在欧盟对三个不同年龄组进行测试以获批准，不过有人认为调查结束后试验不会延迟太久，独立委员会认为药物风险收益仍有利 [6] Solid Biosciences的SGT - 003情况 - 2月中旬Solid公布DMD治疗药物SGT - 003临床数据，6名患者治疗中无严重副作用，未出现急性肝损伤 [8] - 用于输送SGT - 003的SLB101衣壳对肌肉细胞的递送和结合效果比ELEVIDYS所用衣壳高5倍，且剂量低25%，低剂量可降低肝损伤风险，Solid认为拥有一流DMD药物 [9][10] Solid Biosciences发展计划与潜力 - 公司正在加大SGT - 003测试力度，预计Q2初治疗10名患者，年底治疗20名患者，还希望获得加速监管批准，因ELEVIDYS仅用6名患者数据就获加速批准，公司计划今年晚些时候与FDA讨论 [11][12] - 2025年ELEVIDYS预计销售额约20亿美元，而4月4日午盘Solid市值仅2.4亿美元，若公司能抓住机会，有巨大上涨潜力，但FDA批准难以预测，需持续提供有力数据 [13]

CHARLESTOWN, Mass., March 12, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (NASDAQ:SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne), at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Dallas, TX, March ...