Form Bio和PacBio宣布合作推出新资源集，以转变AAV设计和分析最佳实践-动脉网

Form Bio and PacBio Announce Collaboration to Launch New Resources Set to Transform AAV Design and Analysis Best Practices

BioSpace 等信源发布 2024-06-27 22:08

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Companies Form Expert Working Group to Introduce Essential Tools for AAV Development and Research Professionals

公司成立专家工作组，为AAV开发和研究专业人员介绍必要的工具

SAN FRANCISCO / ACCESSWIRE / June 27, 2024 / Form Bio, the provider of advanced computational life sciences technology and PacBio (NASDAQ:PACB), a leading developer of high-quality, highly accurate sequencing solutions, today announced a range of new initiatives aimed at advancing and unifying the AAV industry.

旧金山/ACCESSWIRE/2024年6月27日/先进计算生命科学技术提供商Form Bio和高质量、高精度测序解决方案的领先开发商PacBio（纳斯达克：PACB）今天宣布了一系列旨在推进和统一AAV行业的新举措。

The initiatives, including an AAV data dictionary, open-source tools and more, are being driven by a new AAV Working Group, jointly assembled by Form Bio and PacBio and composed of leading AAV experts across industry and academia. The resulting materials are being made available on a new AAV Resource Hub at aav.formbio.com..

这些举措，包括AAV数据字典、开源工具等，都是由一个新的AAV工作组推动的，该工作组由Form Bio和PacBio联合组建，由业界和学术界的领先AAV专家组成。由此产生的材料正在AAV.formbio.com的新AAV资源中心提供。。

AAV is widely recognized as one of the most promising technologies for gene therapy due to its non-pathogenic nature. However, collaboration on AAV-based programs is often hindered by industry silos that lead to divergent terminology and methods, which can differ widely between various companies and institutions operating at the forefront of AAV innovation.

AAV由于其非致病性而被广泛认为是最有前途的基因治疗技术之一。然而，基于AAV的项目的合作往往受到行业孤岛的阻碍，这些孤岛导致不同的术语和方法，这些术语和方法在处于AAV创新前沿的各种公司和机构之间可能存在很大差异。

In addition, the lack of standard definitions and practices works against uniformity of regulatory submissions, making it harder and more time consuming for regulatory agencies to evaluate potential new therapies..

此外，缺乏标准定义和实践不利于监管提交的统一性，使得监管机构评估潜在的新疗法变得更加困难和耗时。。

In response, Form Bio and PacBio have convened leading experts to form an AAV Working Group dedicated to developing and promoting standardized nomenclature and reporting practices to foster consistency and reliability in AAV research and development. The charter of the newly formed AAV Working Group is to develop standardized nomenclature, establish standardized reporting, and promote widespread adoption of these initiatives..

作为回应，Form Bio和PacBio召集了顶尖专家组成了AAV工作组，致力于开发和推广标准化命名和报告实践，以促进AAV研究和开发的一致性和可靠性。新成立的AAV工作组的章程是制定标准化命名法，建立标准化报告，并促进这些举措的广泛采用。。

Members of the AAV Working Group include:

AAV工作组成员包括：

Nam Tonthat, Ph.D - Asklepios BioPharmaceutical

Nam Tonthat，Asklepios BioPharmaceutical博士

Pranam Chatterjee, Ph.D - Department of Biomedical Engineering, Duke University

杜克大学生物医学工程系博士Pranam Chatterjee

Douglas McCarty, Ph.D - Nationwide Children's Hospital

道格拉斯·麦卡蒂博士-全国儿童医院

Phillip W.L. Tai, Ph.D - Department of Microbiology and Physiological Systems, UMass Chan Medical School; Horae Gene Therapy Center, UMass Chan Medical School

Phillip W.L.Tai博士，麻省大学陈医学院微生物学和生理系统系；麻省大学陈医学院Horae基因治疗中心

Adam Cockrell, Ph.D - Solid Biosciences

Adam Cockrell博士-固体生物科学

Elizabeth Tseng, Ph.D - PacBio

Elizabeth Tseng，PacBio博士

Nadia Sellami, Ph.D - PacBio

Nadia Sellami，PacBio博士

Eric Talevich, Ph.D - Form Bio

Eric Talevich，博士-Form Bio

Alpha Diallo, Ph.D - Form Bio

Alpha Diallo博士-生物型

Amicia Elliott, Ph.D - Form Bio

Amicia Elliott博士-Form Bio

Brandi Cantarel, Ph.D - Form Bio

Brandi Cantarel，Form Bio博士

Claire Aldridge, Ph.D - Form Bio

Claire Aldridge，博士-Form Bio

'Getting AAV-based gene therapies to the clinic requires speed and collaboration among many different parties. But it's become clear that speed and collaboration are being held back by the lack of a common language and practices in dealing with AAV vectors,' said Claire Aldridge, Ph.D, chief strategy officer for Form Bio.

“将基于AAV的基因疗法引入临床需要许多不同方面的速度和合作。Form Bio首席战略官克莱尔·奥尔德里奇博士说，但很明显，由于在处理AAV媒介方面缺乏共同的语言和做法，速度和合作受到了阻碍。

'We see working with our partners at PacBio and bringing together leaders across academia and industry as a critical need in helping AAV-based gene therapies reach their fullest potential faster,' she added..

她补充说：“我们认为，与PacBio的合作伙伴合作，将学术界和业界的领导者聚集在一起，是帮助基于AAV的基因疗法更快地发挥最大潜力的关键。”。。

'PacBio is thrilled to be part of this effort and partnering with Form Bio and leading experts in the field. The value of highly accurate long-read sequencing for gene therapy is clear, and we are excited to see how these resources help researchers develop the next generation of vectorsl,' said Nadia Sellami, Ph.D, segment lead, cell and gene therapy for PacBio..

“PacBio很高兴能够参与这项工作，并与Form Bio和该领域的领先专家合作。PacBio的segment lead、cell and gene therapy博士纳迪亚·塞拉米（NadiaSellami）说，高度准确的长读测序对基因治疗的价值是显而易见的，我们很高兴看到这些资源如何帮助研究人员开发下一代vectorsl。。

The AAV Working Group has generated several key, publicly available resources:

AAV工作组已经产生了几个关键的公开可用资源：

Preprint Publication: Standardized Nomenclature and Reporting for PacBio HiFi Sequencing and Analysis of rAAV Gene Therapy Vectors

预印本出版物：PacBio HiFi测序和rAAV基因治疗载体分析的标准化命名和报告

Open Source Workflow: LAAVA (Long-read AAV Analysis)

开源工作流程：LAAVA（Long read AAV Analysis）

Glossary of Terms: AAV Data Dictionary

术语表：AAV数据字典

Regulatory Case Study: Characterizing Drug Products with NGS

监管案例研究：用NGS表征药品

All of the above resources and more can be explored on the new AAV Resource Hub at aav.formbio.com.

以上所有资源以及更多内容都可以在AAV.formbio.com的新AAV资源中心上进行探索。

These resources aim to streamline development processes and collaboration across the AAV industry. By fostering consistency and clarity in AAV research and development, Form Bio and PacBio are paving the way for more efficient and effective gene therapy solutions, ultimately benefiting patients worldwide..

这些资源旨在简化AAV行业的开发流程和协作。Form Bio和PacBio通过促进AAV研究和开发的一致性和清晰性，为更高效和有效的基因治疗解决方案铺平了道路，最终使全世界的患者受益。。

To access these and other resources or to learn more about getting involved with the AAV Working Group, please visit aav.formbio.com.

要访问这些和其他资源或了解更多有关参与AAV工作组的信息，请访问AAV.formbio.com。

About Form Bio

关于Form Bio

Form Bio provides award-winning software and AI solutions for cell and gene therapy leaders. By combining data, technology and expertise, Form's solutions accelerate timelines from discovery to clinic by providing drug developers with rapid in silico characterization, prediction, simulation and optimization of their therapeutics - enabling higher yields, enhanced safety and shorter, less-expensive development cycles.

Form Bio为细胞和基因治疗领导者提供屡获殊荣的软件和AI解决方案。通过结合数据、技术和专业知识，Form的解决方案为药物开发人员提供了快速的计算机表征、预测、模拟和优化治疗方法，从而加快了从发现到临床的时间表，从而实现了更高的产量、更高的安全性和更短、更便宜的开发周期。

With cross-disciplinary expertise spanning software engineering, biology, bioinformatics and data science, the Form Bio team collaborates closely with customers on their most pressing and strategic challenges and opportunities. For more information, visit www.formbio.com or follow the company at https://www.linkedin.com/company/formbio..

凭借跨越软件工程、生物学、生物信息学和数据科学的跨学科专业知识，Form Bio团队在客户最紧迫和战略性的挑战和机遇上与客户密切合作。欲了解更多信息，请访问www.formbio.com或关注公司https://www.linkedin.com/company/formbio..

About PacBio

关于PacBio

PacBio (NASDAQ: PACB) is a premier life science technology company that designs, develops, and manufactures advanced sequencing solutions to help scientists and clinical researchers resolve genetically complex problems. Our products and technologies stem from two highly differentiated core technologies focused on accuracy, quality and completeness which include our HiFi long-read sequencing and our SBB® short-read sequencing technologies.

PacBio（纳斯达克股票代码：PACB）是一家领先的生命科学技术公司，设计、开发和制造先进的测序解决方案，以帮助科学家和临床研究人员解决遗传复杂问题。我们的产品和技术源自两项高度分化的核心技术，专注于准确性、质量和完整性，其中包括我们的HiFi长读测序和SBB®短读测序技术。

Our products address solutions across a broad set of research applications including human germline sequencing, plant and animal sciences, infectious disease and microbiology, oncology, and other emerging applications. For more information, please visit www.pacb.com and follow @PacBio..

我们的产品解决了广泛的研究应用问题，包括人类种系测序、植物和动物科学、传染病和微生物学、肿瘤学以及其他新兴应用。有关更多信息，请访问www.pacb.com并关注@PacBio。。

PacBio products are provided for Research Use Only. Not for use in diagnostic procedures.

PacBio产品仅供研究使用。不用于诊断程序。

Forward-Looking Statements

前瞻性声明

This press release may contain 'forward-looking statements' within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the U.S. Private Securities Litigation Reform Act of 1995. All statements other than statements of historical fact are forward-looking statements, including statements relating to PacBio's collaboration with Form Bio and the AAV Working Group; the potential benefits and impacts of the AAV Working Group; the benefits or potential benefits of AAV-based programs; and other future events.

本新闻稿可能包含经修订的《1934年证券交易法》第21E节和《1995年美国私人证券诉讼改革法》所指的“前瞻性声明”。除历史事实陈述外，所有陈述均为前瞻性陈述，包括与PacBio与Form Bio和AAV工作组合作有关的陈述；AAV工作组的潜在利益和影响；基于AAV的计划的好处或潜在好处；以及其他未来的活动。

You should not place undue reliance on forward-looking statements because they are subject to assumptions, risks, and uncertainties and could cause actual outcomes and results to differ materially from currently anticipated results, including the risk that PacBio's collaboration with Form Bio and the AAV Working Group may not be successful or deliver the benefits that PacBio currently anticipates.

您不应过度依赖前瞻性陈述，因为它们受到假设、风险和不确定性的影响，并可能导致实际结果和结果与当前预期结果产生重大差异，包括PacBio与Form Bio和AAV工作组的合作可能不成功或无法实现PacBio目前预期的利益。

Additional factors that could materially affect actual results can be found in PacBio's most recent filings with the Securities and Exchange Commission, including PacBio's most recent reports on Forms 8-K, 10-K, and 10-Q, and include those listed under the caption 'Risk Factors.' These forward-looking statements are based on current expectations and speak only as of the date hereof; except as required by law, PacBio disclaims any obligation to revise or update these forward-looking statements to reflect events or circumstances in the future, even if new information becomes available..

可能对实际结果产生重大影响的其他因素可以在PacBio向美国证券交易委员会提交的最新文件中找到，包括PacBio在表格8-K、10-K和10-Q中的最新报告，以及在标题“风险因素”下列出的报告这些前瞻性声明基于当前的预期，仅在本协议日期发表；除法律要求外，PacBio不承担修改或更新这些前瞻性声明以反映未来事件或情况的任何义务，即使有新信息可用。。

Contact Information

联系方式

Angela Anderson

安吉拉·安德森

Form Bio

angela@formbio.com

Jake Robison

杰克·罗宾逊

Inizio Evoke Comms

启动Evoke通信

jake.robison@inizioevoke.com

Todd Friedman