Role of Biomolecules and Biologics in Precision Medicine, Personalized Medicine, and Emerging Therapies

Abstract

In the 1990s, DNA sequencing technologies could only read bite-sized pieces of DNA. Then came the human genome project (HGP), a thirteen-year international effort, 1990-2003, with the primary goal of discovering the complete set of human genes, sequencing nucleotides, and making the information accessible worldwide for further biological studies. We have come a long way since that time in terms of sequencing the genes of the human genome. Now the researchers can sequence the DNA and analyze gene-expressed proteins in individual cells, allowing them to dissect the complexities of genetic diseases with exceptional details. Currently, technologies are available for single-cell or multi-omics platforms to analyze genotype and phenotype. The completion of this one-of-a-kind project created public expectations for immediate, better health care delivery and possible cures for 'so called' incurable diseases. The HGP was the single most influential investment made in modern basic science research. A monumental breakthrough in medicine has given us the ability to sequence the DNA in cancer cells to identify possible errors in mutations. The impact of the HGP's success was so significant that President Barack Obama initiated a very ambitious new 'precision medicine' research initiative and announced the launch of this project during his State of the Union Address in 2015. The benefits of precision and personalized medicine include predicting susceptibility to diseases, improving disease diagnostics, preempting disease progression, customizing disease prevention strategies, and developing personalized drugs and therapies. As examples of emerging therapies, we have discussed the role of biomolecules and biologics in precision medicine applications like 'The All of Us,' personalized medicine approaches for monogenic diseases like hemophilia, sickle cell disease, and other rare genetic disorders, and CRISPR gene-editing technologies. Biomolecules play an essential role in all life processes, a variety of signaling processes, which are vital for normal functioning of physiological responses, in the early diagnosis of risk factors for various diseases, in the development of diseases and their progress. Furthermore, biomolecules, RNAs, DNAs, molecular and cellular engineering, genetic engineering of biologics, cells, tissues, and organs, play an important role in emerging therapeutic applications. The majority of the therapies discussed in this review are regulated as biologics under the Public Health Services Act of the USA. There is great interest in developing targeted therapy or precision medicine therapy for monogenic diseases, organ transplant applications, and tumor management, designed to interfere with targeted molecules for cancer-causing genes to slow the spread of cancer cells. Because molecular engineering, the development of biologics, gene-editing applications, and biomanufacturing are key components of emerging therapies, a keynote series was organized at INTERPHEX in November of 2021. INTEPHEX is the premier event that offers the latest intelligence, cutting-edge technologies, and state-of-the-art innovation for product development for pharmaceutical and biotechnology platforms. In an earlier article in this journal, we described drug discovery and development in the COVID Age; this overview provides a birds-eye view of the salient findings in each emerging area of medicine—precision medicine, personalized medicine, and emerging therapies.