SCIENCE
[/vc_column_text][vc_column_text css=”” animation=”animation left-to-right”]At the heart of our approach is our podocyte-specific, gene therapy viral vector (capsid), our targeted genetic elements (kidney-specific promoters), our method of administration, and our in-house panel of analytics to ensure that we manufacture the best product.
[/vc_column_text][thb_button_text animation=”animation bottom-to-top” link=”url:%23INTRO|title:LEARN%20MORE|target:_blank”][vc_empty_space mobile_height=”10vh” height=”20vh”][/vc_column_inner][/vc_row_inner][/vc_column][/vc_row][vc_row thb_full_width=”true” thb_row_padding=”true” thb_column_padding=”true” content_placement=”top” parallax=”content-moving” el_class=”align-center” css=”.vc_custom_1627894316726{margin-bottom: 6vh !important;padding-top: 5vh !important;padding-right: 5vw !important;padding-bottom: 10vh !important;padding-left: 5vw !important;background-color: #ffffff !important;background-position: center;background-repeat: no-repeat !important;background-size: contain !important;}” el_id=”INTRO”][vc_column offset=”vc_col-lg-10″][vc_row_inner content_placement=”top” css=”.vc_custom_1627894301175{padding-top: 12vh !important;padding-bottom: 6vh !important;}”][vc_column_inner width=”1/4″][thb_image retina=”retina_size” animation=”animation bottom-to-top” alignment=”aligncenter” image=”288″ img_size=”full”][/thb_image][vc_empty_space height=”1vh”][vc_column_text]Kidney
[/vc_column_text][vc_column_text css=””]The kidney is the organ that filters 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.
[/vc_column_text][vc_empty_space mobile_height=”7vh” height=””][/vc_column_inner][vc_column_inner width=”1/4″][thb_image retina=”retina_size” animation=”animation bottom-to-top” alignment=”aligncenter” image=”289″ img_size=”full”][/thb_image][vc_empty_space height=”1vh”][vc_column_text]Nephron
[/vc_column_text][vc_column_text css=””]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.
[/vc_column_text][vc_empty_space mobile_height=”7vh” height=””][/vc_column_inner][vc_column_inner width=”1/4″][thb_image retina=”retina_size” animation=”animation bottom-to-top” alignment=”aligncenter” image=”290″ img_size=”full”][/thb_image][vc_empty_space height=”1vh”][vc_column_text]Glomerulus
[/vc_column_text][vc_column_text css=””]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.
[/vc_column_text][vc_empty_space mobile_height=”7vh” height=””][/vc_column_inner][vc_column_inner width=”1/4″][thb_image retina=”retina_size” animation=”animation bottom-to-top” alignment=”aligncenter” image=”1070″ img_size=”full”][/thb_image][vc_empty_space height=”1vh”][vc_column_text]Podocyte
[/vc_column_text][vc_column_text css=””]The podocyte is the key cell type responsible for the filtration of the blood at a cellular level. The podocytes are like the fingers of a hand, they wrap around blood vessels like interlocking fingers.
[/vc_column_text][vc_empty_space mobile_height=”7vh” height=””][/vc_column_inner][/vc_row_inner][vc_row_inner][vc_column_inner width=”1/2″][vc_empty_space height=”5vh”]
PODOCYTES
[/vc_column_text][vc_empty_space height=”5vh”][vc_column_text css=”” animation=”animation left-to-right”]The podocyte – the key to kidney health.
[/vc_column_text][vc_empty_space height=”5vh”][vc_column_text css=”” animation=”animation bottom-to-top”]The podocyte is a highly 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.
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 called foot processes or 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).
[/vc_column_text][/vc_column_inner][/vc_row_inner][/vc_column][/vc_row][vc_row thb_full_width=”true” thb_row_padding=”true” thb_column_padding=”true” full_height=”yes” content_placement=”top” parallax=”content-moving” el_class=”align-center” css=”.vc_custom_1727171882984{border-top-width: 20px !important;border-right-width: 20px !important;border-bottom-width: 20px !important;border-left-width: 20px !important;padding-top: 10vh !important;padding-right: 5vw !important;padding-bottom: 10vh !important;padding-left: 5vw !important;background-color: #e6f2f3 !important;background-position: center;background-repeat: no-repeat !important;background-size: contain !important;border-left-style: solid !important;border-right-style: solid !important;border-top-style: solid !important;border-bottom-style: solid !important;border-radius: 35px !important;border-color: #ffffff !important;}” el_id=”PIPELINE”][vc_column offset=”vc_col-lg-9″][vc_empty_space height=”1vh”][thb_image retina=”retina_size” animation=”animation bottom-to-top” image=”140″ img_size=”150×150″][/thb_image][vc_empty_space height=”3vh”][vc_column_text css=”” animation=”animation bottom-to-top”]OUR STRONG PIPELINE
[/vc_column_text][vc_row_inner thb_max_width=”” content_placement=”top”][vc_column_inner][vc_column_text css=”” animation=”animation bottom-to-top”]Our goal is to bring about a step change in gene therapy development.
[/vc_column_text][vc_empty_space height=”5vh”][/vc_column_inner][/vc_row_inner][vc_row_inner thb_max_width=”” content_placement=”top”][vc_column_inner width=”1/2″][vc_column_text css=”” animation=”animation bottom-to-top”]Our growing pipeline has programmes in development targeting both rare monogenic diseases and more prevalent non-genetic diseases. Both types of diseases can lead to end-stage renal disease, with devastating consequences to patients. The lead program PS-002 is focused on complement driven glomerular diseases and will initially be developed for IgA nephropathy.
[/vc_column_text][/vc_column_inner][vc_column_inner width=”1/2″][vc_column_text css=”” animation=”animation bottom-to-top”]Our flexible platform allows us to leverage our capability to develop therapies for high unmet need glomerular diseases. Examples of such diseases are monogenic podocyte diseases (e.g. NPHS2 driven steroid resistant nephrotic syndrome and focal segmental glomerulosclerosis), glomerular basal membrane disorders and diabetic kidney disease.
[/vc_column_text][/vc_column_inner][/vc_row_inner][vc_empty_space height=”5vh”][thb_image retina=”retina_size” animation=”animation bottom-to-top” alignment=”aligncenter” image=”1424″ img_size=”full”][/thb_image][vc_empty_space height=”5vh”][thb_button_text animation=”animation bottom-to-top” link=”url:%23PUBLICATIONS|title:PUBLICATIONS|target:_blank”][vc_empty_space mobile_height=”10vh” height=””][/vc_column][/vc_row][vc_row thb_full_width=”true” thb_row_padding=”true” thb_column_padding=”true” full_height=”yes” content_placement=”top” parallax=”content-moving” bg_gradient_direction=”-90″ el_class=”align-center” css=”.vc_custom_1727171974627{border-top-width: 20px !important;border-right-width: 20px !important;border-bottom-width: 20px !important;border-left-width: 20px !important;padding-top: 6vh !important;padding-bottom: 6vh !important;background-color: #ffffff !important;background-position: center;background-repeat: no-repeat !important;background-size: contain !important;border-left-style: solid !important;border-right-style: solid !important;border-top-style: solid !important;border-bottom-style: solid !important;border-radius: 35px !important;border-color: #ffffff !important;}” el_id=”PUBLICATIONS”][vc_column el_class=”align-center”][vc_row_inner thb_max_width=”” content_placement=”top” css=”.vc_custom_1626702159621{padding-right: 5vw !important;padding-left: 5vw !important;}”][vc_column_inner width=”1/2″][thb_image retina=”retina_size” animation=”animation bottom-to-top” image=”141″ img_size=”100×100″][/thb_image][vc_empty_space height=”3vh”][vc_column_text css=”” animation=”animation bottom-to-top”]PUBLICATIONS/PRESENTATIONS
[/vc_column_text][vc_empty_space height=”3vh”][vc_column_text css=”” animation=”animation bottom-to-top”]Sharing our scientific results to advance innovation in the field.
[/vc_column_text][vc_empty_space height=”3vh”][vc_column_text css=”.vc_custom_1727172309627{padding-right: 10vw !important;}” animation=”animation bottom-to-top”]We are committed to transparency and sharing our scientific results with the public to advance innovation in the field.
[/vc_column_text][vc_column_text css=”.vc_custom_1727172319946{padding-right: 6vw !important;}” animation=”animation bottom-to-top”]Recent publications and/or presentations from our team include:
[/vc_column_text][vc_column_text css=”.vc_custom_1725465272982{padding-right: 6vw !important;}” animation=”animation bottom-to-top”]Wen Y. Ding et al., Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome. Sci. Transl. Med. 15, eabc8226 (2023). DOI:10.1126/scitranslmed.abc8226
[/vc_column_text][vc_empty_space height=”5vh”][/vc_column_inner][vc_column_inner width=”1/2″]PLATFORM
[/vc_column_text][vc_empty_space height=”2vh”][vc_column_text animation=”animation bottom-to-top”]We are powered by the first gene therapy platform specifically targeting kidney diseases.
[/vc_column_text][vc_empty_space height=”2vh”][vc_column_text animation=”animation bottom-to-top”]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.
[/vc_column_text][/vc_column_inner][/vc_row_inner][vc_row_inner content_placement=”top” css=”.vc_custom_1627396511961{padding-top: 6vh !important;}”][vc_column_inner width=”1/3″][thb_image retina=”retina_size” animation=”animation fade-in” alignment=”aligncenter” image=”415″ img_size=”300×300″][/thb_image][vc_empty_space height=”1vh”][vc_column_text]Delivery Approach
[/vc_column_text][vc_column_text]Our unique delivery approach enables us to target the kidney locally, thus reducing vector dose requirements and avoiding systemic distribution.
[/vc_column_text][vc_empty_space mobile_height=”7vh” height=””][/vc_column_inner][vc_column_inner width=”1/3″][thb_image retina=”retina_size” animation=”animation top-to-bottom” alignment=”aligncenter” image=”413″ img_size=”300×300″][/thb_image][vc_empty_space height=”1vh”][vc_column_text]AAV Vector Capsid & Gene Expression Promoter
[/vc_column_text][vc_column_text]Leveraging our capsids and promoters across programs may enable us to shorten development timelines, reduce uncertainty and minimise cost across development and manufacturing.
[/vc_column_text][vc_empty_space mobile_height=”7vh” height=””][/vc_column_inner][vc_column_inner width=”1/3″][thb_image retina=”retina_size” animation=”animation bottom-to-top” alignment=”aligncenter” image=”517″ img_size=”300×300″][/thb_image][vc_empty_space height=”1vh”][vc_column_text]CMC Processes & Analytics
[/vc_column_text][vc_column_text]Our robust CMC processes and proprietary set of CMC analytics are developed in house. These will offer multiple economic, IP and regulatory benefits.
[/vc_column_text][vc_empty_space mobile_height=”7vh” height=””][/vc_column_inner][/vc_row_inner][vc_row_inner][vc_column_inner el_class=”text-center” css=”.vc_custom_1627393022259{padding-right: 3vw !important;padding-left: 3vw !important;}”][vc_empty_space height=”5vh”][thb_button_text animation=”animation bottom-to-top” link=”url:%23LINKS|title:EXPLORE|target:_blank”][/vc_column_inner][/vc_row_inner][/vc_column][/vc_row][vc_row thb_full_width=”true” thb_row_padding=”true” thb_column_padding=”true” el_id=”LINKS” css=”.vc_custom_1627473212244{margin-bottom: 3vh !important;border-top-width: 25px !important;border-right-width: 25px !important;border-bottom-width: 25px !important;border-left-width: 25px !important;padding-right: 1vw !important;padding-left: 1vw !important;border-left-color: #ffffff !important;border-left-style: solid !important;border-right-color: #ffffff !important;border-right-style: solid !important;border-top-color: #ffffff !important;border-top-style: solid !important;border-bottom-color: #ffffff !important;border-bottom-style: solid !important;border-radius: 35px !important;}”][vc_column][thb_content_carousel thb_columns=”medium-3″ thb_pagination=”” autoplay=”true” thb_margins=”medium-padding”][thb_fancybox height=”50vh” animation=”animation bottom-to-top-3d” thb_text_color=”fancy-light” image=”994″ bg_gradient1=”#82bec3″ bg_gradient2=”#82bec3″ border_radius=”15px” link=”url:https%3A%2F%2Fpurespringtx.com%2Fpatients%2F|title:Patients”]