Purespring Therapeutics shares preclinical data at ERA Congress

London—24 May 2024—Purespring Therapeutics, a trailblazing company in the field of gene therapy for kidney diseases, has presented pioneering preclinical data at the 61st European Renal Association (ERA) Congress. This data showcases the effectiveness of adeno-associated virus (AAV) gene therapy in targeting podocytes to replace defective genes or modulate protein production, offering a novel therapeutic approach for a range of kidney diseases.

The presentation, titled “A novel podocyte gene therapy enables pathway to clinical translation for the treatment of glomerular diseases,” is being delivered at the ERA Congress in Stockholm, Sweden, which runs from May 23-26. Purespring’s innovative AAV gene therapy demonstrates a significant breakthrough in addressing the technical and physiological challenges traditionally associated with gene therapy in kidney cells.

Sam Illingworth, Head of Translational Research at Purespring, presented the development of a new AAV gene therapy that can effectively deliver transgenes to podocytes—a specialized kidney cell central to many glomerular diseases. The research highlighted the progress with the PS-001 gene therapy, showing positive results in both murine and large animal models. This therapy successfully targeted transgenes to podocytes, corrected defective genes, and restored kidney function, indicating a promising new treatment modality for various kidney diseases.

According to Alice Brown, Chief Scientific Officer at Purespring, the new data underscores the potential of AAV gene therapy to efficiently, specifically, and safely deliver transgenes to podocytes. This progress marks a significant step towards clinical development for PS-001 and other kidney-related conditions in their pipeline. Brown expressed enthusiasm about sharing these findings with the international nephrology community.

Julian Hanak, CEO of Purespring, emphasized the broader implications of the podocyte-targeting approach, which could potentially halt, reverse, and even cure chronic kidney diseases. Chronic kidney disease affects over 840 million people globally, imposing a substantial burden on patients, their families, and healthcare systems. The successful demonstration of this approach in preclinical models is a crucial milestone in addressing these challenges.

The preclinical studies included toxicological assessments to ensure the safety of the treatment, which showed no adverse effects on renal or other organ functions. The PS-001 gene therapy is being developed to treat steroid-resistant nephrotic syndrome caused by mutations in the NPHS2 gene. This therapy might become the first podocyte-targeted gene therapy to enter clinical trials.

The research utilized an AAV gene therapy platform featuring a capsid highly effective at transducing human podocytes and a promoter that drives gene expression specifically in these kidney cells. The PS-001 product, which encodes the NPHS2 gene, was tested in a murine model of the disease. The in vivo studies demonstrated successful NPHS2 delivery, podocin expression in glomeruli, and improvement in renal function. This was evidenced by the resolution of severe proteinuria, improved serum albumin levels, and reduced glomerulosclerosis.

To translate these findings to a clinical setting, additional experiments were conducted in pigs. Administration of PS-001 led to transgene mRNA expression in a significant portion of kidney glomeruli, with podocin protein specifically detected in podocytes. No gene expression was found in off-target organs like the liver, confirming the specificity of the therapy. Moreover, toxicological evaluations showed no treatment-related safety concerns.

Purespring Therapeutics, founded on the research of Professor Moin Saleem from the University of Bristol, is dedicated to advancing gene therapies for both monogenic and non-monogenic chronic renal diseases. Their groundbreaking work in targeting podocytes using AAV gene therapy represents a significant advancement in the treatment of kidney diseases, which are currently inadequately addressed by existing therapies.

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