Transforming Scientists into Drug Developers

Companies like Pall, Cytiva, and Precision NanoSystems – one of the newest additions to the Danaher Life Sciences portfolio – can provide all of the technologies and expertise necessary to manufacture a drug. All scientists need to focus on is discovery.

What is the story behind Precision NanoSystems?
Taylor: My educational background is a combination of an engineering physics degree with a PhD in genetics. After my PhD, I co-founded the company with Euan Ramsay, and two professors:
Dr Carl Hansen and Dr Pieter Cullis, who is a world-renowned professor and industrialist in the area of lipid nanoparticle delivery systems. The company was formed around the promise of new therapeutic modalities, such as RNA, and the challenges of enabling and delivering these as therapeutics. Over the years, we have put together the full technology stack to make what we call genomic medicines. This includes manufacturing technology, such as continuous flow manufacturing for making nanoparticle RNA delivered drugs and lipid nanoparticle delivery systems. We also offer services to clients and work with drug developers to help them get their genomic medicines to market.

How did Precision NanoSystems come to join Danaher Corp’s Life Sciences Platform?
Glover: James and I have a history; we did our PhDs together in the genetics program at the University of British Columbia, Vancouver. Then, we headed off our separate ways. My PhD focused on advanced therapies, such as cell therapies and gene therapies. I was very interested in how to manufacture these therapies and I have built my career around developing tools and technologies to help bring advanced therapies to market.
Today, I run the gene therapy business at Pall Corporation where there is a strong focus on large-scale viral vector manufacturing, which is the primary method of delivering genetic material both in vivo and ex vivo at this point in time. However, there are also alternatives to viral vectors. While exploring these alternatives, I bumped into James at a conference and he told me about the work Precision NanoSystems was doing.
I instantly saw that there was great synergy between his company and what Pall was doing with gene therapy manufacturing.
Everything progressed from there. In June 2021, it was announced that Precision NanoSystems would join Danaher’s Life Sciences platform, complementing the portfolios of Cytiva and Pall. Now, the companies are getting to know each other in a more meaningful way – discovering how our combined expertise can help drug developers get exciting genomic medicines to patients.

What trends are you seeing in the industry right now?
Glover: Today, we have so many options for tackling disease. The pharma industry started off with small molecules and then graduated to proteins (primarily monoclonal antibodies), and now we are seeing the rise of cell and gene therapies, which are tackling unmet needs that neither small molecules or proteins have been able to address. Cell and gene therapies are a long-term driver of change within the industry, but COVID-19 has also accelerated change by bringing viral vectors, and specifically mRNA, to the forefront. mRNA therapeutics have been in development for many years, but COVID-19 has been a tremendous catalyst for the field. We now find ourselves at a really exciting time; when we start thinking about new or unmet clinical needs there is a wider range of tools and technologies that can be applied. Both Pall and Precision NanoSystems can support large-scale manufacturing for many different kinds of therapeutics.

Taylor: I am really excited by the possibilities of genomic medicine and its continued clinical validation. There are many tools in the genomic medicine toolbox that allow us to manipulate disease-causing genes in all the various ways required to treat disease. As a few examples: Small interfering RNA (siRNA) allow us to silence disease-causing genes. Whether it is a rare disease, whereby the patient inherits a gene that expresses a problem causing protein, or in a cancer, whereby the patient’s genes mutate to express proteins that drive the cancer, silencing these disease causing genes can have significant impact on the patient. Messenger RNA (mRNA) allows us to express proteins that are missing or not working, or in the case of vaccines we can teach the immune system to recognize pathogens. mRNA COVID-19 vaccines are the tip of the iceberg on the impact mRNA will make in vaccines and the treatment of many diseases. New gene editing technologies, like CRISPR, allow us to fix genetic problems directly in a patient’s genome. Gene editing technologies are enabled by delivering an RNA guide strand and an mRNA that expresses the endonuclease to enable sequence specific gene editing. Similarly, there are wide range of other genetic modulators, like epigenetic regulators, that can be expressed in cells via delivery of mRNA, creating a completely new era of what is possible in molecular medicine.
The power of genomic medicine is immense – and the industry is now recognizing the massive impact it can have on society and the human condition.

How do you work with customers?
Taylor: We support innovative drug development by helping our clients to make their next big therapeutic and we have developed solutions across the full drug development paradigm. At Precision NanoSystems, we typically work with clients from the ideation of their drug project through clinical development. Our benchtop equipment, such as our NanoAssemblr Ignite system, is often one of the earliest “employees” in a startup, and represents the start of a multi-year partnership with our clients!
We are developing the full stack of technologies required to make genomic medicines and provide these to our clients across the full drug development process. Our platforms include manufacturing technologies, delivery technologies, and the expertise needed to help these companies move forward with their drug candidates. We support our clients from early stage discovery, through clinical manufacturing, and all the way up to the commercial stage. We’re now reaching the stage with genomic medicines that once you know how you want to manipulate a disease causing gene, you can create the associated genomic medicine candidate within days for pre-clinical testing. Through our platform, we enable any scientist to be a genomic medicine developer. I believe we are at a tipping point where the broader scientific community is realizing how accessible these technologies are and we will see a significant acceleration of innovation in medicine as a result. Now that we are part of the Danaher group, we are working with our colleagues to develop truly comprehensive, end-to-end drug development and manufacturing solutions.

Glover: Companies like Pall, Precision NanoSystems, and our sister company Cytiva have significant experience, particularly on the manufacturing side.
A fantastic example of the value Pall can bring as a manufacturing partner is seen in our work with AstraZeneca and the University of Oxford on their COVID-19 vaccine. They partnered with us early on in the process and we contributed our expertise on how our equipment could be used to manufacture viral vectors on a large scale. We designed a manufacturing process and deployed it to a particular contract manufacturing organization within eight weeks. I don’t think anyone in the industry compiles records, but I’d like to bet that is a record timeframe!
Precision NanoSystems strengthens our know-how further by adding more biological expertise. Together, we are a powerful force that can help companies to bring therapeutics to market faster and with more likelihood of success.

How do you expect the field to evolve?
Taylor: In genomic medicines, new types of modalities are being invented, discovered, and developed every day. mRNA is in the spotlight right now and it is very useful for vaccines for expressing antigens, as well as in other areas such as where an expressing protein is missing or not working properly, but there are many other tools in the genetic medicine toolbox. I think genomic medicine will become the largest class of therapeutics in the not-so distant future – and it will be accessible to all scientists, similarly to how easy it is to make a software app today.
In the future, I believe that every country will have the ability to efficiently set up in-country manufacturing of genomic vaccines and therapeutics. This will increase the security of supply, allow nations to more rapidly respond to future pandemics, and enable countries to treat their citizens more effectively. I also believe that we will reach the stage where bespoke genetic medicines can be designed for the molecular basis of an individual’s disease, and single drug batches can be manufactured for patients.
We are heading towards new therapeutic paradigms that were simply not possible before, and this is happening faster than most people realize.

Glover: Some people say 2021 will be the year of mRNA; others say it will be the year of the lipid nanoparticle. mRNA therapies have been in development for many years, but delivery was always a challenge. We now have a solution – and Precision NanoSystems has been a real pioneer in this area. As Pall, Cytiva, and Precision NanoSystems come together, I’m really excited about how we can deliver mRNA factories to the field as quickly as possible and work with clients on ground breaking new medicines.

Dr Clive Glover is the General Manager, Gene Therapy at Pall and leads the cell and gene therapy business. His work has been published in numerous scientific journals, and he has presented at many conferences. Previously he was responsible for driving product development efforts around cell therapy at GE Healthcare and has also held positions in marketing and product management at STEMCELL Technologies. Clive holds a PhD in Genetics from the University of British Columbia. When not at work,
Clive spends a lot of time singing in Welsh choruses and hiking the Welsh hills.

Dr James Taylor is a Co-Founder and General Manager of Precision NanoSystems, and the leader of the company since invention. James holds a B.A.Sc. in Engineering Physics from the University of British Columbia (UBC), Canada and a PhD in genetics from UBC and the Institute for Systems Biology in Seattle, Washington, USA. James worked at the Seattle-based venture capital firm, Accelerator Corporation, concurrent with his PhD, and the Centre for Drug Research and Development in Vancouver, British Columbia, following. James has extensive experience in the science and commercialization of microfluidics, nanotechnology and systems biology.

Created by and originally published by The Medicine Maker