Hyderabad Startup Targets Kidney Disease with Gene Editing

Helex Inc., a Hyderabad-based biotechnology startup, is developing a first-in-class mechanism for the safe and effective delivery of gene editing drugs directly into kidney cells. This innovative approach tackles a major medical challenge, with the company aiming to make these advanced therapies accessible and affordable globally, particularly for India.

The startup's focus is on genetic kidney diseases, with their lead candidate being a single-dose gene-editing drug for autosomal dominant polycystic kidney disease (ADPKD). This condition causes cysts to grow in the kidneys, impairing function over time.

Helex employs proprietary lipid nanoparticles (LNPs) to deliver the drug, enabling it to reach and 'edit' the faulty genome within kidney cells. While gene editing has shown success in other organs, the kidney has presented unique delivery challenges, which Helex's technology is designed to overcome.

The company has demonstrated the efficacy of its lipid nanoparticles in delivering and editing target cells in animal models, including mice, rodents, and non-human primates. Helex is now preparing to advance its program towards an investigational new drug (IND) application with the US Food and Drug Administration to enable clinical trials.

Helex has secured $6.2 million in pre-seed and seed funding and is preparing for a Series A round to support phase 1 trials. The company operates with a hybrid model, partly based in the US and partly in India, to leverage expertise while maintaining cost efficiency. They are also exploring partnerships to license their technology for other kidney diseases, generating revenue and sustaining operations.