Next-generation therapies – including antibodies, targeted protein degraders, and cell and gene therapies – hold tremendous potential for treating diseases. However, their development and manufacture are complex and often fraught with challenges. From high costs and long development timelines to regulatory hurdles, the journey from discovery to market is far from easy. 

One of the biggest obstacles faced in developing next-generation therapies is their high biological complexity. A deep understanding of disease biology is required to target specific cells or molecular components and generate a desirable therapeutic outcome. Consequently, an incomplete understanding of the underlying molecular mechanisms has led to being unable to identify a proper target.

Therefore, to ensure successful drug development and manufacture, biological complexity must be accounted for. This starts from as early as drug discovery, with the need for model systems and screening assays that better reflect human biology, capturing subtle molecular changes that can influence safety and efficacy. 

During drug discovery, physiologically relevant disease models, such as patient-derived induced pluripotent stem cells (iPSCs) and organoids, should be used to help capture human diversity. Recognizing this, industrialized iPSC-based platforms are central to drug discovery at Evotec, being used from early lead optimization through to regulatory safety studies.

This includes the use of emerging “clinical-trial-in-a-dish” models that aim to replicate diverse patient responses in vitro. These models can help identify candidates with strong efficacy and flag those with potential toxicity. iPSCs are also paving the way for scalable off-the-shelf therapies, enabling the scalable, cost-efficient, and high-throughput production of cell therapies. However, given their biological complexity, the true power of iPSCs can only be unlocked when next-generation technologies, including omics, are used to inform the development process.   

Over the last few decades, molecular biology techniques have evolved drastically. From studying a handful of proteins requiring large quantity of input biological material  (e.g. using Western Blots), scientists can now simultaneously analyze thousands of genes, transcripts, proteins, and metabolites, thanks to the rise of low input and high-throughput omics technologies. Multiomics techniques have become an indispensable part of drug discovery, with the ability to quickly generate extraordinary amounts of relevant biological data.

More recently, the following omics approaches have helped to drive further advances in drug discovery: 

1. Single-cell and spatial resolution – Transitioning from traditional bulk omics to single-cell and spatial approaches (e.g. single cell transcriptomics, spatial transcriptomics,  spatial proteomics, high-sensitivity proteomics) is revolutionizing drug discovery, providing insights into the molecular states and spatial organization of individual cells or cell types.
2. Time-resolved multiomics – Merging omics data, including genomics, transcriptomics, proteomics, and metabolomics over a time course  (e.g. from longitudinal patient data), is helping to provide a deeper understanding of disease progression, and can support development of drugs which act even before the on-set of symptoms. On the other hand, in cell culture experiments a link between the omics read-outs at different time points can support understanding of causal biological relationships, e.g. between transcript and protein level.
3. AI/ML driven data analysis – Machine learning and artificial intelligence approaches are becoming increasingly used in multi-omics analysis, helping to integrate complex datasets and identify patterns. These advanced approaches substantially improve speed and accuracy, while enabling a deeper, more holistic understanding of disease mechanisms. Download our free whitepaper for further insights into how AI/ML can be leveraged in drug discovery. 

At Evotec, we’re embracing cutting-edge advances in omics with our PanOmics platform . Our proprietary E.MPD (Evotec Molecular Patient Database) is central to this platform, generating data from thousands of patient samples, helping to unravel complex disease biology and drive advances in drug discovery. Adopted omics techniques include mass spectrometry-based proteomics, which is a particularly useful approach to discover hits for previously “undruggable” proteins or identify novel protein biomarkers which are not accessible by antibody-based technologies. You can learn more about the power of MS-based proteomics in this whitepaper. 

In addition to hit identification, our PanOmics platform is innovating other aspects of drug discovery, including disease modeling (both in vitro and in vivo), target identification, biomarker discovery, and patient stratification. The seamless integration of multi-omics throughout the drug discovery pipeline ultimately increases the probabilities of success, helping to ensure more effective candidate selection, reduced attrition rates, and a faster path to the clinic.

To accelerate the discovery of next-generation therapies, agnostic technologies, including omics, are becoming increasingly important. These technologies aren’t limited to specific diseases or drug modalities, and instead focus on uncovering the underlying molecular mechanisms. Many omics technologies are classified as agnostic, as they’re unbiased and do not require any domain-related knowledge about the drug modality or therapeutic area. 

Experiments can be designed without forming an a priori hypothesis on the target proteins, generating flexibility in drug discovery and development. An example of a flexible agnostic technology that can be used in drug discovery is the use of mass spectrometry-based proteomics, replacing traditional targeted immuno-based assays to help capture the full complexity of the biological system. Drug repurposing is another emerging trend that is increasing the flexibility of drug discovery. This approach uses advanced computational methods, including transcriptomics and artificial intelligence, to explore potential new uses for drugs that are already on the market. 

For those looking to develop innovative modalities, partnering with a CRO/CDMO like Evotec is key for success. At Evotec, we provide standardized quality-assured platforms, with the seamless integration of cutting-edge technologies to remain at the forefront of drug discovery and development. We also believe integration is a crucial element for success, with our fully integrated drug discovery and development program, INDiGO® , helping to de-risk candidates and ensure a smooth transition from initial concept to final product.  
As therapies become more complex and individualized, there is a greater need for cost-efficient manufacturing with higher success rates. Lower costs and risks can be achieved by having cell line development, process development, and clinical and commercial manufacturing under one roof, as in the case with our Just – Evotec Biologics’ J.POD sites . Located in the US and Europe, these sites provide the infrastructure for continuous manufacturing, paving the way for scalable and affordable biotherapeutics. 
 

In addition to our technical capabilities, the people behind the partnerships play an equally important role. Our team of dedicated scientists works closely with our partners to drive each project forward, leveraging broad cross-disciplinary technological and therapeutic areas expertise, and valuing the importance of communication, flexibility, and adaptability. 
 

You can learn more about our partnerships here , and for further insights on the development of next-generation therapies, visit our research hub.