March 2024
When and how did you first get involved with Purespring?
Purespring caught my attention when I was looking for exciting new developments in nephrology. At that time I was head of global clinical development at CSL Vifor, a company focused on nephrology. I got to know Moin Saleem, Purespring’s founder and now Chief Scientific Adviser, who is also Professor of Paediatric Renal Medicine at the University of Bristol and saw him present on the technology. When a role became available at Purespring, I was very keen to join the company.
Prior to Purespring, you held positions within the clinical development teams at CSL Vifor, AstraZeneca, Bayer, and Antisoma. How has this experience informed your approach to gene therapy at Purespring?
What I bring to Purespring is broader nephrology drug development experience, a network with other stakeholders in the nephrology space and some knowledge around endpoints and late-stage development.
The Purespring team already had a lot of gene therapy experience when I joined. Purespring is taking a safe and well-known path to bring our programs to the patients, de-risking the approach significantly. The innovation lies instead in the cell type we target, and how we achieve that targeting.
What do you think are the biggest challenges for a company in gene therapy at the moment?
Many companies are struggling to achieve the type of curative efficacy that was originally envisaged as one of the drivers of gene therapy. This is still the goal of gene therapy and absolutely achievable, but it is more challenging to implement than some people anticipated.
Also, several programs have experienced toxicity issues when pushing the dose to maximize efficacy. Investors have also become more cautious, a situation which the recent funding environment has amplified. There are signs that this is changing now though, as more and more VC deals have been announced lately. This is great news for companies in gene therapy, as well as for patients.
What has been missing from innovation in kidney therapeutics, and how will gene therapy change this?
Kidney therapeutics have been fundamentally revolutionized over the past 10-15 years. We have had more new drugs reporting successful clinical results over the past few years than ever before, and companies are investing more in nephrology than ever.
There is competition in some indications, such as IgA nephropathy, which is really healthy. However, few of the novel nephrology therapies are truly disease modifying, and many patients continue to require chronic treatment for life. Kidney gene therapy offers potential to cure many conditions.
Your scientific founder, Moin Saleem and his colleagues, published some exciting data earlier this year establishing transduction of the podocyte with AAV gene therapy. How does that change the game for Purespring and for kidney medicine more generally?
The breakthrough paper published by Moin and collaborators in Science Translational Medicine is the first indication that it is indeed possible to target the podocyte with gene therapy.
Podocytes are the key kidney cells: they control filtration, inflammation and structure of the glomeruli. However, they only make up a very small fraction of the cells in the kidney. Hence the biotech industry has been attempting to specifically target podocytes for decades, with limited, if any, success. The recent publication provides evidence that podocyte targeted gene therapy is feasible and efficacious, and that podocytes can be specifically targeted. For Purespring the publication was great news, as we can now openly share much more details around our technology with the external world, and it establishes Purespring as the leader in podocyte targeted gene therapy.
You have worked in all phases of drug development and medical across large pharma and biotech, how will you collaborate with Purespring’s corporate leadership to ensure that scientific decisions align with the company’s overall goal?
Information sharing is key. Also, for a company like Purespring which is founded on breaking science and alone in an entire therapeutic area (gene therapy for glomerular diseases and podocytopathies) the strategic thinking is really key. Purespring has a diverse team of leaders well suited to make the right strategic decisions at a key timepoint for the company.
What are the next steps for Purespring and what are you excited about that’s coming up?
As well as the product development we are resourcing ‘in-house,’ we are also capitalising on the interest and enthusiasm in our platform to deliver genes to the kidney and are looking for the ideal partners to take our projects and technology forward. We want to ensure we augment and exploit the opportunities created by the novel Purespring technology in full. We are excited to share more details on this in 2024.