The Quest for Safer, More Targeted Therapies for Patients

In the span of one week, two patient groups’ lives were irrevocably changed with the approval of first-in-class gene therapy treatments for Hemophilia A and Duchenne’s Muscular Dystrophy (DMD). Both conditions are life-threatening but with these new treatments, it’s possible for a diagnosed individual to shift their thinking from “managing their disease” to living without it.

Innovators in biopharma continue to pursue these types of tailored therapies and vaccines using a variety of platforms from cell and gene therapy to antibodies, to molecular biology such as messenger RNA (mRNA). Due to the recent success of mRNA vaccines and initial phase one and two results, leaders in the field are entertaining the possibility of a cancer vaccine approved for use in the next five years. Without a doubt, the race is on for scientists. Time is critical for diagnosed patients, and Waters continues to be at the front of the line supporting the development of these life-saving medicines.

Advancing a shared mission and complementary analytical technologies

This year, Waters acquired Wyatt Technology to build on our revitalized innovation engine and accelerate the development of biological and cell, and gene therapies for patients around the world.

Waters and Wyatt share a mission to raise awareness of our solutions and instruments so that scientists have the insights they need to drive their own development projects that aspire to increase the availability and affordability of integral therapies. The power of this combination will extend the reach of cutting-edge technologies and scientific expertise in more than 100 countries.

As Waters Division vice president and general manager, I’m thrilled to work closely with my new colleague Michelle Chen, Vice President of Application Sciences. Our newly integrated team will foster a culture that is deeply rooted in science and customer success.

We share our respective answers to primary questions about the state of our technologies, and how we will use our global footprint to deliver life-changing medicines to patients faster.

Q: With time being so imperative for biotherapeutics, how will Waters build upon Wyatt’s successful legacy and accelerate value creation for our customers?

A: Fraser: Both Waters and Wyatt have a proud track record of innovation in biotherapeutics. This reputation has been largely driven by working closely with customers to understand their most important unmet needs and developing solutions that solve their biggest challenges.

Now that we are one company, we can combine our joint legacies and continue to focus on:

• Innovations that deliver analytical insight.
• Highest levels of robustness and reliability.
• Increased levels of usability.

These three areas combined will ensure that our customers have the correct information they need at the time they need it and will enable them to make the correct decisions to advance their innovation programs.

Q: What are scientists’ current challenges with analytical work and how are we addressing these needs?  

A: Michelle: Integration of instruments from different manufacturers has always been a significant challenge for analytical scientists in both the biopharmaceutical and material industries. Specifically, addressing the compatibility of instruments and aggregating data from them for answers is often time-consuming and error prone. For many years, Wyatt and Waters instruments have worked well together but were not fully integrated. As a unified company, we offer our customers improved options for cohesive solutions.

Q: How do scientists currently measure the critical parameters of biotherapeutics?

A: Michelle: Critical parameters of biotherapeutics depend on product, application, and process. They include identity, impurities, aggregation, and other critical quality attributes (CQAs). In the aforementioned treatments for Hemophilia A and DMD, both gene-based therapeutics are delivered by adeno-associated viral vectors (AAV). For these AAV-based gene products, there are close to 10 physical attributes that require routine measurements throughout the development cycle. Traditionally, six or more assays are needed to measure these attributes. It is costly not only in time, resources, and sample quantity but also in validating all these different assays and associated reagents.

By using a method developed at Wyatt, which incorporates size exclusion chromatography with multiangle light scattering (SEC-MALS), all these physical parameters can be measured in one single run. We also have a similar multi-attribute quantification (MAQ) solution for mRNA and its delivery vehicle called lipid nanoparticle (LNP). Now working with the scientists and engineers at Waters to better integrate the upstream liquid chromatograph, columns, and software, we are making Wyatt AAV and LNP solutions more comprehensive, robust, user-friendly, and accessible to customers.

Q: What will the combination of Waters and Wyatt instruments, technologies, and people look like?

A: Fraser: Over time, our customers can expect to see a few things here. Our instruments will be better designed to fit together, both from a software and hardware integration standpoint. This doesn’t mean that the Wyatt detectors will not be compatible with liquid chromatography (LC) from other vendors, but it does mean that customers that use Waters LCs with Wyatt detectors will have a best-in-class user experience. We will also see more LC columns that are fine-tuned to give the highest quality results with Wyatt detection capabilities; two are already available and more are coming for workflows such as AAVs and LNPs.

From a people perspective, the Wyatt team will always be the experts in helping customers select the best instrument for their workflows and in supporting and servicing those instruments. At the same time, our new Wyatt colleagues are building strong relationships with the broader Waters team and training them on the capabilities of the Wyatt portfolio. This will ensure that our entire team is set up to support customers across the globe.

Q: Are we expecting to expand our global footprint and multi-industry presence and how?

A: Fraser: Wyatt is a pioneer and recognized leader in light scattering technology, which is key to the characterization of monoclonal antibodies and cell and gene therapies.  

Light scattering is a useful technique for interrogating the carrier molecule for gene therapies such as AAV and LNP because of the depth of information it provides in terms of ID, safety, and efficacy. As these molecules become “smarter” for more directed and efficient delivery, light scattering will continue to be used to establish genomic load, ID, and charge – three critical pieces for efficacious therapy.

However, light scattering’s utility isn’t only for biologic applications. Wyatt, just like Waters, started their business in the field of polymers, and this space continues to be rich with examples of how polydispersity is linked to a macro property of a polymer, like flow and other mechanical properties, which are direct measurements of the Waters-TA rheometer and dynamic mechanical analyzer.

The synergy between Wyatt and Waters starts with the applications. Through Waters’ broader and deeper exposure worldwide, we are excited to be able to make these solutions more easily accessible.  

Q: What is on the horizon for analytical instrument providers that will help scientists accelerate their timelines?

A: Michelle: As analytical instrumentation providers, we will continue to listen to our customers, and provide robust, automated, and ease-of-use instruments, software, and workflows. Of course, these products will be accompanied by our world-class training and support. As Fraser mentioned earlier, both Wyatt and Waters share the same philosophy of delighting customers and ensuring customer success. Both of us look forward to working together with our colleagues in the biopharma and material fields as partners to address unmet analytical needs, and improve the time to market, affordability, safety, and efficacy of their products.

We can envision a world where inherited genetic disorders could be cured or prevented through the types of therapies our work directly accelerates. This is a world that is better than we found it. Our goal is to create new value through the expanded global reach and combination of Waters and Wyatt technology. The delivery of these two pieces along with our deep scientific expertise, will support our customers to make life-changing therapies more accessible.

---

Additional Resources:

Biotherapeutics Solutions

References: