Helex
Helex is a biotechnology company dedicated to revolutionizing genomic medicines, especially for high-burden kidney diseases. They develop gene editing-based therapeutics using proprietary platforms like EPIC-Cure™ and lipid nanoparticle technology. Their mission is to serve patients with high unmet medical needs through innovative, safe, and targeted gene therapies. They focus on addressing large unmet needs in chronic kidney disease, creating first-in-class medicines, and leveraging advanced genome data and delivery systems to improve safety and efficacy.
Industries
Nr. of Employees
small (1-50)
Products
Preclinical program: ADPKD (PKD1)
Preclinical gene editing therapeutic program targeting Autosomal Dominant Polycystic Kidney Disease due to PKD1 mutations.
Preclinical program: ADPKD (PKD2)
Preclinical gene editing therapeutic program targeting Autosomal Dominant Polycystic Kidney Disease due to PKD2 mutations.
Preclinical program: ARPKD
Preclinical development of gene editing approaches for Autosomal Recessive Polycystic Kidney Disease.
Preclinical program: Tubulointerstitial kidney disease
Preclinical research program applying gene editing and delivery platforms to tubulointerstitial kidney diseases.
Preclinical program: ADPKD (PKD1)
Preclinical gene editing therapeutic program targeting Autosomal Dominant Polycystic Kidney Disease due to PKD1 mutations.
Preclinical program: ADPKD (PKD2)
Preclinical gene editing therapeutic program targeting Autosomal Dominant Polycystic Kidney Disease due to PKD2 mutations.
Preclinical program: ARPKD
Preclinical development of gene editing approaches for Autosomal Recessive Polycystic Kidney Disease.
Preclinical program: Tubulointerstitial kidney disease
Preclinical research program applying gene editing and delivery platforms to tubulointerstitial kidney diseases.
Services
3D genome-based guide-RNA design services
Design and safety analysis of guide RNAs using cell- and disease-specific 3D genome data and bioinformatics/ML to optimize specificity and estimate long-term impact.
Kidney-targeted delivery platform collaborations
Collaborative development or licensing of kidney-targeted lipid nanoparticle formulations and delivery strategies for genetic payloads.
Preclinical development and translational R&D
In-house preclinical study execution, proof-of-concept testing and translational R&D for genetic kidney disease programs.
Strategic partnership and licensing engagement
Business development and collaboration for platform expansion, in-licensing of editors and joint projects to apply delivery/design platforms to new indications.
3D genome-based guide-RNA design services
Design and safety analysis of guide RNAs using cell- and disease-specific 3D genome data and bioinformatics/ML to optimize specificity and estimate long-term impact.
Kidney-targeted delivery platform collaborations
Collaborative development or licensing of kidney-targeted lipid nanoparticle formulations and delivery strategies for genetic payloads.
Preclinical development and translational R&D
In-house preclinical study execution, proof-of-concept testing and translational R&D for genetic kidney disease programs.
Strategic partnership and licensing engagement
Business development and collaboration for platform expansion, in-licensing of editors and joint projects to apply delivery/design platforms to new indications.
Expertise Areas
- Gene editing therapeutic design
- Kidney-targeted delivery
- 3D genome and epigenetics analysis
- Guide-RNA design and off-target safety assessment
Key Technologies
- CRISPR and next-generation genome editors
- Lipid nanoparticles (LNPs)
- Ionizable lipids
- PEGylation and nanoparticle surface engineering