24/7 BIOPHARMA - issue 1 / October 2024

WUXI APPTEC

Could European research advance more quickly by embracing end-to-end CRDMOs?

As a scientist, I often wonder whether we are truly progressing in our quest to find a cure for chronic diseases or merely slowing down their inevitable march. I ask myself whether we are progressing from bench to bedside in the right way and do we have the right tools to get there? For example, with so many new biotech hubs now growing in Europe surely we should embrace the CRDMO approach – an approach pioneered and still led by WuXi AppTec [more on this later]. With the recent advancements in tackling some of the most challenging diseases—Parkinson’s, Alzheimer’s, and idiopathic pulmonary fibrosis (IPF)—I find myself genuinely excited. These are conditions that have perplexed researchers for decades, and yet, we are on the cusp of therapies that could transform how we treat them. So while reading through a September series of interviews from WuXi AppTec’s [WXPress], I was struck by just how far we’ve come and felt inspired by what’s still possible when we get the mix of ingredients right. By this, I mean a good blend of new innovations, expertise and capabilities to complete the complex journey of a therapy from bench to patient. The reason for my newfound optimism: well, there was a time when the notion of reversing neurodegenerative or fibrotic diseases seemed like science fiction.

Now, we’re developing therapies that not only halt disease progression but also hold the potential to reverse some of the damage. As a researcher who has spent countless hours in the lab, this feels like a moment of profound accomplishment for the scientific community. Take Parkinson’s disease, for example. I’ve followed with particular interest the advancements in induced pluripotent stem cells (iPSCs) and the potential they offer. During my PhD, I worked with iPSC-derived hepatocyte-like cells to treat liver injury in mouse models, focusing on engraftment and functional restoration. Reflecting on my own work, I cannot help but draw parallels to the iPSC-based therapies now being developed for Parkinson’s. It’s thrilling to see the same technology I once explored in the lab is now at the forefront of clinical trials and offers real promise in treating neurological diseases. Similarly, in the case of idiopathic pulmonary fibrosis (IPF), which affects approximately 150,000 individuals in the United States, the signs of disease reversal have now emerged. Endeavor BioMedicines’ ENV-101 has demonstrated the ability to reverse key markers of lung fibrosis in IPF. The improvements in lung function and architecture seen in clinical trials are unprecedented.

88

TWENTYFOURSEVENBIOPHARMA Issue 1 / October 2024