De-Risking Target Validation with Data-Driven Genomic Insights
ROME, April 3, 2025 -- 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.
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.
"Understanding which targets are truly viable for drug development is a complex challenge," said Sara Tomaselli, PhD, Head of Functional Genomics at IRBM. "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."
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. Multi-omics integration, including high-content imaging, single-cell transcriptomics, and proteomics, provides deeper functional characterization of drug targets.
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.
"We take a careful, methodical approach to functional genomics—one that integrates well with the complexities of drug discovery," said Carlo Toniatti, MD, PhD, Chief Scientific Officer at IRBM. "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."
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.
To learn more about IRBM's Functional Genomics Platform and collaboration opportunities, visit www.irbm.com.
About 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.
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