Helex is at the forefront of developing innovative gene editing-based therapeutics specifically targeting genetic kidney diseases, with a mission to address the significant unmet medical needs of over 800 million individuals worldwide suffering from chronic kidney disease. The company employs advanced technologies such as its proprietary EPIC-Cure™ drug design platform, which utilizes 3D genome data to enhance the precision and safety of gene editing. This platform allows for the design of targeted guide-RNAs that minimize off-target effects, ensuring a more reliable therapeutic outcome. Additionally, Helex's approach incorporates comprehensive safety assessments that evaluate both short-term and long-term impacts of genome editing on cellular function.

To facilitate the effective delivery of these genetic medicines, Helex has developed a proprietary lipid nanoparticle technology that enables targeted delivery to the kidneys. This non-viral delivery system is designed to reduce immunogenicity and avoid the risks associated with viral vectors, such as insertional mutagenesis. By optimizing the parameters of these lipid nanoparticles, Helex aims to achieve kidney tropism, ensuring that therapeutic payloads are directed specifically to the affected organs and cells. Through the combination of these cutting-edge technologies, Helex is poised to revolutionize the treatment landscape for genetic kidney diseases, significantly improving the quality of life for patients.

United States

Helex is taking the promise of non-viral targeted delivery and genome editing to the kidney, developing first-in-class genetic medicines for renal indications. Helex combines its proprietary kidney tropic lipid nanoparticle delivery system with the Epic-Cure(TM) 3D genome-based drug designer platform to create a robust renal franchise of precise and scalable therapeutics. Our lead asset is a mutation-agnostic, potential one-and-done therapy for Autosomal Dominant Polycystic Kidney Disease that impacts over 4 million people worldwide.

Helex

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