Genomic Medicine Keeps Innovating and Manufacturing Needs to Keep Pace

Genomic medicine — the therapeutic use of cells and genetic materials — is at the center of a medical revolution. Approvals of genomic medicines are growing each year, fueled by new precision medicine platforms that target diseases without current effective treatments. Viral vectors, primarily adeno-associated virus (AAV), efficiently treat some diseases caused by monogenic mutations. Gene-modified cell therapies, notably CAR-T cell therapies, successfully treat some late-stage blood cancers but face obstacles for use with solid tumors. Challenges remain for both cell and gene therapies, including too-long development, manufacturing, and batch release timelines. More recently, we have witnessed the emergence of  RNA-based therapies. The COVID-19 pandemic showed how such therapies could be developed quickly and at large scale. mRNA has proven its ability to instruct cells to express antigens or proteins in a transient manner but is less efficient if the therapy requires systemic protein replacement. Novel RNA variants and oligonucleotides, such as self-amplifying RNA (saRNA) and circular RNA (circ RNA) for the first, and short interfering RNA (siRNA), guide RNA (gRNA), and antisense oligonucleotides (ASO) for the latter, are now being explored. Lastly, induced pluripotent stem cells (iPSCs) and exosomes, used as therapeutic agents or delivery tools, have gained interest in the scientific community.

All these modalities, their current and future variants, demand manufacturing solutions that can produce them at the needed scale and to stringent quality standards. Only when effective science is matched with efficient manufacturing can cost-effective therapies be made available to patients who are waiting for them.

Learn more: https://www.pharmafocuseurope.com/manufacturing/genomic-medicine-keeps-innovating-manufacturing