- New preclinical data demonstrates PS-003 restores the key collagen protein missing in X-linked Alport Syndrome (XLAS) and is presented alongside findings from one of the largest genetic studies of Alport Syndrome to date
- Findings support the rapid advancement of PS-003, towards first in-human studies, with the goal of addressing the underlying genetic pathophysiology of Alport Syndrome
- New global genetic data from more than 1.2 million genomes reveal the significant scale and diversity of Alport Syndrome across populations in the US, UK, Europe and Japan
London, UK, [14] October 2026 - Purespring Therapeutics, a precision nephrology company, pioneering podocyte-targeted, genetically-driven therapies where unmet need is greatest, today announced that it will be giving two oral presentations at the American Society of Nephrology (ASN) Kidney Week 2026, being held from 21-25 October 2026 in Denver, Colorado.
The presentations will cover preclinical data for PS-003, a novel gene therapy candidate for the treatment of X-linked Alport Syndrome (XLAS), and one of the largest genetic studies of Alport syndrome to date. XLAS is one of the most common inherited causes of kidney failure with a huge unmet medical need driven by the absence of approved therapies. XLAS is caused by mutations in a collagen gene, COL4A5, in the glomerular basement membrane. Development of treatments that tackle the underlying genetic cause of the disease have been hampered by the complexity of the COL4A5 gene and the difficulty targeting the podocyte, the cell type in which it is expressed. An oral presentation titled "GlomThera platform enables durable COL4A5 expression for the treatment of X-Linked Alport Syndrome"will outline how PS-003 uses Purespring's podocyte-targeting GlomThera platform, already in clinical development for IgA nephropathy, to demonstrate delivery of functional COL4A5 gene to podocytes. The preclinical data package will show that PS-003 and a PS-003 murine surrogate can produce full-length COL4A5 protein capable of driving functional collagen heterotrimer formation and secretion both in human podocytes and in a mouse model respectively. It will also demonstrate vector delivery in Göttingen minipigs with no treatment-related safety concerns. Together, this data supports the progression of PS-003 towards first-in-human testing.
Title: GlomThera platform enables durable COL4A5 expression for the treatment of X-Linked Alport Syndrome
Session title: Glomerular Diseases: Therapeutic Strategies Emerging from Basic Research [OR1401-2]
Date & time: 24 October 2026, 5.20pm MDT
Session room: Mile High Ballroom 4D
Presenter: Sarah Smith-Moore, Head of Cell and Molecular Biology, Purespring Therapeutics
A separate oral presentation titled "The global mutational landscape in COL4A3, COL4A4 and COL4A5, and estimating the Alport Syndrome (AS) population" will report one of the largest genetic studies of Alport Syndrome to date. Drawing on more than 1.2 million genomes from the US, UK, Europe and Japan, the study estimates the frequency of disease-causing variants in COL4A3, COL4A4 and COL4A5 across these populations. The findings reveal the scale of the disease burden and indicate that X-linked Alport Syndrome relating to COL4A5 mutations account for most Alport-related kidney failure, despite being associated with relatively fewer variants. The data also highlights G624D, a later-onset variant that accounts for a substantial proportion of adult-onset cases in the US and Europe. This finding suggests that genetic testing may be underused in older patients with suspected XLAS. The study further indicates that intronic variants contribute significantly to the burden of disease. Real-world data from Natera's RenasightTM database from nephrology clinics shows a marked greater incidence of patients with COL4A3 or COL4A4 variants when compared to the general US population.
The approach used for PS-003 may also be extended beyond XLAS to treat forms of Alport Syndrome caused by variants in COL4A3 and COL4A4.
Title: The global mutational landscape in COL4A3, COL4A4 and COL4A5, and estimating the Alport Syndrome (AS) population
Session: Noncystic Genetic Diseases: Multi-Omic Discovery and Emerging Therapeutic Approaches [OR1202]
Date & time: 23 October 2026, 5:20pm MDT
Session room: Mile High Ballroom 4D
Presenter: Fredrik Erlandsson, Chief Medical Officer, Purespring Therapeutics
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For further information, contact:
Purespring:
contact@purespringtx.com
+44 (0)20 3855 6324
LinkedIn
ICR Healthcare:
Sarah Elton-Farr, Emily Johnson
purespring@icrhealthcare.com
About Purespring
Purespring Therapeutics is a clinical-stage precision nephrology company pioneering podocyte-targeted, genetically-driven therapies where unmet need is greatest.
The Company's proprietary, category defining GlomThera platform is designed to enable local administration of lower dose genetic therapies directly to the podocyte - a specialised kidney cell implicated in a majority of glomerular disease - with the potential for durable treatment effects. Purespring's multi-asset pipeline is focused on renal indications affecting large and underserved patient populations. The Company's lead programmes span both monogenic and non-monogenic disease, targeting Alport Syndrome and IgA nephropathy (IgAN) respectively. PS-003 is advancing toward clinical development in Alport Syndrome, a rare inherited kidney disease with no approved targeted therapies, with the goal of addressing the underlying genetic cause and potentially modifying disease progression. PS-002, which is the first kidney targeted gene therapy ever to have been administered to a patient, aims to evaluate a differentiated approach for IgAN patients with the goal of delivering durable efficacy and an improved safety profile.
Purespring is backed by leading biotech investors, including Syncona Limited, Sofinnova Partners, Gilde Healthcare, Forbion, and the British Business Bank, and has raised £115m ($150m) to date.
For more information, please visit purespringtx.com and follow us on LinkedIn.
About Alport syndrome
Alport Syndrome is a monogenic kidney disorder caused by pathogenic variants in genes encoding for type IV collagen. These mutations disrupt the structure and integrity of the glomerular basement membrane, leading to progressive renal damage that can result in kidney failure. The X-linked Alport (XLAS) form, estimated to affect 1 in 2,230 individuals, typically presents in males during childhood or early adulthood with persistent haematuria, followed by proteinuria, hypertension and a declining renal function. Disease severity varies by genetic subtype, but progression in males with X-Linked AS is often rapid, with many patients reaching end-stage kidney disease at a median age of 25. With no FDA approved therapies, most male patients diagnosed with XLAS will require haemodialysis and/or a kidney transplant. This highlights the urgent need for disease-modifying therapies that address the underlying cause of disease and can slow or prevent progression to kidney failure. PS-003 is designed to deliver the COL4A5 gene directly to the kidney in XLAS to restore local production of functional type IV collagen trimers with the potential to meaningfully transform patient outcomes.
