Helex
Helex is an early preclinical biotechnology company (US and India) developing genomic medicines for inherited kidney diseases. The company focuses on kidney-targeted non-viral lipid nanoparticle delivery and an AI/bioinformatics-driven drug design platform that integrates 3D genome and epigenetic data to design and evaluate genome editing and other nucleic-acid therapeutics. Helex operates preclinical programs for inherited kidney indications including ADPKD and seeks strategic partnerships and licensing opportunities.
Industries
Nr. of Employees
small (1-50)
Products
Preclinical gene editing program for ADPKD (PKD1-driven)
Lead preclinical program applying non-viral, kidney-targeted lipid nanoparticle delivery of genome editing therapeutics aimed at autosomal dominant polycystic kidney disease due to PKD1 mutations.
Preclinical gene editing program for ADPKD (PKD2-driven)
Preclinical program targeting ADPKD cases associated with PKD2 mutations.
Preclinical program for autosomal recessive polycystic kidney disease (ARPKD)
Early discovery and preclinical work addressing ARPKD using programmable delivery and computational design technologies.
Preclinical programs for tubulointerstitial kidney disease
Early-stage therapeutic discovery applying the company's delivery and design platforms to tubulointerstitial kidney disease indications.
Targeted Drug Delivery Technology
Proprietary lipid nanoparticle technology designed for in vivo kidney delivery, directing therapeutic payloads to specific organs and cells.
Helex Platform
A scalable and programmable platform built on proprietary bioinformatics and intelligent AI-powered systems to identify unique sequences and enable the design of gene editing apparatus considering efficiency and long-term safety.
Preclinical gene editing program for ADPKD (PKD1-driven)
Lead preclinical program applying non-viral, kidney-targeted lipid nanoparticle delivery of genome editing therapeutics aimed at autosomal dominant polycystic kidney disease due to PKD1 mutations.
Preclinical gene editing program for ADPKD (PKD2-driven)
Preclinical program targeting ADPKD cases associated with PKD2 mutations.
Preclinical program for autosomal recessive polycystic kidney disease (ARPKD)
Early discovery and preclinical work addressing ARPKD using programmable delivery and computational design technologies.
Preclinical programs for tubulointerstitial kidney disease
Early-stage therapeutic discovery applying the company's delivery and design platforms to tubulointerstitial kidney disease indications.
Targeted Drug Delivery Technology
Proprietary lipid nanoparticle technology designed for in vivo kidney delivery, directing therapeutic payloads to specific organs and cells.
Helex Platform
A scalable and programmable platform built on proprietary bioinformatics and intelligent AI-powered systems to identify unique sequences and enable the design of gene editing apparatus considering efficiency and long-term safety.
Services
Discovery through IND-enabling preclinical activities for genome editing programs targeting kidney diseases.
Strategic partnerships to apply kidney-targeted delivery technology and AI-based design tools to additional renal indications or external programs.
Discovery through IND-enabling preclinical activities for genome editing programs targeting kidney diseases.
Strategic partnerships to apply kidney-targeted delivery technology and AI-based design tools to additional renal indications or external programs.
Expertise Areas
- Lipid nanoparticle drug delivery
- Genome editing therapeutics
- 3D genome and epigenetics-informed therapeutic design
- Guide RNA design and off-target safety assessment
Key Technologies
- Lipid nanoparticle delivery
- Ionizable lipid chemistry
- CRISPR/Cas genome editing
- Guide RNA design algorithms
News & Updates
Selected to present a poster describing a lipid nanoparticle-driven gene editing therapeutic addressing PKD1-driven ADPKD at ASN Kidney Week, Houston, November 6–9, 2025.
Planned presentation on progress of a gene editing-based therapeutic for PKD1-driven ADPKD at the ESGCT Annual Meeting in Seville, Spain (October 8, 2025).
Oral presentation scheduled at ASGCT 2025 describing a lipid nanoparticle-mediated CRISPR-based therapy for ADPKD.
Company CEO selected to present a symposium talk titled 'Harnessing Cell and Gene Therapies: New Frontiers in Kidney Treatment' (scheduled May 16, 2025).
Received Outstanding Poster Award at ASGCT 2024 for an abstract on a therapeutic intervention for autosomal dominant polycystic kidney disease.
Presented three posters at ASGCT 2024 in Baltimore covering CRISPR-Cas9 therapeutic approaches for ADPKD and guide RNA design and safety assessment capabilities.
Selected to present a poster describing a lipid nanoparticle-driven gene editing therapeutic addressing PKD1-driven ADPKD at ASN Kidney Week, Houston, November 6–9, 2025.
Planned presentation on progress of a gene editing-based therapeutic for PKD1-driven ADPKD at the ESGCT Annual Meeting in Seville, Spain (October 8, 2025).
Oral presentation scheduled at ASGCT 2025 describing a lipid nanoparticle-mediated CRISPR-based therapy for ADPKD.
Company CEO selected to present a symposium talk titled 'Harnessing Cell and Gene Therapies: New Frontiers in Kidney Treatment' (scheduled May 16, 2025).
Received Outstanding Poster Award at ASGCT 2024 for an abstract on a therapeutic intervention for autosomal dominant polycystic kidney disease.
Presented three posters at ASGCT 2024 in Baltimore covering CRISPR-Cas9 therapeutic approaches for ADPKD and guide RNA design and safety assessment capabilities.