podocin

EMA designation provides orphan status to medicines being developed for rare conditions that are intended for the diagnosis, prevention or treatment of diseases affecting fewer than 5 in 10,000 people in the European UnionPurespring has previously demonstrated the potential of PS-002, the Company’s lead programme, as an important novel targeted treatment for primary IgANPS-002 is progressing towards a Phase I/II clinical trial, supported by a strong preclinical data package London – 28 April 2025 - Purespring Therapeutics, a pioneering company focused on transforming the treatment of kidney diseases, today announces that the European Medicines Agency (EMA) has granted orphan drug designation to PS-002, Purespring’s lead programme, in IgA nephropathy (IgAN). Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion adopted by the EMA Committee for Orphan Medicinal Products. Orphan designation provides incentives to developers of medicines for limited patient populations, including market exclusivity, protocol assistance (guidance on study design and scientific evaluation) and regulatory fee reductions. IgAN is a common, chronic autoimmune kidney disease that primarily affects young adults. In patients with the disease, a protein called immunoglobulin A (IgA) becomes trapped in the filters of the kidney, known as glomeruli, causing inflammation, damage and scarring to the whole kidney. There are currently no approved therapeutic options to stop disease progression, nor a cure, and about one third of high risk IgAN patients will go on to lose kidney function within five years and require a kidney transplant or dialysis. Haseeb Ahmad, Purespring’s Chief Executive Officer, commented: “Orphan drug designation will significantly support Purespring’s goal of progressing PS-002 through clinical development and, more broadly, with bringing solutions for kidney disease indications to patients. We’re delighted that the EMA has recognised the promise of this potential breakthrough medicine and we look forward to working with them on the development pathway. “By developing a novel approach to treat IgAN, we believe we can offer hope to patients across Europe and globally who currently have limited therapeutic options, furthering our mission to transform the lives of people suffering from kidney diseases.” Supported by an oversubscribed £80 million / $105 million Series B financing, which completed in October 2024, Purespring is progressing PS-002 towards a Phase I/II clinical trial for IgAN, with additional gene therapy programmes in development for the treatment of both rare and common kidney diseases. Purespring is the first company to successfully treat kidney disease models by directly targeting the podocyte, a specialised cell implicated in approximately 60% of renal disease, through its proprietary adeno-associated viral (AAV) gene therapy platform. As detailed in a presentation at the American Society of Nephrology (ASN) Kidney Week 2024, Purespring has previously demonstrated the potential of PS-002 as an important novel modality to treat IgAN. For further information, contact: Purespring: Peter Mulcahycontact@purespringtx.com+44 (0)20 3855 6324LinkedIn ICR Healthcare Amber Fennell, Jessica Hodgsonpurespring@icrhealthcare.com Notes to Editors About Purespring Purespring is developing therapies to halt or prevent kidney disease, one of humankind’s most poorly treated disease areas. Founded on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, Purespring is the first company to successfully treat kidney disease by targeting the podocyte, a specialised cell that is implicated in the majority of renal disease. Purespring’s platform approach enables streamlined gene therapy development for both acquired and genetic renal diseases, offering the potential to halt, reverse and even cure both rare and common kidney diseases. The Company currently has a pipeline of programmes in development including the lead asset for treatment of IgA Nephropathy (IgAN) and other complement mediated kidney disease. The Company also has programmes for disease caused by mutations in the gene NPHS2, as well as other monogenic glomerular kidney diseases. Based in London, the Purespring team combines world-leading expertise in podocyte biology and kidney disease with a wealth of experience in gene therapies, anchored in a culture of diversity, creativity and delivery. Purespring is backed by leading biotech investors, including Syncona Limited, Sofinnova Partners, Gilde Healthcare, Forbion, and the British Business Bank and has raised £115m ($149m) to date. For more information please visit: purespringtx.com and follow us on LinkedIn.

Results from the study underpin the potential of Purespring’s lead AAV gene therapy programme, PS-002, as an important novel modality to treat IgAN Data support progression towards a Phase I/II clinical trial London – 28 October 2024 - Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, presented preclinical data at the American Society of Nephrology (ASN) Kidney Week 2024 showing that transgenes can be effectively targeted to podocytes to modulate complement activation and reduce signs of kidney disease in animal models of IgA nephropathy (IgAN), an autoimmune kidney disease. The presentation, “Podocyte gene therapy enables glomerular complement modulation for IgA Nephropathy (IgAN) treatment,” was presented on 26 October at the American Society of Nephrology (ASN) Kidney Week 2024, taking place in San Diego, USA from October 24–27. PS-002, uses a modified virus to treat podocytes, with the research demonstrating the administration of PS-002 in a mouse model of IgAN reduced signs of kidney dysfunction, lowered complement deposition and ameliorated kidney scarring and other structural characteristics of kidney disease. In pigs, treatment with PS-002 resulted in elevated and prolonged gene expression in kidney tissues, with no safety issues. Ambra Cappelletto, Principal Scientist at Purespring, commented: “Our data demonstrate that targeting podocytes to modulate complement activation is an effective therapeutic strategy, and PS-002 paves the way to become the first gene therapy in development for the treatment of IgA nephropathy. Purespring’s gene therapy platform, exemplified by PS-002, demonstrates therapeutic genetic material can be delivered with high efficiency to podocytes, opening up a new and highly differentiated modality with the potential to treat a broad range of kidney diseases.” Julian Hanak, Purespring’s Chief Executive Officer, added: “Chronic kidney disease is an area of huge unmet need, affecting around 840 million people globally, and we were thrilled to demonstrate the potential of our novel platform technology with the international nephrology community. The data presented at ASN Kidney Week 2024 demonstrate the potential of our gene therapy platform technology to deliver working copies of genes with high efficiency and specificity directly to the podocyte, and as we further progress the development of PS-002, we hope that these promising results will translate into clinically meaningful benefits for patients.” Purespring recently announced it had raised £80 million ($105 million) in a Series B financing to support the initiation of a Phase I/II clinical trial for IgAN. There is currently no cure for IgAN, and about one third of patients will go on to lose their kidney function within five years and require a kidney transplant or dialysis. Purespring is the first company to successfully treat kidney disease models by directly targeting the podocyte, a specialised cell implicated in approximately 60% of renal diseases, through its proprietary adeno-associated virus (AAV) gene therapy platform. The data presented at ASN follows earlier critical preclinical data presented by Purespring at the 61st European Renal Association (ERA) Congress in May establishing the potential of AAV gene therapy to deliver transgenes to the podocyte to replace defective genes or to modulate protein production. For further information, contact: Purespring: Julian Hanak, CEOcontact@purespringtx.com+44 (0)20 3855 6324LinkedIn ICR Healthcare Amber Fennell, Jessica Hodgsonpurespring@consilium-comms.com Notes to Editors About Purespring Purespring is developing gene therapies to halt or prevent kidney disease, one of humankind’s most poorly treated disease areas. Founded on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, Purespring is the first company to successfully treat kidney disease by targeting the podocyte, a specialised cell that is implicated in the majority of renal disease. Purespring’s platform approach enables streamlined gene therapy development for both monogenic and non-monogenic renal diseases, offering the potential to halt, reverse and even cure both rare and common kidney diseases. The Company currently has a pipeline of programmes in development including the lead asset for treatment of IgA Nephropathy (IgAN) and other complement mediated kidney disease. The Company also has programmes for diseases caused by mutations in the gene NPHS2, as well as other monogenic glomerular kidney diseases. Based in London, the Purespring team combines world-leading expertise in podocyte biology and kidney disease with a wealth of experience in gene therapies, anchored in a culture of diversity, creativity and delivery. Purespring is backed by leading biotech investors, including Syncona Limited, Sofinnova Partners, Gilde Healthcare, Forbion, and British Patient Capital and has raised £115M to date. For more information please visit: purespringtx.com and follow us on LinkedIn. Attachment Cross-sectional images of IgA nephropathy murine kidneys.

Purespring Therapeutics raises £80/$105 million in a Series B financing to transform the treatment of kidney diseases Oversubscribed financing led by Sofinnova Partners in collaboration with Gilde Healthcare, Forbion, British Patient Capital and founding investor Syncona Funds raised will support Purespring in advancing its AAV pipeline of gene therapy programmes to treat kidney disease, including its lead programme targeting IgA Nephropathy (IgAN) London – 9 October 2024 - Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, today announces it has raised £80/$105 million in a Series B financing. The oversubscribed round was led by Sofinnova Partners, in collaboration with a prominent syndicate including Gilde Healthcare, Forbion, British Patient Capital and founding investor Syncona Limited. Purespring is the first company to successfully treat kidney disease models by directly targeting the podocyte, a specialised cell implicated in approximately 60% of renal diseases, through its proprietary adeno-associated viral (AAV) gene therapy platform. The Company currently has a pipeline of programmes in development. This includes its lead programme PS-002, in development for IgA Nephropathy (IgAN), and other complement mediated kidney diseases, a programme targeting nephrotic syndrome, and an undisclosed glomerular kidney disease programme. Purespring’s platform technology allows working copies of genes to be delivered with high efficiency and specificity to podocytes, opening up a new and highly differentiated modality for disease modifying treatments of a broad range of kidney diseases. Proceeds will be used to support Purespring’s pipeline including the initiation of a Phase I/II clinical trial for IgAN, a common, chronic kidney disease primarily affecting young adults. About one third of IgAN patients will go on to lose their kidney function within five years and require a kidney transplant or dialysis. Julian Hanak, Purespring’s Chief Executive Officer, commented: “Nearly one tenth of the world’s population, around 840 million people, suffer from chronic kidney disease. For many of them, there are few options beyond dialysis and transplantation. Our novel treatment platform and deep understanding of kidney disease puts us in a position to stop, reverse and even cure kidney disease. The funds raised will allow us to bring our novel treatments to patients in the clinic. “I’m really pleased to be able to welcome such a high-quality investor syndicate to support Purespring on this journey. Having sophisticated, experienced and committed investors on board, who understand drug development and the urgency of bringing these much-needed therapies to patients will maximise the opportunities for the company and the patients we treat.” Maina Bhaman, Partner at Sofinnova Partners, added: “Purespring Therapeutics is pioneering the treatment of kidney disease. The team’s ambition and calibre epitomise Sofinnova’s mission of scientific and entrepreneurial excellence in delivering transformative treatments to patients. We are excited to support the Purespring team as they bring their groundbreaking innovation to the global stage.” Michael Kyriakides, Investment Partner at Syncona Investment Management and Board Director of Purespring Therapeutics, said: “Syncona founded Purespring with the vision of creating the world’s leading kidney gene therapy company, and we have been impressed with its progress. Today’s financing and the strong syndicate of investors that the Company has attracted broadens Purespring’s financial scale and clearly validates its technology, strategy and execution to date. We look forward to working alongside Purespring and its new syndicate as it prepares for its Phase I/II clinical trial.” The financing follows critical scientific milestones for Purespring. Earlier this year, Purespring presented preclinical data at the 61st European Renal Association (ERA) Congress establishing the potential of AAV gene therapy to deliver transgenes to the podocyte to replace defective genes or to modulate protein production, underpinning the potential of AAV as an important novel modality to treat a broad range of kidney disease. In 2023, a paper published in Science Translational Medicine demonstrated that Purespring had successfully used AAV gene therapy to transduce the podocyte in vivo for the first time – a major milestone in overcoming the hurdles which have previously limited development in this area. Purespring will present its IgAN data publicly for the first time at the American Society of Nephrology (ASN) Kidney Week congress on October 26 in San Diego (abstract #SA-OR63). For further information, contact: Purespring: Julian Hanak, CEOcontact@purespringtx.com+44 (0)20 3855 6324LinkedIn ICR Consilium Amber Fennell, Jessica Hodgsonpurespring@consilium-comms.com Notes to Editors About Purespring Purespring is developing gene therapies to halt or prevent kidney disease, one of humankind’s most poorly treated disease areas. Founded on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, Purespring is the first company to successfully treat kidney disease by targeting the podocyte, a specialised cell that is implicated in the majority of renal disease. Purespring’s platform approach enables streamlined gene therapy development for both monogenic and non-monogenic renal diseases, offering the potential to halt, reverse and even cure both rare and common kidney diseases. The Company currently has a pipeline of programmes in development including the lead asset for treatment of IgA Nephropathy (IgAN) and other complement mediated kidney disease. The Company also has programmes for diseases caused by mutations in the gene NPHS2, as well as other monogenic glomerular kidney diseases. Based in London, the Purespring team combines world-leading expertise in podocyte biology and kidney disease with a wealth of experience in gene therapies, anchored in a culture of diversity, creativity and delivery. Purespring is backed by leading biotech investors, including Syncona Limited, Sofinnova Partners, Gilde Healthcare, Forbion, and British Patient Capital and has raised £115M to date. For more information please visit: purespringtx.com and follow us on LinkedIn. About Syncona Syncona's purpose is to invest to extend and enhance human life. We do this by creating, building and scaling companies to deliver transformational treatments to patients in areas of high unmet need. We aim to build and maintain a diversified portfolio of 20-25 globally leading life science businesses, across development stage, modality and therapeutic area, for the benefit of all our stakeholders. We focus on developing treatments that deliver patient impact by working in close partnership with world-class academic founders and experienced management teams. Our balance sheet underpins our strategy, enabling us to take a long-term view as we look to improve the lives of patients with no or poor treatment options, build sustainable life science companies and deliver strong risk-adjusted returns to shareholders. About Sofinnova Partners Sofinnova Partners is a leading European venture capital firm in life sciences, specializing in healthcare and sustainability. Based in Paris, London and Milan, the firm brings together a team of professionals from all over the world with strong scientific, medical and business expertise. Sofinnova Partners is a hands-on company builder across the entire value chain of life sciences investments, from seed to later-stage. Founded in 1972, Sofinnova Partners is a deeply-established venture capital firm in Europe, with 50 years of experience backing over 500 companies and creating market leaders around the globe. Today, Sofinnova Partners has over €2.8 billion under management. For more information, please visit: www.sofinnovapartners.com.