The podocyte is the specialised kidney cell type implicated in many kidney diseases. We are the first company to treat kidney diseases by directly targeting the podocyte with AAV gene therapy.
PODOCYTES
The podocyte is at the core of kidney function and at the heart of our scientific approach.
Podocytes are a key kidney cell type, whose function is impaired in most kidney diseases.
The kidneys perform an essential job filtering the blood. Podocytes make up one of the most critical components of that filtering apparatus. The podocytes have long cellular extensions like foot processes (called pedicels), after which the cells are named.
The pedicels wrap around the blood vessels and leave little gaps called slits between them. Blood is filtered through these gaps, each known as a filtration slit or slit diaphragm (in other words, analogous to the spaces between the teeth of a zipper).
Kidney
The kidney is the organ that is filtering the blood. Blood to be filtered comes via the renal artery (in red), blood filtered leaves in the renal vein (in blue), and the waste products are eliminated in the urine.
Nephron
Each kidney is divided into individual functional units called nephrons. If the kidney is like a factory, the nephrons are the individual workers, and there are close to one million nephrons per kidney.
Glomerulus
The glomerulus is a key element of the nephron where important filtration mechanisms happen. The glomerulus is like the hand of the worker, deciding what is filtered out and what is kept in the blood.
Podocyte
The podocyte is one of the key cell types responsible for the filtration of the blood at a cellular level. The podocytes are like the fingers of a hand, and they wrap around blood vessels like fingers interlocking.
PODOCYTES
We only have a finite number of podocytes in our kidneys: unlike other human cells such as liver cells or skin cells, podocytes do not regenerate over our lifetime. Injuries to the podocytes lead to issues in the filtration barrier, reducing the kidney’s filtration capacity, causing kidney diseases.
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At Purespring, our unique approach targets the podocyte, allowing us to directly treat a significant proportion of kidney diseases.
This is the first time that treatments for kidney diseases have been created that are solely focused on targeting the podocyte. Our targeted approach has the potential to radically change the treatment of kidney diseases. We know the podocyte like no one else.
With the explosion in understanding of kidney biology over the last 20 years led by our scientific founder, Professor Moin Saleem, the stage is now set for a revolution in the treatment of kidney diseases.
GENE THERAPY
What is Gene Therapy?
Purespring’s therapies are based on a novel technology called gene therapy.
Many diseases are caused by mutations in specific genes, leading them to lose their appropriate function. The gene therapy will restore normal function by providing the healthy version of a gene.
Gene therapies are delivered with an innocuous virus called AAV, for adeno-associated virus. This virus contains the gene and other elements to make sure that the gene is expressed in the correct parts of the body.
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PLATFORM
We are powered by the first gene therapy platform specifically targeting kidney diseases.
Our platform is built on the need to target the podocyte for maximal efficacy, and will be our key to success in delivering treatments for patients with kidney diseases.
Several pioneering components of our platform enable this podocyte-specific targeting.
Delivery Approach
Our unique delivery approach enables us to target the kidney locally, thus reducing vector dose requirements and avoiding systemic distribution.
AAV Vector Capsid & Gene Expression Promoter
Leveraging our capsids and promoters across programs may enable us to shorten development timelines, reduce uncertainty and minimise cost across development and manufacturing.
CMC Processes & Analytics
Our robust CMC processes and proprietary set of CMC analytics are developed in house. These will offer multiple economic, IP and regulatory benefits.
