Preclinical Studies Highlight Novel Gene Therapy for IgA Nephropathy Treatment

Complement Activation and the Role of Podocytes
IgAN arises from dysfunctions in the immune system’s complement pathway, contributing to kidney damage. PS-002 targets podocytes, specialized kidney cells that play a key role in maintaining the glomerular filtration barrier. Researchers found that modulating complement activation in podocytes effectively mitigated symptoms and structural damage associated with the disease in animal models.

Promising Results in Preclinical Trials
In preclinical mouse models, PS-002 demonstrated reduced kidney dysfunction, decreased complement deposition, and improved kidney structure, including reduced scarring. Testing in pigs further validated these findings, with prolonged and elevated gene expression observed in kidney tissues and no reported safety concerns.

Principal Scientist Ambra Cappelletto of 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.”

Advancing Towards Clinical Trials
The success of these trials supports the progression of PS-002 towards Phase I/II clinical trials, marking a significant milestone in the development of gene therapies for kidney diseases. Purespring CEO Julian Hanak emphasized the urgency of addressing chronic kidney disease, which affects 840 million people globally:
*”The data presented at ASN Kidney Week 2024 demonstrate the potential of our gene therapy platform to deliver working copies of genes with high efficiency and specificity directly to the podocyte. We hope these promising results will translate into meaningful clinical benefits for patients.”*

Innovative AAV Gene Therapy Platform
Purespring’s proprietary adeno-associated virus (AAV) platform exemplifies the potential for gene therapies to target podocytes directly, a novel approach for treating kidney diseases. This breakthrough comes after the company showcased related research at the 61st European Renal Association Congress earlier this year.

Future Prospects
With recent Series B financing of £80 million ($105 million), Purespring is preparing to launch Phase I/II clinical trials for PS-002. As IgAN remains without a cure, and about one-third of patients face kidney failure within five years, the development of PS-002 offers new hope for patients seeking alternatives to dialysis and transplantation.

Gene therapy is revolutionizing the treatment landscape for chronic conditions, and PS-002 stands at the forefront of this progress, with the potential to transform care for IgA nephropathy and other kidney diseases.

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