Study summary
Problem
IgA nephropathy (IgAN) is a chronic kidney disease caused by the build up of
abnormal IgA antibodies in the kidney filtration systems (glomeruli) causing
inflammation. The human body has a powerful system called complement system that works to protect against infectons. In IgAN, this complement system becomes overactivated and fuels inflammation in the kidneys that further accelerates the damage. There are currently some new medicines being developed that block overactivation of this complement system, and these have shown benefit in reducing damage in IgAN. However these new medicines have side effects as they block the complement system in the whole body and not just in the kidneys, increasing the risk of serious systemic infections .
Methods
Purespring is developing a special way, using gene therapy methods that aim to downregulate complement system in the kidneys. The product is called PS-002 and it delivers a gene to the kidney cells that will enable these cells to become biofactories producing a powerful anti-inflammatory substance called Complement Factor I (CFI). The gene is delivered directly to the kidneys using a harmless viral vector via a one-time infusion. This will be the very first time PS-002 is given to the humans. Participants will be closely monitored for safety, immune response, and improvements in kidney function over 48 weeks, with an optional long-term follow-up.
Relevance of Requested Recall of Patient Volunteers
The NIHR BioResource enables recall of IgAN patients who have consented to be contacted for research and are therefore open to consider new therapies. This targeted recruitment is vital for identifying potentially suitable participants quickly and efficiently. Considering the novelty of gene therapy approach for IgAN it will help to ensure that the individuals most likely to benefit from this new approach are included in this first in human study and contribute to the valuable research.
Benefit to Patients and Impact
If successful, PS-002 could offer the first gene therapy based treatment that alters the IgAN disease progression, potentially reducing inflammation, preserving kidney function, and improving quality of life. With a single administration, a gene therapy would provide a long-lasting, potentially permanent therapeutic effect. The study will also generate valuable data on gene therapy safety and delivery to the kidneys.
Data Use and Disclosure Risks
All data and samples will be coded to protect participant identity. Only authorised personnel can link data to individuals. Any remaining samples will be securely destroyed or used for further approved research. Disclosure risks are minimal and managed through strict data protection protocols.