Delivering therapeutic cargo to kidney cells has long been considered one of the most difficult challenges in drug delivery. Helex recently announced an oversubscribed $3.5 million seed round to advance its non-viral gene therapy approach for genetic kidney diseases, bringing its total funding to over $6 million.
Key Points
- The company is developing programmable lipid nanoparticle (LNP) therapeutics designed to deliver therapeutic cargo directly to kidney cells. Its lead program targets Autosomal Dominant Polycystic Kidney Disease (ADPKD), a genetic disorder affecting more than 12 million people worldwide.
- Helex’s approach aims to use a single-dose non-viral gene editing therapy that could potentially halt or slow disease progression. The company combines proprietary kidney-tropic LNPs with its AI-driven Epic-Cure™ platform for drug design.
- The seed round was led by pi Ventures, with participation from Bluehill Capital, SOSV, and a global syndicate of investors.
- The company operates from New York and ASPIRE BioNEST in Hyderabad, India, with support from Bayer Co.Lab Cambridge.
Patients with ADPKD currently have limited treatment options beyond symptom management, with disease often progressing to dialysis or kidney transplant.
The Data
- The funding will support development of the ADPKD program toward IND enabling studies and pipeline expansion.
- ADPKD is caused primarily by mutations in the PKD1 or PKD2 genes and leads to gradual formation of kidney cysts and decline in renal function.
- The company was founded by three King’s College London alumni: Dr. Poulami Chaudhuri (CEO), Rohini Kalvakuntla (CBO), and Anirudh Nishtala (COO).
- Helex recently appointed Dr. Suman Alishetty, described as a lipid expert and previous scientific co-founder of AexeRNA (acquired by BioNTech), as Vice President of Drug Delivery.
Industry Context
ADPKD represents a significant unmet medical need, with current treatments limited to symptom management and not effective for all patients.
Roopan Aulakh, Managing Partner at pi Ventures
ADPKD patients face what the company describes as a challenging trajectory, often requiring dialysis or kidney transplant. The disease affects millions globally, yet patients have limited treatment options, with tolvaptan (Jynarque) being the only FDA-approved disease-modifying therapy for ADPKD. Helex positions its technology as addressing the longstanding challenge of delivering therapeutic cargo to kidney cells, which has historically limited development of targeted kidney therapies.
The company’s approach combines non-viral gene delivery with what it describes as AI-based drug design through its Epic-Cure™ platform. Helex uses lipid nanoparticles engineered to target kidney cells specifically. The founders bring backgrounds in non-viral delivery, business development for cell and gene therapies, and drug development operations. Helex aims to build what it calls a “robust franchise of renal programs,” though the recent announcement focused exclusively on the ADPKD program.
