Helex: 10 Key Things You Must Know

Overview

Helex is a pioneering biotechnology company dedicated to developing advanced genomic medicines targeted at genetic kidney diseases. Founded in 2021, the company focuses on creating groundbreaking therapies using proprietary lipid nanoparticle (LNP) delivery systems combined with cutting-edge gene editing and AI-based drug design technologies. Notably, Helex's lead program targets Autosomal Dominant Polycystic Kidney Disease (ADPKD), a chronic genetic kidney condition affecting millions worldwide. By addressing one of the hardest challenges in biotech — targeted delivery of genetic medicines to kidney cells — Helex aims to revolutionize treatment options for patients who currently rely largely on symptom management or face severe outcomes like dialysis and transplantation. This article unveils ten key facts about Helex, exploring its technology, team, mission, and impact.

1. Founding and Vision

Helex was founded in 2021 by three scientists and entrepreneurs who are alumni of King’s College London: Dr. Poulami Chaudhuri (CEO), Rohini Kalvakuntla (CBO), and Anirudh Nishtala (COO). Their shared vision is to create curative, one-time genetic therapies for high-burden kidney diseases that have gone largely untreated at the genomic level. Driven by extensive expertise in molecular biology, gene editing, and healthcare business strategy, they focus on bridging gaps between complex science and patient needs. Helex aims not only to innovate therapeutically but also to ensure accessibility and affordability for patients worldwide.

2. Targeting Genetic Kidney Diseases

Chronic kidney diseases (CKD) affect about 1 in 7 people globally, with genetic forms accounting for roughly 30% of cases. Among these, ADPKD is one of the most common inherited disorders, impacting over 12 million people worldwide. It causes progressive cyst formation in the kidneys, leading to kidney failure in many patients. Current management mainly involves symptom control, with limited curative options. Helex’s genetic medicines aim to modify or halt the disease progression at its molecular root, potentially transforming patient outcomes and quality of life.

3. Proprietary Lipid Nanoparticle Delivery Platform

One of Helex’s key technological breakthroughs is its proprietary kidney-tropic lipid nanoparticle system designed for precise, non-viral delivery of gene editing components directly to kidney cells. Delivery to the kidney has been notoriously difficult due to the organ’s complex cellular architecture. By developing bespoke LNPs, Helex can overcome these challenges, enabling highly targeted treatments that avoid off-target effects and improve therapeutic efficacy. This innovation opens the door for a new class of medicines that can be administered in a single dose for long-lasting benefits.

4. Epic-Cure™ 3D Genome-Based Drug Designer Platform

Helex combines its delivery expertise with an AI-driven drug design platform called Epic-Cure™, which leverages 3D genome mapping, deep learning, and bioinformatics to design, validate, and optimize gene editing therapies. This platform allows the company to predict potential outcomes and off-target effects of CRISPR-based genome edits with unprecedented precision. It accelerates the discovery pipeline and improves the safety profile of new therapeutics, making the approach scalable for multiple kidney diseases beyond ADPKD.

5. Leadership and Scientific Team

Led by Dr. Poulami Chaudhuri, who has over 15 years of experience in lipid nanoparticle-based delivery and molecular biology, the Helex team comprises experts in gene editing, drug delivery, and biotechnology operations. Dr. Chaudhuri’s background includes postdoctoral research in 3D genome structure and epigenetics at premier institutions. The team also includes co-founders Rohini Kalvakuntla and Anirudh Nishtala, bringing expertise in business strategy and pharmaceutical development, respectively. The team’s multidisciplinary approach underpins Helex’s innovative drive and patient-centered mission.

6. Recent Funding and Growth

In 2024, Helex successfully raised $3.5 million in an oversubscribed seed funding round led by pi Ventures and supported by Bluehill Capital, SOSV, and a global syndicate of investors. This brought their total funding above $6 million. The capital infusion is driving the company’s IND-enabling studies for their ADPKD therapy and expanding their AI and LNP technology platforms. This financial backing confirms strong investor confidence in Helex’s approach and accelerates progress toward clinical development.

7. Lead Therapeutic Focus: Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Helex’s lead drug candidate targets ADPKD, a genetic disorder primarily caused by mutations in the PKD1 or PKD2 genes. Affecting millions across the globe, ADPKD leads to cysts in the kidney, causing pain, hypertension, and kidney failure often necessitating dialysis or transplant. Helex’s potential one-dose gene editing therapy aims to halt or significantly slow disease progression by correcting or bypassing disease-causing mutations. If successful, it would represent a major advance over current symptom-managing treatments.

8. Addressing the Unmet Need in Kidney Therapeutics

Approximately 700 million people worldwide suffer from chronic kidney diseases, with many progressing to end-stage renal failure. Less than 3% of eligible patients receive kidney transplants annually, leaving most reliant on dialysis, which severely impacts quality of life. Helex focuses on this underserved area, striving to provide curative genetic medicines that reduce the need for invasive procedures. By harnessing genomic medicine for kidney diseases, Helex hopes to alleviate the physical, emotional, and financial burdens on patients and healthcare systems.

9. Global Presence and Collaborative Ecosystem

Helex operates primarily out of New York, USA, with a significant research and development base at the ASPIRE BioNEST incubator at the University of Hyderabad, India. These locations enable strong access to talent, research infrastructure, and global biotech ecosystems. Strategic partnerships, including support from Bayer Co.Lab Cambridge and backing from biotech accelerators like SOSV IndieBio, enhance Helex’s capabilities in science, clinical development, and commercialization, fostering innovation at the intersection of advanced therapeutics and global health.

10. Future Prospects and Pipeline Expansion

Beyond its lead ADPKD program, Helex plans to leverage its programmable LNP delivery and Epic-Cure™ platform to develop a robust pipeline of therapies targeting other genetic kidney diseases with high unmet needs. The company is also expanding the reach of its non-viral delivery technology to enable gene editing in cell types that were previously difficult to target. Their platform’s scalability positions Helex as a potential leader in kidney genomic medicines with the ability to impact millions of patients worldwide.

Conclusion

Helex exemplifies a new frontier in biotechnology by combining innovative drug delivery, precision gene editing, and AI-driven drug design to tackle complex genetic kidney diseases. Its mission centers on transforming patient outcomes for disorders like ADPKD, one of the most common inherited kidney diseases with limited treatment options. With a strong leadership team, cutting-edge technology, and growing investor support, Helex is poised to deliver novel, curative therapies that could improve the lives of millions globally. As genomic medicine continues to evolve, how Helex advances its pipeline and scales these innovations will be crucial in shaping the future landscape of renal therapeutics.