Despite decades of progress in nephrology, millions of patients with autosomal dominant polycystic kidney disease (ADPKD) still face a grim reality - a lifelong struggle that often ends in dialysis or kidney transplant. ADPKD is the most common genetic kidney disorder, affecting nearly 12 million patients globally. It is caused by mutations in one of the two genes, PKD1 (nearly 85% of all patients) and PKD2, which lead to the formation of cysts that gradually impair kidney function.
Today, there is still no curative treatment. The only approved drug, Tolvaptan, can slow cyst growth but comes with severe side effects, including liver toxicity. As a result, it is prescribed only to patients with no liver contraindications and does not fundamentally address the root cause of the disease. The unmet need remains huge.
At pi Ventures, we believe the next decade in healthcare will belong to programmable therapeutics, interventions that can precisely correct disease mechanisms at their genetic or molecular origin. Helex exemplifies this vision and we are excited to lead their $3.5M Seed round.
Helex’s approach represents a novel lipid nanoparticle (LNP) driven gene editing therapy for ADPKD. Their strategy stands out on three fronts:
- Mutation-agnostic mechanism: Instead of targeting specific genetic variants, Helex’s approach focuses on a regulatory pathway common to all PKD1 mutation patients, making it potentially applicable across the entire patient population.
- Kidney-targeted delivery system (LNP): Delivering therapeutic cargo to the kidney has been a critical challenge, due to the complexity of the kidney structure and the multiple cell types present in it. Most commercially available LNPs accumulate in the liver. Helex has engineered LNPs optimized for kidney delivery, tuning their size, charge, and composition to overcome the kidney’s filtration barriers and reach affected tubular cells and collecting ducts.
- Precision-designed guide RNA: Using their AI led proprietary platform, EPIC-Cure™, they have incorporated 3D genome structure, epigenetic context, and disease-specific tissue data, to design the guide RNA (gRNA) for the Cas9 editor to ensure robust on-target editing efficiency with minimal to no off-target effects for a safe and precise therapy.
Together, these components form a highly defensible and integrated therapeutic platform. Helex has already demonstrated encouraging early data to showcase biodistribution and functional efficacy in animal models and patient cell lines. These results validate the potential of Helex’s approach and have already sparked interest from global pharma companies exploring potential collaborations. With their latest funding round, Helex will advance into preclinical studies focused on demonstrating efficacy and safety across multiple disease models, setting the stage for clinical trials ahead. While ADPKD is the lead indication, Helex’s platform combining programmable delivery system and the AI powered drug design platform can be extended beyond gene editing to develop curative therapeutics (including mRNA therapy, RNA editing, gene therapy etc) for various genetic renal diseases such as Alport Syndrome, which affects nearly 1 million patients worldwide.
Helex’s founders - Poulami, Rohini and Anirudh are long-time friends turned co-founders and together make a complementary founding team blending science, business, and clinical execution. They are bold, deeply committed to their mission, and driven by a shared vision to build a global biotech company rooted in both India and the US. They are supported by a world class advisory team of clinicians, highly experienced gene therapy developers and business advisors to help steer the company progress.
With Helex, we now have a strong therapeutics portfolio encompassing small molecules (Ahammune), antibodies (ImmunitoAI), medical nanorobots (Theranautilus) and gene therapy. At pi, we often move between worlds - from companies exploring the cosmos to those reprogramming life at the cellular level. It’s a journey across scales, but driven by one constant: innovation as the engine of progress. We are proud to partner with such disruptive companies as they shape the future.