Platform
Our proprietary, category defining GlomThera™ platform is the only platform designed to deliver therapeutics, which either replace or augment our gene of interest, 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 well-established techniques.
This enables dosing more than 10-fold lower than systemically administered AAV gene therapies, reducing systemic exposure while optimising the potential therapeutic window.
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 cells of interest = durable efficacy
Effective transduction with right capsid
High Yield Manufacturing.
Suspension culture
Translatable through to commercial
Local Delivery.
Leverages established, routine procedures and devices
Direct uptake into the kidney
Low Dose.
<10% of systemic gene therapy
Minimal off-target exposure
Pipeline
Pipeline.
Purespring’s multi-asset pipeline is focused on renal indications affecting large and underserved patient populations with significant unmet need.
Our lead programmes span both monogenic and non-monogenic disease, targeting IgA nephropathy (IgAN) and Alport Syndrome, respectively. PS-002, which is in Phase I/II clinical studies, aims to evaluate a differentiated approach for patients with durable efficacy and an improved safety profile. PS-003 is rapidly 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.
Our aim is to leverage the versatility and targeted approach of our GlomtheraTM platform to develop therapies for additional glomerular diseases with unmet medical need.
While our current early-stage candidates are focused on rare monogenic disorders, including genetic FSGS driven by NPHS2, our platform also offers the potential to target more prevalent indications which drive ESRD.
Publications