Platform
Our proprietary GlomThera™ platform is the only platform designed to deliver therapeutics directly to the podocyte, offering a novel approach to the treatment of kidney diseases.
The GlomThera™ platform uses adeno-associated virus (AAV) gene therapy—a proven approach for delivering genetic material to cells and is administered through established interventional radiology techniques.
This enables dosing more than 10-fold lower than systemically administered AAV gene therapies, reducing systemic exposure while optimising the potential for therapeutic effect.
GlomThera™ uses a validated AAV capsid that directly targets the core of the podocyte with precision.
MoA
Where precision powers performance.
01.
Payload
Interchangeable payload delivered with precision promoters targets site of disease
02.
AAV Capsid
Clinically precedented AAV capsid to deliver our payload to podocytes
03.
Podocyte
The cells take up our therapy and express our payload as functional proteins
Our platform is versatile, supporting different genetic payloads to address multiple glomerular diseases using our core delivery system.
Transforming kidney disease treatment via precision podocyte‑targeted delivery.
Optimised Efficacy.
Non-dividing = durable efficacy
Effective transduction with right capsid
High Yield Manufacturing.
Suspension culture
Translatable through to commercial
Local Delivery.
Leverages routine procedures and devices
Direct uptake into the kidney
Low Dose.
<10% of systemic gene therapy
Minimal off-target exposure
Pipeline
Pipeline.
Our growing pipeline has programmes in development targeting both monogenic diseases and non-genetic diseases.
Both types of diseases can lead to end-stage renal disease, with devastating consequences to patients. Our lead clinical programme PS-002 is focused on complement-driven glomerular diseases and is initially being developed for IgA nephropathy, with pipeline opportunities to treat additional glomerular complement disorders and monogenic disorders.
Our aim is to leverage the versatility of our platform to develop therapies for additional glomerular diseases with unmet medical need.
While our current early-stage candidates are focused on rare monogenetic disorders, including NPHS2 driven steroid resistant nephrotic syndrome and focal segmental glomerulosclerosis, our platform also offers the potential to target other complement-driven glomerular diseases and more common indications such as diabetic kidney disease.
Publications