#Italy #Drug Discovery #Rome #CRISPR Technology #IRBM Functional Genomics

IRBM Expands Functional Genomics Platform to Enhance Drug Discovery

Introduction

In the ever-evolving landscape of drug discovery, IRBM (Istituto di Ricerca di Biomedicina) is making significant strides with its newly expanded Functional Genomics Platform. This cutting-edge development aims to enhance the validity of target identification and to streamline the creation of cellular models that mirror disease conditions. By integrating a range of advanced techniques and methodologies, IRBM is positioned to lead the charge in early drug discovery efforts.

Comprehensive Approach to Functional Genomics

The newly established platform is comprehensive, integrating CRISPR-based methodologies, advanced screening capabilities, cell engineering, and molecular analysis. These components are essential for both target identification and validation, which can often be hindered by uncertainty in selecting viable drug targets. The platform focuses on de-risking target validation, a critical step for pharmaceutical companies as they assess potential candidates for new therapies.

Enhancements with CRISPR Methodologies

With the expansion of its genome editing toolkit, IRBM is implementing innovative CRISPR tiling techniques. This systematic approach maps functional regions within both coding and regulatory domains. Additionally, the incorporation of base and prime editing facilitates precise genetic modifications—key enhancements that strengthen the platform's capabilities.

Sara Tomaselli, PhD, Head of Functional Genomics at IRBM, emphasizes the complexity of identifying viable drug targets: “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.” This strategic focus ensures that the drug discovery process is grounded in reliable scientific verification.

Advanced Screening for Deeper Insights

The screening functionalities of the platform leverage both genome-wide and targeted CRISPR libraries. These libraries cater to various applications, including knockout (CRISPR KO), inhibition (CRISPRi), and activation (CRISPRa) studies. By enabling detailed exploration of gene functions and their interrelated pathways, IRBM enhances its capacity for high-resolution functional characterization.

Patient-Relevant Models and Multi-Omics Integration

IRBM’s expertise in cell engineering plays a vital role in developing patient-relevant models, particularly through the utilization of induced pluripotent stem cells (iPSCs). This breadth of application supports disease modeling across diverse domains such as oncology, neurodegeneration, and rare genetic disorders. In conjunction with high-content imaging and single-cell transcriptomics, this multi-omics integration provides a robust framework for elucidating drug targets at unprecedented depths.

A Holistic Drug Discovery Ecosystem

The functionalities encompassed within IRBM's Functional Genomics Platform are not standalone; they form part of a broader, fully integrated drug discovery ecosystem. Here, genetic insights seamlessly transition into impactful preclinical models and drug development strategies. The synthesis of functional genomics with medicinal chemistry and biomarker discovery positions IRBM as a pivotal player in the biopharma industry's ongoing evolution.

Supporting Biopharmaceutical Innovation

Carlo Toniatti, MD, PhD, Chief Scientific Officer at IRBM, elaborates on the organization’s methodology: “We take a careful, methodical approach to functional genomics—one that integrates well with the complexities of drug discovery.” By aligning its operations with biopharmaceutical innovators, IRBM enables targeted validation with increased confidence, thus streamlining the drug development process.

Collaborative Opportunities and Conclusion

For biotech startups and established pharmaceutical companies, IRBM offers access to functional genomics expertise without the stringent requirement for in-house infrastructure. Their tailored solutions span genetic screening, engineered cell models, and mechanistic studies, granting clients scientifically robust support to navigate pivotal moments in drug discovery.

To explore how IRBM's expanded Functional Genomics Platform can revolutionize your drug discovery initiatives or to learn about partnership opportunities, visit www.irbm.com. As the drug discovery landscape continues to shift, IRBM remains at the forefront, offering researchers the tools and insights necessary to innovate and succeed.