IRBM扩展功能基因组学平台以加强早期药物发现-动脉网

IRBM Expands Functional Genomics Platform to Strengthen Early Drug Discovery

CISION 等信源发布 2025-04-03 14:45

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De-Risking Target Validation with Data-Driven Genomic Insights

使用数据驱动的基因组洞察进行去风险化靶点验证

ROME

罗马

，

April 3, 2025

2025年4月3日

/PRNewswire/ -- IRBM, an integrated Contract Research Organization (CRO) specializing in preclinical drug discovery, has established a comprehensive Functional Genomics Platform to support genetic target identification and validation, and generation of disease-relevant cellular models. The platform integrates CRISPR-based methodologies, screening capabilities, cell engineering, and advanced molecular and cellular analysis to drive both target discovery and validation..

/PRNewswire/ -- IRBM是一家专注于临床前药物发现的综合合同研究组织（CRO），已建立了一个全面的功能基因组学平台，以支持遗传靶点的识别与验证，以及生成与疾病相关的细胞模型。该平台整合了基于CRISPR的方法、筛选能力、细胞工程以及先进的分子和细胞分析，以推动靶点的发现与验证。

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Stepwise workflow for CRISPR-based genome editing, including base and prime editing, a key focus of IRBM’s expanded Functional Genomics Platform.

基于CRISPR的基因组编辑（包括碱基编辑和先导编辑）的逐步工作流程，这是IRBM扩展的功能基因组学平台的一个关键重点。

To further enhance target validation, IRBM is expanding its genome editing toolkit with CRISPR tiling to systematically map functional domains within coding and regulatory regions, along with base and prime editing to introduce precise nucleotide modifications. These next-generation approaches—combined with the platform's high-throughput CRISPR screening, including stem cell engineering—are instrumental in building robust disease models and improving the reliability of target validation..

为了进一步加强靶点验证，IRBM正在扩展其基因组编辑工具包，采用CRISPR平铺技术系统地绘制编码区和调控区中的功能域，同时结合碱基编辑和先导编辑引入精确的核苷酸修饰。这些下一代方法——连同平台的高通量CRISPR筛选（包括干细胞工程）——在构建强大的疾病模型和提高靶点验证的可靠性方面发挥了关键作用。

'Understanding which targets are truly viable for drug development is a complex challenge,'

“了解哪些靶点真正适合药物开发是一项复杂的挑战，”

said

说

Sara Tomaselli

萨拉·托马塞利

, PhD, Head of Functional Genomics at IRBM.

，博士，IRBM功能基因组学主管。

'With this in-house platform, we're strengthening our ability to systematically evaluate gene function, de-risk early discovery, and support our partners in making well-informed decisions.'

“通过这个内部平台，我们正在加强系统评估基因功能、降低早期发现风险的能力，并支持我们的合作伙伴做出明智的决策。”

The platform's screening capabilities include genome-wide and targeted CRISPR libraries for knockout (CRISPR KO), inhibition (CRISPRi), and activation (CRISPRa), enabling in-depth exploration of gene function and associated pathways. In parallel, IRBM's cell engineering expertise—spanning patient-relevant models and iPSC-derived systems—supports disease modeling across oncology, neurodegeneration, and rare disorders.

该平台的筛选能力包括全基因组和靶向CRISPR文库，用于敲除（CRISPR KO）、抑制（CRISPRi）和激活（CRISPRa），从而实现对基因功能及其相关通路的深入探索。同时，IRBM的细胞工程专业知识涵盖与患者相关的模型和iPSC衍生系统，支持在肿瘤学、神经退行性疾病和罕见病领域的疾病建模。

Multi-omics integration, including high-content imaging, single-cell transcriptomics, and proteomics, provides deeper functional characterization of drug targets..

多组学整合，包括高内涵成像、单细胞转录组学和蛋白质组学，提供了对药物靶点更深入的功能表征。

IRBM's Functional Genomics Platform is part of a broader, fully integrated drug discovery ecosystem, ensuring that genetic insights translate into meaningful preclinical models and drug development strategies. By combining functional genomics with expertise in medicinal chemistry, biomarker discovery, and preclinical pharmacology, IRBM enables biopharma innovators to approach target validation with greater confidence..

IRBM的功能基因组学平台是一个更广泛、完全集成的药物发现生态系统的一部分，确保遗传学见解转化为有意义的临床前模型和药物开发策略。通过将功能基因组学与药物化学、生物标志物发现和临床前药理学的专业知识相结合，IRBM使生物制药创新者能够更有信心地进行靶点验证。

'We take a careful, methodical approach to functional genomics—one that integrates well with the complexities of drug discovery,'

“我们对功能基因组学采取谨慎、系统的方法——这种方法能够很好地与药物发现的复杂性相结合，”

said

说

Carlo Toniatti

卡洛·托尼亚蒂

, MD, PhD, Chief Scientific Officer at IRBM.

医学博士，哲学博士，IRBM首席科学官。

'Our goal is to help our partners uncover high-quality targets and generate data that can guide and inform decision making throughout the entire drug discovery process.'

“我们的目标是帮助我们的合作伙伴发现高质量的目标，并生成能够指导和告知整个药物发现过程决策的数据。”

IRBM's collaborative approach allows biotech startups, pharmaceutical companies, and venture-backed teams to access functional genomics expertise without the need for in-house infrastructure. With customized solutions spanning genetic screening, engineered cell models, and mechanistic studies, IRBM provides practical, scientifically rigorous support at key points in drug discovery..

IRBM的协作方式使生物技术初创公司、制药公司和风险投资支持的团队能够在无需内部基础设施的情况下获取功能基因组学的专业知识。通过涵盖基因筛选、工程细胞模型和机制研究的定制化解决方案，IRBM在药物发现的关键环节提供实用且科学严谨的支持。

To learn more about IRBM's Functional Genomics Platform and collaboration opportunities, visit

要了解更多关于IRBM的功能基因组学平台及合作机会，请访问

www.irbm.com

。

About IRBM

关于IRBM

IRBM is a contract research organization (CRO) focused on early-stage drug discovery. With a track record of delivering preclinical candidates across oncology, neurodegeneration, and infectious diseases, IRBM partners with biotech and pharmaceutical innovators to advance therapeutic development with scientific precision and transparency..

IRBM是一家专注于早期药物发现的合同研究组织（CRO）。凭借在肿瘤学、神经退行性疾病和传染病领域交付临床前候选药物的良好记录，IRBM与生物技术和制药创新者合作，以科学的精确性和透明性推动治疗开发。

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