Solid Biosciences to Present at the American Society of Gene and Cell Therapy Annual Meeting
Solid Biosciences to Present at the American Society of Gene and Cell Therapy Annual Meeting
— Presentations to highlight AAV manufacturing and purification improvements, vector biology updates,
and a comprehensive non-clinical data overview of the company's lead SGT-003 program —
— 重点介绍 AAV 制造和纯化的改进、载体生物学更新的演讲，
以及该公司主要 SGT-003 计划的全面非临床数据概述—
CHARLESTOWN, Mass., May 07, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present an oral presentation and six posters and at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting, Baltimore, May 7-11.
马萨诸塞州查尔斯敦，2024年5月7日（GLOBE NEWSWIRE）——开发神经肌肉和心脏疾病精准基因药物的生命科学公司Solid Biosciences Inc.（纳斯达克股票代码：SLDB）将在5月7日至11日在巴尔的摩举行的美国基因与细胞疗法学会（ASGCT）2024年年会上发表口头报告和六张海报。
The data presentations will focus on Solid Biosciences' research and manufacturing capabilities aimed at advancing the field of gene therapy. They will cover various aspects of adeno-associated vector (AAV) manufacturing, novel capsid development, and non-clinical studies with a particular emphasis on the company's lead SGT-003 program.
数据展示将重点介绍Solid Biosciences旨在推进基因疗法领域的研究和制造能力。它们将涵盖腺相关载体 (AAV) 制造、新型衣壳开发和非临床研究的各个方面，特别侧重于该公司领先的 SGT-003 项目。
"We are excited to share our latest scientific findings and advancements at the ASGCT Annual Meeting," said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences. "Our presentations underscore our commitment to developing innovative therapies that address unmet medical needs in neuromuscular and cardiac diseases. We look forward to engaging with the gene therapy community and advancing the field together."
Solid Biosciences总裁兼首席执行官Bo Cumbo表示：“我们很高兴在ASGCT年会上分享我们的最新科学发现和进展。”“我们的演讲突显了我们对开发创新疗法的承诺，以满足神经肌肉和心脏疾病中未得到满足的医疗需求。我们期待与基因疗法界合作，共同推动该领域的发展。”
The presentations will feature advancements in gene therapy manufacturing, featuring novel mechanisms to enhance AAV production and refine purification methods. Additionally, innovative approaches in vector biology will be presented, leveraging in silico modeling for AAV vector design and showcasing capsid modifications for improved cardiac tissue targeting. Highlights from non-clinical studies of SGT-003 also will be shared, including findings on microdystrophin expression and functional efficacy in mouse models.
这些演讲将介绍基因疗法制造的进步，介绍增强AAV生产和完善纯化方法的新机制。此外，还将介绍载体生物学的创新方法，利用计算机模拟建模进行AAV载体设计，并展示为改善心脏组织靶向而进行的衣壳修改。还将分享 SGT-003 非临床研究的要点，包括关于小鼠模型中微肌营养不良蛋白表达和功能功效的发现。
The presentations will cover key areas of Solid Biosciences' research and development initiatives, including:
演讲将涵盖固态生物科学研发计划的关键领域，包括：
Oral Presentation
口头演讲
A6 - AAV Vectors - Product Development Manufacturing and Approval Considerations
A6-AAV Vectors-产品开发、制造和批准注意事项
Novel Mechanism to Increase AAV Yield through Blocking AAV Transduction of Manufacturing HEK293 Cells During AAV Production
通过阻断 AAV 生产过程中制造 HEK293 细胞的 AAV 转导来提高 AAV 产量的新机制
Lead Author: Xiaofei E
主要作者：小飞 E
Abstract #28
摘要 #28
May 7, 2:15-2:30pm ET
美国东部时间5月7日下午 2:15-2:30
Poster Presentations
海报演示
Process Development & Manufacturing
工艺开发与制造
High-Throughput Workflows to Accelerate Development of Chromatographic Purification of rAAV Viral Vectors
高通量工作流程可加速 RaAv 病毒载体的色谱纯化开发
Lead Author: Ryan DeGroot
主要作者：瑞安·德格鲁特
Abstract 1543
摘要 1543
May 10, 12:00-1:30pm ET
美国东部时间 5 月 10 日下午 12:00-1:30
Identification of an AAV Affinity Chromatography Elution Buffer that Maximizes Product Recovery and Minimizes Product Degradation
鉴定可最大限度地提高产品回收率并最大限度地减少产品降解的 AAV 亲和色谱洗脱缓冲液
Lead Author: Sierra VanSuch
主要作者：塞拉·范索奇
Abstract 1517
摘要 1517
May 10, 12:00-1:30pm ET
美国东部时间 5 月 10 日下午 12:00-1:30
Increasing Quality and Productivity with Dual Transfection (DT) for AAV Production
通过 AAV 生产的双转染 (DT) 提高质量和生产率
Lead Author: Sarath Mandava
主要作者：萨拉斯·曼达瓦
Abstract 1541
摘要 1541
May 10, 12:00-1:30pm ET
美国东部时间 5 月 10 日下午 12:00-1:30
Vector Biology
矢量生物学
Engineered Cardioskeletal-Directed AAV Capsids That Detarget the Liver
精心设计的以心骨为导向的 AAV Capsids 可使肝脏脱靶向
Lead Author: Widler Casy
主要作者：威德勒·卡西
Abstract 480
摘要 480
May 8, 12:00-1:30pm ET
美国东部时间 5 月 8 日下午 12:00-1:30
Designing Therapeutic Recombinant AAV Vectors Using In Silico Vector Modeling
使用模拟向量建模设计治疗性重组 AAV 载体
Lead Author: Ethan Waple
主要作者：伊桑·瓦普尔
Abstract 466
摘要 466
May 8, 12:00-1:30pm ET
美国东部时间 5 月 8 日下午 12:00-1:30
Non-Clinical Studies
非临床研究
Systemic Delivery of SGT-003 Microdystrophin Gene Therapy Using the Novel Capsid AAV-SLB101 Ameliorates Muscle Pathology and Rescues Muscle Function in the mdx Mouse Model of Duchenne Muscular Dystrophy
使用新型衣壳 AAV-SLB101 系统性传递 SGT-003 微营养不良蛋白基因疗法可改善杜兴氏肌肉萎缩症 mdx 小鼠模型中的肌肉病理并挽救肌肉功能
Lead Author: Jamie Marshall
主要作者：杰米·马歇尔
Abstract 1391
摘要 1391
May 9, 5:30-7pm ET
美国东部时间 5 月 9 日下午 5:30-7
About Solid Biosciences
Solid Biosciences is a life sciences company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid's mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com.
关于固体生物科学
Solid Biosciences是一家生命科学公司，专注于推进候选基因疗法产品组合，包括用于治疗杜兴氏肌营养不良症（杜兴纳）的 SGT-003、用于治疗儿茶酚胺能多态性心动过速（CPVT）的 SGT-501、用于治疗 BAG3 介导的扩张型心肌病的 AVB-401 以及用于治疗致命心脏病的其他资产。Solid 正在推进其针对罕见神经肌肉和心脏疾病的多元化产品线，汇集了科学、技术、疾病管理和护理领域的专家。Solid 以患者为中心，由直接受影响的人创立，其使命是改善患有这些毁灭性疾病的患者的日常生活。欲了解更多信息，请访问 www.solidbo.com。
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the company; the company's planned oral and poster presentations; and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," "working" and similar expressions. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the ability to recognize the anticipated benefits of Solid's acquisition of AavantiBio; the company's ability to advance SGT-003, SGT-501, AVB-401 and other preclinical programs and capsid libraries on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies of the company's product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, SGT-501, AVB-401 and other candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company's views as of the date hereof and should not be relied upon as representing the company's views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.
前瞻性陈述
本新闻稿包含1995年《私人证券诉讼改革法》所指的 “前瞻性陈述”，包括有关公司未来预期、计划和前景的陈述；公司计划的口头和海报陈述；以及其他包含 “预期”、“相信”、“继续”、“可能”、“估计”、“打算”、“计划”、“潜力”、“预测”、“项目” 等词语的声明、” “应该”、“目标”、“将”、“工作” 和类似的表达。任何前瞻性陈述均基于管理层当前对未来事件的预期，并受到许多风险和不确定性的影响，这些风险和不确定性可能导致实际业绩与此类前瞻性陈述中列出或暗示的业绩存在重大不利差异。这些风险和不确定性包括但不限于与认识 Solid 收购 Aavantibio 的预期收益的能力相关的风险；该公司按照预期的时间表或根本推进 SGT-003、SGT-501、AVB-401 和其他临床前项目和衣壳库的能力；获得并维持美国食品药品管理局和其他监管机构的必要批准；在临床试验中复制公司候选产品的临床前研究中发现的积极结果；获得，维护或保护知识产权与其候选产品相关的权利；与其他寻求开发杜兴和其他神经肌肉和心脏疗法和基因疗法的公司成功竞争；管理开支；并在必要的时间表内筹集继续开发 SGT-003、SGT-501、AVB-401 和其他候选药物、实现其其他业务目标并继续经营所需的大量额外资金。有关其他风险和不确定性以及其他重要因素的讨论，其中任何一个都可能导致公司的实际业绩与前瞻性陈述中包含的有所不同，请参阅公司最近向美国证券交易委员会提交的文件中的 “风险因素” 部分以及对潜在风险、不确定性和其他重要因素的讨论。此外，本新闻稿中包含的前瞻性陈述代表公司截至本新闻稿发布之日的观点，不应以此作为公司自发布之日起任何日期的观点。该公司预计，随后的事件和事态发展将导致公司的观点发生变化。但是，尽管公司可能会选择在未来的某个时候更新这些前瞻性陈述，但该公司明确表示不承担任何更新这些前瞻性陈述的义务。
Media Contact:
Glenn Silver
FINN Partners
glenn.silver@finnpartners.com
媒体联系人：
格伦·西尔弗
FINN 合作伙伴
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Source: Solid Biosciences Inc.
资料来源：固态生物科学公司
译文内容由第三方软件翻译。