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Helex Secures $3.5M Seed Funding to Develop Non-Viral Gene Therapy for Polycystic Kidney Disease

2 days ago3 min read

Key Insights

  • Helex raised $3.5 million in an oversubscribed seed round led by pi Ventures to advance non-viral gene therapies targeting genetic kidney diseases.

  • The company is developing programmable lipid nanoparticle therapeutics to deliver therapeutic cargo directly to kidney cells, addressing a major drug delivery challenge.

  • Helex's lead program targets Autosomal Dominant Polycystic Kidney Disease (ADPKD), which affects over 12 million people worldwide with no current disease-modifying treatments.

Therapeutics company Helex announced the completion of an oversubscribed $3.5 million seed funding round to advance its non-viral gene therapy platform targeting genetic kidney diseases. The round was led by pi Ventures, with participation from Bluehill Capital, SOSV, and a global syndicate of investors, bringing the company's total funding to over $6 million since its founding in 2021.

Breakthrough Kidney-Targeted Delivery Platform

Helex is developing programmable non-viral lipid nanoparticle (LNP) therapeutics designed to deliver therapeutic cargo directly to kidney cells, addressing what has long been considered one of the greatest challenges in drug delivery. The company's approach represents a paradigm shift in treating chronic and rare kidney disorders by enabling targeted, disease-modifying medicines for patients who currently have no effective treatment options.
"This is more than a new treatment; it's a new hope," said Dr. Poulami Chaudhuri, co-founder and CEO of Helex. "For far too long, patients with ADPKD have had to rely only on symptom management, with no true disease-modifying options. We wish to change that. By enabling targeted delivery of genomic medicines directly to the kidney, we are developing a transformative therapy that is also accessible and affordable for every patient who needs it."

Targeting ADPKD's Massive Unmet Need

The company's lead program focuses on Autosomal Dominant Polycystic Kidney Disease (ADPKD), an inherited disorder caused primarily by mutations in the PKD1 or PKD2 genes. ADPKD affects more than 12 million people worldwide, leading to gradual formation of kidney cysts and decline in renal function. Patients with ADPKD face a devastating trajectory that often culminates in dialysis or kidney transplant.
Helex aims to develop a single-dose non-viral gene editing therapy that could halt or significantly slow disease progression. Current treatments for ADPKD are limited to symptom management and are not effective for all patients, representing a significant unmet medical need.

Advanced Technology Platform

The funding will accelerate pre-clinical development of the lead ADPKD program while advancing Helex's proprietary kidney-tropic LNP delivery system and its Epic-Cure 3D genome-based drug design platform. According to Chaudhuri, this platform examines disease and cellular details to maximize gene editing accuracy and minimize off-target effects.
"Such therapies allow you to actually go into the basis of it and cure the disease itself," Chaudhuri explained. "For example, in our DNA, we have about 6 billion bases or letters. If any letter goes wrong, there is a life-threatening disease that can actually happen. Advanced therapeutics offer an opportunity to get to the base of the disease, correct the DNA once and for all."

Market Opportunity and Expansion Plans

The company also plans to expand its product pipeline toward other kidney indications, both independently and through collaborations with other medical companies. India's advanced therapeutics market is estimated at $13 billion in 2025 and projected to reach $52.4 billion by 2035, representing a compound annual growth rate of 15%.
"ADPKD represents a significant unmet medical need, with current treatments limited to symptom management and not effective for all patients," said Roopan Aulakh, Managing Partner at pi Ventures. "Gene therapies are emerging as one of the most promising frontiers in medicine. Helex's first-in-class mechanism of action has the potential to be a curative therapy and positions the company to build a differentiated pipeline of therapies for kidney diseases."
Founded by Poulami Chaudhuri, Anirudh Nishtala, and Rohini Kalvakuntla, Helex represents part of a growing trend of investors backing startups developing advanced therapeutics to revolutionize healthcare through gene and cell therapy approaches.
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