Annogen and CIMA Universidad de Navarra Forge Research Collaboration to Develop Kidney Cell-Specific Promoters for Gene Therapies

Annogen, a leader in making gene therapies safer and more effective through advanced gene expression engineering, today announced a new research collaboration with the Cima Universidad de Navarra, a leading Spanish biomedical research center. The partnership, led by Dr. Gloria González-Aseguinolaza and Dr. Rafael Aldabe from CIMA, will leverage Annogen’s proprietary SuRE™ platform for in vivo screening using CIMA’s proprietary in vivo cell-specific kidney delivery system to identify and characterize highly specific promoters for kidney-targeted gene therapy.

This collaboration aims to address a critical hurdle in the development of targeted gene therapies for kidney diseases: the need for precise and cell-type-specific gene expression. Current gene therapy approaches often struggle with off-target delivery and expression, which can lead to reduced efficacy and unwanted side effects. By identifying promoters that are active only in specific kidney cell populations, this research seeks to pave the way for safer and more effective treatments for a range of renal conditions. With Chronic Kidney Disease (CKD) representing a significant public health challenge, and many cases resulting from monogenic defects that current symptomatic treatments cannot cure, gene therapy that can boast of tightly regulated cell-specific expression offers a promising approach for a safe, long-lasting therapeutic solution.

"We are incredibly excited to partner with Dr. González-Aseguinolaza and Dr. Aldabe Arregui, whose deep expertise in kidney biology and gene therapy will be invaluable to this project," said Dr. Joris van Arensbergen, CEO and founder of Annogen. "The kidney is a complex organ with diverse cell types, and achieving cell-specific gene expression will be a game changer for effective therapeutic interventions. Our SuRE™ platform, with its ability to conduct in vivo screening, is uniquely suited to uncover the precise regulatory elements needed for this challenge."

The research will focus on identifying promoters that can drive gene expression exclusively within the specific renal cell types that are required for successful therapeutic outcomes following gene therapy. The in vivo SuRE™ screening approach will allow for the evaluation of millions of potential promoter sequences directly within a living organism, ensuring that the identified elements are functional and specific within their native physiological context.

"We have a great deal of optimism in launching this collaborative effort that will benefit from the inherent expertise and experience of both institutions in the development of innovative therapeutic modalities" commented Dr. Gloria González-Aseguinolaza, Principal Investigator at CIMA. "Our overarching goal is to rapidly advance the process for identifying and validating safer and more effective gene therapies to provide some window of hope for the patients and families who suffer from these diseases."

Dr. Rafael Aldabe, also a Principal Investigator at CIMA, added, "Gene therapy targeting the kidney is an area of research primed for astounding advances. The primary obstacle to overcome, as with many gene therapies, is in achieving appropriate delivery and expression. We foresee the fruit of this collaboration providing a key milestone in this fight that can be utilized by us and others striving to cure hereditary kidney diseases via gene therapy."

The collaboration underscores both Annogen’s and CIMA’s commitment to advancing innovative solutions for unmet medical needs and highlights the power of combining advanced genomic screening technologies with specialized organ biology expertise.