Genomic Medicine Demands Manufacturing Innovation

Genomic medicine is at the forefront of a medical revolution. Every year, there is an increasing number of approvals for genomic medicines, driven by innovative precision medicine platforms targeting diseases that currently lack effective treatments. Viral vectors, particularly adeno-associated virus (AAV), have effectively treated diseases stemming from monogenic mutations. Gene-modified cell therapies, such as CAR T cell therapies, have successfully treated some late-stage blood cancers but face challenges when applied to solid tumors. Both cell and gene therapies still face challenges, including lengthy development, manufacturing, and batch-release timelines.

RNA-based therapies have emerged in recent years, highlighted by the rapid development and large-scale production during the COVID-19 pandemic. mRNA has demonstrated its ability to instruct cells to express antigens or proteins transiently, though it is less efficient for therapies requiring systemic protein replacement. Novel RNA variants and oligonucleotides, such as self-amplifying RNA (saRNA) and circular RNA (circRNA), as well as short interfering RNA (siRNA), guide RNA (gRNA), and antisense RNA (aRNA), are now being explored. Additionally, induced pluripotent stem cells (iPSCs) and exosomes are gaining interest within the scientific community as therapeutic agents or delivery tools.

All these modalities, along with their current and future variants, require manufacturing solutions that can produce them at the necessary scale and quality standards. The promise of these therapies can only be realized when effective science is paired with efficient manufacturing to provide cost-effective treatments to patients in need.