MAY 2023LIFE SCIENCES REVIEW 19CXO INSIGHTSickle cell disease (SCD) is an inherited disorder of the red blood cells where abnormal sickle-shaped red blood cells clump together to block capillaries and deprive tissues and organs of oxygen, resulting in cellular damage and severe pain. "Pain crises" are the most common cause of hospitalization for people with SCD, with many frequently spending days, weeks, or even months in the hospital [3]. Without treatment, SCD can lead to serious complications such as organ damage, pulmonary hypertension, and heart failure [4]. Currently, the only curative treatment for SCD is hematopoietic stem cell transplantation [5]; however, more than 80 percent of patients do not have a matched donor.Scientists are researching new potential cell and gene therapies based on novel genetic technologies, including CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) gene editing to target disease-related genes identified using next generation sequencing (NGS). For instance, Dr. Matthew Porteus and his research team at Stanford Medicine are pioneering the development of CRISPR-Cas9 based approaches into genomic medicines for SCD and beta-thalassemia, another inherited blood disorder [6,7]. Similarly, the Hendel Lab headed by Dr. Ayal Hendel at Bar Ilan University in Israel is developing CRISPR-Cas9 based genome editing as a curative therapy for genetic diseases, focusing on hematopoietic genetic disorders such as severe combined immunodeficiency (SCID) [8].Alongside the development of new therapies for individuals with rare and orphan diseases, genomic medicines are also being developed for people with more common diseases, such as cancer. Dr. Katy Rezvani and her lab are looking into whether CRISPR-Cas9 gene editing can be used to further modify and improve the effectiveness of natural killer cells already engineered with chimeric antigen receptors (CAR-Ts) [9,10]. At Integrated DNA Technologies (IDT), we have worked with many translational medicine developers, including all three of the researchers just mentioned, and are witnessing first-hand how rapidly genomic medicine is evolving, with new approaches being considered all the time. Challenges still hinder the genomic medicine revolutionAs promising as these genetic-based therapeutic approaches seem, several key hurdles must still be overcome before we can realize the full potential of the genomic medicine revolution. A critical issue is how to address the broad range of scales required for manufacturing clinical grade genomic medicines, from as little as a one-shot treatment personalized for a single patient to billions of vaccine doses for the world's population. The requirement to be able to simultaneously scale out and up is a serious challenge for biopharmaceutical companies.Traditionally, the main business model of pharma and biopharma companies relies on the economies of scale gained by increasing the manufacture and distribution of drugs to large markets. But what HOW COMPANIES ARE SUPPORTING THE GENOMIC MEDICINE EVOLUTIONBy Demaris Mills, President, Integrated DNA Technologies, Danaher CorporationSDemaris Mills
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