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
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.
Collaborative development or licensing of kidney-targeted lipid nanoparticle formulations and delivery strategies for genetic payloads.
In-house preclinical study execution, proof-of-concept testing and translational R&D for genetic kidney disease programs.
Business development and collaboration for platform expansion, in-licensing of editors and joint projects to apply delivery/design platforms to new indications.
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.
Collaborative development or licensing of kidney-targeted lipid nanoparticle formulations and delivery strategies for genetic payloads.
In-house preclinical study execution, proof-of-concept testing and translational R&D for genetic kidney disease programs.
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
News & Updates
Helex to present a Lipid Nanoparticle-Mediated CRISPR-based Therapy at ASGCT 2025
Helex will present their lipid nanoparticle-mediated CRISPR therapy for ADPKD at the American Society of Gene & Cell Therapy conference in New Orleans, May 2025.
Helex awarded Aegis Graham Bell Award 2025 in Life Sciences
Helex received the prestigious award for innovation in life sciences, recognizing their advancements in gene editing therapies.
Helex's CEO Poulami Chaudhuri to present at Kidney Treatment Symposium in May 2025
Poulami Chaudhuri, PhD, will speak on new frontiers in kidney gene therapies at the ASGCT conference.
Helex's Chief Growth Officer Rohini Kalvakuntla interviewed by Cure magazine
An interview highlighting Helex's pioneering work in developing therapies for genetic kidney diseases.
Biotech startup Helex brings precision to gene editing
An article discussing Helex's innovative approach to gene editing and their proprietary platforms.
All the top 10 companies from IndieBio’s latest New York Cohort
Helex is featured among the top biotech startups in IndieBio’s recent cohort.
Helex to present a Lipid Nanoparticle-Mediated CRISPR-based Therapy at ASGCT 2025
Helex will present their lipid nanoparticle-mediated CRISPR therapy for ADPKD at the American Society of Gene & Cell Therapy conference in New Orleans, May 2025.
Helex awarded Aegis Graham Bell Award 2025 in Life Sciences
Helex received the prestigious award for innovation in life sciences, recognizing their advancements in gene editing therapies.
Helex's CEO Poulami Chaudhuri to present at Kidney Treatment Symposium in May 2025
Poulami Chaudhuri, PhD, will speak on new frontiers in kidney gene therapies at the ASGCT conference.
Helex's Chief Growth Officer Rohini Kalvakuntla interviewed by Cure magazine
An interview highlighting Helex's pioneering work in developing therapies for genetic kidney diseases.
Biotech startup Helex brings precision to gene editing
An article discussing Helex's innovative approach to gene editing and their proprietary platforms.
All the top 10 companies from IndieBio’s latest New York Cohort
Helex is featured among the top biotech startups in IndieBio’s recent cohort.