When and how did you first get involved with Purespring?
Before joining Purespring, I was Senior Vice President and Global Head of CMC at NightstaRx Therapeutics, one of Syncona’s first gene therapy companies, which was acquired by Biogen in 2019. Through my work with NightstaRx I came to be involved with the foundation of Purespring.
Founding Purespring was an exciting and challenging time for us: we were building the team from the ground up, developing a mission and culture, and occupying a new office and facilities. This all happened against the backdrop of the COVID-19 pandemic, and we built the Company through both the 2020 and 2021 national lockdowns and all the obstacles they brought. It just shows what can be achieved by a focused and motivated team.
You have three decades of experience in the biotechnology industry, including leadership positions at Renovo, Cobra Biologics, NightstaRx, and Biogen. What are the most important lessons you have learned?
To succeed in delivering products and value in a pioneering company it’s not enough just to have good science, you also need exceptional teamwork, planning, a good relationship with your investors and, importantly, the best people immersed in a great culture with a strong belief in the company purpose and ambition.
Purespring has a team of all the talents: it not only has a strong track record in developing and manufacturing gene therapies but a collaborative spirit to pursue a common goal – improving the lives of the 840 million people around the world affected by chronic kidney disease.
In particular, you played a central role at NightstaRx and later Biogen, with CMC, which is one of the most critical elements for gene therapy development. How has this experience informed your approach to gene therapy at Purespring?
I think it’s helped give me the ‘long view’. Not only does Purespring have to keep showing that our products can transform the way chronic kidney disease is treated, but we must know how to bring them to patients. This is why we set up Purespring to be a fully integrated company, containing within it all the building blocks necessary to move rapidly through clinical validation and ultimately impact the treatment of chronic kidney disease.
The big picture is important here, as is a plan that takes the products all the way to approval and beyond. Using the team’s – and Syncona’s – deep industry expertise and insights, we are tailoring our approach to address the key challenges in gene therapy such as delivery, manufacturing, and toxicity to bring our gene therapies closer to patients.
What do you think are the biggest challenges for a company in gene therapy at the moment?
There are a number of big challenges in gene therapy development, most urgent among them being delivery, manufacturing, and toxicity—in particular, targeted delivery to the organ while limiting toxicity.
At a macro level, the broader industry is also experiencing challenges such as access to funds to build and scale, attracting key talent, and an evolving global regulatory landscape.
At Purespring, we try to tackle each of these head on. A good example of this is our approach to the podocyte, as the key cell in the kidney, with a targeted delivery. Also, through our gene therapy platform we can use the same AAV vector capsid and promoter for all our products, shortening development timelines, reducing risk and uncertainty while minimising cost across product development and manufacturing. This approach also enables us to achieve efficacy cost-effectively, with a lower dose and improved patient safety. Our robust CMC processes for the whole product pipeline offer time, cost, reliability, and regulatory benefits.
In gene therapy there is a supportive ecosystem, from researchers and scientists to medical professionals, investors, regulators and, crucially, patients. They all see the potential of gene therapy and want to see it progress and succeed – this positive sentiment is immensely helpful and allows us to address any challenges and make progress.
What has been missing from innovation in kidney therapeutics and how will gene therapy change this?
Up until recently, there has been a lack of innovation in the treatment of kidney diseases with a dearth of disease modifying treatments with high efficacy, a good safety profile, and manageable side-effects. Many products available over the last 10 to 20 years treat the symptoms of chronic kidney diseases but do not address the underlying disease and often have to be taken for the duration of a patient’s life, or until they stop being effective. Many patients end up on dialysis awaiting a kidney transplant.
One of the key areas where we are innovating is in targeting the podocyte, one of the most critical components of the kidney. It is also an ideal target for AAV gene therapy as it is terminally differentiated and does not regenerate or divide – all factors that make it suitable as a target and prevent dilution of any gene therapy administered.
By using gene therapy to target the podocyte directly, Purespring has the potential to address the cause of the problem, meaning that transformational disease modifications are in our sights.
What are the next steps for Purespring and what are you excited about that’s coming up?
We’re continuing to build out the team and looking forward to progressing with our clinical development. But ultimately, this is just the beginning. We know that kidney diseases affect a huge population globally, and our targeted approach with the best team, has the potential to radically change the treatment for these patients across a range of indications.