Clinical Trials
We have US FDA IND clearance and UK CTA approval for our Phase I/II clinical trial of PS-002 in patients with IgAN and are actively recruiting participants at sites across the US and in the UK.
Backed by a wealth of preclinical data, PS-002 has demonstrated itself as an important novel modality to treat IgAN. By enabling local modulation of complement through delivery of the complement factor I (CFI) gene to the podocyte, it has the potential to provide a differentiated approach with durable efficacy and an improved safety profile compared with approved therapies, as well as those currently in development.
For more information go to clinicaltrials.gov
Our Phase I/II clinical trial has been informed by insight sessions with current IgAN patients and carers across the US and UK, with feedback highlighting the opportunity for a “once-and-done” therapy. This was presented at the American Society of Nephrology Kidney Week in November 2025, and conducted in partnership with the IgA Nephropathy Foundation and the UK National Institute for Health and Care Research (NIHR).
These outputs have helped inform our Phase I/II clinical trial design for PS-002 and will inform future development of this asset and our broader pipeline.
Therapeutic Areas
IgA Nephropathy
IgA nephropathy (IgAN) is the most common primary glomerulonephritis worldwide, with an estimated global prevalence of 2.5 per 100,000 individuals.
It is a chronic, progressive autoimmune disease characterised by the formation of and subsequent deposition of immunoglobulin A (IgA) in the glomeruli of the kidney, leading to persistent inflammation, tissue injury and progressive loss of kidney function.
IgAN often presents in adolescents and young adults with haematuria, proteinuria, and hypertension. Approximately 20–40% of patients develop progressive loss of kidney function, leading to end-stage kidney disease within 20 years of diagnosis, while high-risk patients may require dialysis or transplantation in as few as five years.
Current and other planned therapeutic approaches for IgAN are not sufficient for all patients and can pose tolerability challenges, underscoring the urgent need for disease-modifying and kidney-targeted therapies. By enabling local modulation of complement through augmentation of the complement factor I (CFI) gene, PS-002, which has entered first-in-human studies, aims to provide a differentiated approach with durable efficacy and an improved safety profile.
IgA nephropathy (IgAN) is the most common primary glomerulonephritis worldwide, with an estimated global prevalence of 2.5 per 100,000 individuals.
Alport
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 (GBM), 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 with persistent haematuria, followed by proteinuria, hypertension and declining renal function. For males, this often occurs during childhood or early adulthood. Disease severity varies by genetic subtype and sex, but progression in males with XLAS is often rapid, with many patients reaching end-stage kidney disease (ESRD) at a median age of 25.
With no approved therapies, most males and a significant portion of female patients diagnosed with XLAS will reach ESRD and 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 heterotrimers with the potential to meaningfully transform disease severity and patient outcomes.
Our broader pipeline includes assets which will target those with Autosomal Recessive Alport Syndrome (ARAS) and those with Alport kidney disease.
NPHS2
NPHS2-driven FSGS
NPHS2 is an important driver of genetic FSGS.
The NPHS2 gene encodes podocin, with mutations in this gene causing disruptions to its normal function. When podocin is misfolded or absent, the kidney’s filtration system becomes compromised, allowing proteins that should remain in the bloodstream to leak into the urine (leading to proteinuria). This process leads to progressive scarring of the glomeruli and can result in FSGS, where segments of the kidney’s filtering units become scarred and non-functional.
NPHS2-related FSGS typically presents in early childhood, often within the first few years of life. Unlike other forms of nephrotic syndrome, NPHS2-associated disease is inherently steroid-resistant, making management challenging.
Additionally, NPHS2 nephropathy is thought to be responsible for around 4% of adults with FSGS.
Without effective treatment, many patients experience progressive kidney function decline, with some requiring dialysis or kidney transplantation within years of diagnosis.
Current treatment approaches are primarily focused on symptom management, rather than targeting the underlying genetic cause of the disease.
Platform
The GlomThera™ platform is based on validated technologies, integrating and converging best-in-class approaches and is de-risked through tried and tested vector engineering.