Purespring Therapeutics Presents New Data on Alport Syndrome Treatment

London, UK, [14] October 2026, Purespring Therapeutics, a precision nephrology company, announced it will present new data on its pipeline progress in Alport Syndrome at the American Society of Nephrology (ASN) Kidney Week 2026, taking place 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, affecting approximately 1 in 2,230 individuals, with males typically presenting during childhood or early adulthood. The disease is characterized by persistent haematuria, proteinuria, hypertension, and a declining renal function, with many patients reaching end-stage kidney disease by a median age of 25.

Purespring's PS-003 uses the company's podocyte-targeting GlomThera™ platform, already in clinical development for IgA nephropathy. The preclinical data package demonstrates that PS-003 can restore the key collagen protein missing in XLAS, capable of driving functional collagen heterotrimer formation and secretion in both human podocytes and a mouse model. Additionally, the data show vector delivery in Göttingen minipigs with no treatment-related safety concerns. This supports the progression of PS-003 towards first-in-human testing.

The second presentation will report on 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 findings reveal the significant scale and diversity of Alport Syndrome across populations. X-linked Alport Syndrome, relating to COL4A5 mutations, accounts for most Alport-related kidney failure, despite being associated with relatively fewer variants. The study highlights G624D, a later-onset variant that accounts for a substantial proportion of adult-onset cases in the US and Europe. This suggests that genetic testing may be underused in older patients with suspected XLAS.

The data also indicate that intronic variants contribute significantly to the burden of disease. Real-world data from Natera’s Renasight™ database from nephrology clinics show a marked greater incidence of patients with COL4A3 or COL4A4 variants when compared to the general US population. Purespring's PS-003 may also be extended beyond XLAS to treat forms of Alport Syndrome caused by variants in COL4A3 and COL4A4.

Purespring Therapeutics is backed by leading biotech investors, including Syncona Limited, Sofinnova Partners, Gilde Healthcare, Forbion, and the British Business Bank, and has raised £115 million ($150 million) to date. The company's lead programme, PS-003, aims to address the underlying genetic cause of XLAS and potentially modify disease progression, offering a transformative approach to treating this debilitating condition.