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 we are opening clinical study centres in the UK and the US.
Backed by a wealth of preclinical data, PS-002 has demonstrated itself as an important novel modality to treat IgAN, delivering the complement factor I (CFI) gene to podocytes. This approach has the potential to alleviate complement-driven glomerular diseases at the site of disease without affecting systemic complement activation.
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. Feedback highlighted the opportunity for a “once-and-done” therapy.
These outputs will help optimise clinical trial design and inform future development of PS-002 and other planned gene therapies for renal disorders.
Glomerular
Glomerular Kidney Diseases
Purespring has developed the first and only glomerular-targeted delivery system to change the trajectory of kidney disease.
Glomerular diseases constitute a diverse set of conditions including genetic disorders, inflammatory or autoimmune disorders, and conditions secondary to systemic diseases. Glomerular diseases affect patients of all ages and are the main cause for kidney failure in up to 25% of patients receiving dialysis, as well as the main cause for kidney failure among children, adolescents, and young adults.
Early stages of a glomerular disease may not cause any symptoms, but as the ability of the kidney to filter blood to create urine is impaired, it will often become possible to detect proteins and sometimes blood in the urine.
Patients sometimes notice when they develop frothy or dark colour urine. In many cases a kidney biopsy, using a needle to collect a small piece of kidney tissue, is required to diagnose glomerular disease. A genetic screen will also frequently be performed, especially in young patients suspected of a glomerular disease.
At Purespring, we have made it our mission to empower patients with kidney diseases to live fuller, healthier lives through the development of targeted, first-in-class genetic therapies to preserve renal function. We aim to treat kidney disease at its source, prevent renal failure and avoid the need for dialysis or transplantation.
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 deposition of immunoglobulin A (IgA) in the glomeruli of the kidney, leading to complement activation, 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 experience progressive loss of kidney function, leading to end-stage kidney disease within 20 years of diagnosis. 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.
IgA nephropathy (IgAN) is the most common primary glomerulonephritis worldwide, with an estimated global prevalence of 2.5 per 100,000 individuals.
NPHS2
NPHS2 Mutations
Kidney diseases caused by NPHS2 mutations represent a significant group of genetic glomerular disorders that primarily manifest as steroid-resistant nephrotic syndrome (SRNS) and focal segmental glomerulosclerosis (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 kidney disease 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.
Common symptoms include severe swelling, particularly around the eyes and face in the morning, progressing to the legs, abdomen, and other body parts throughout the day.
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.