What we asked: “Looking ahead to the next 5–10 years, what will be the key disruptors and/or what can be improved upon in the pharma industry?”
Response from: Josh Mandel-Brehm, President & CEO, CAMP4 Therapeutics
“The Human Genome Project has had an incalculable impact on our understanding of disease drivers and potential therapeutic strategies. Our first read of the genome focused on characterizing the sequence of genes encoding proteins, which provided a roadmap to drug the genome. Armed with this information, our industry has developed an arsenal of therapeutics using multiple modalities that are primarily designed to either inhibit protein function or downregulate the expression of specific genes, including silencing target genes. Despite this progress, we lack deeper insights into the regulation of gene expression to develop a broadly applicable approach to upregulate gene expression and address the more than 1,200 diseases where even a modest increase in gene expression could potentially be transformative for patients.
“In the last decade, technological advancements in next-generation sequencing techniques have provided us with a growing understanding of the mechanisms underlying gene regulation, shining light on a completely new target class for drug development. Through the development of novel approaches and creative applications of proven therapeutic strategies, we have an incredibly vast opportunity to not only to directly address diseases caused by an under expressed gene, but also leverage this newfound capability to advance regenerative medicine as well as disease prevention and health optimization.”
Response from: Carleen Kluger, Group Leader Clinical Proteomics, Evotec
“Multi-omics analysis of large patient cohorts, such as the UK Biobank, German NAKO, deCODE from Iceland or the biobanks managed by Fingenious, could revolutionize precision medicine and drug discovery. Currently, characterization of healthy individuals and patients prior to any medical intervention, diagnosis or recruitment to a clinical trial is based on a small panel of selected biomarkers. In the future, I believe that a full multi-omics screen (comprising of genomics, transcriptomics, proteomics, and metabolomics) performed on minimally invasive samples, such as biofluids, would provide a far more comprehensive and beneficial picture. To unlock the full potential of these molecular fingerprints, population sized cohorts, with multi-omics data from hundreds of thousands of individuals, would need to be generated to provide the background on which samples can be matched. Although each omic technology alone provides significant insights, only together can they create a full picture. While a patient’s current health status (which is influenced by nutrition and lifestyle) can be assessed using proteomics and metabolomics measurements, genomics will remain indispensable for analysis of genetic risk factors.
“Moving from separate databases from individual studies and trials to a continuous and extendible database would be a big step for the pharma industry. Generating a reference framework with a standardized multi-omics format to which individual patient samples can be mapped could be a huge step forward, enabling not only precise diagnosis and treatment, but also the design and development of new drugs. First steps are already being made to generate such data sets; however, a standardized multi-omics format, as well as easy integration of additional data or cohorts into already existing databases, is still missing.
“This is a joint effort which the pharma industry needs to work on together to continue our journey for medicines that matter.”
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