Health · Medical Research · 3 hrs ago

Helex develops kidney-targeted gene-editing therapy for genetic disease

Helex develops kidney-targeted gene-editing therapy for genetic disease

Hyderabad biotech Helex is developing a gene-editing treatment designed to reach kidney cells.

The company was founded by Poulami Chaudhuri, Anirudh Nishtala and Rohini Kalvakuntla, who want to develop advanced medicines that are affordable in India and elsewhere.

Its lead candidate is a single-dose therapy for ADPKD, a genetic condition in which fluid-filled cysts grow in the kidneys and reduce their function over time.

Helex says its lipid nanoparticles are designed to carry the drug into targeted kidney cells, where it could edit faulty genes and potentially slow or reverse the disease.

No gene-editing therapy has yet reached the kidney, while the existing drug to slow cyst growth can carry a risk of liver injury.

Helex has filed four patents and presented research posters at international scientific meetings, but the company is still developing its approach.

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What people are saying

Poulami ChaudhuriHelex chief executive officer
While gene editing has been validated in patients for other indications such as liver diseases, no therapy has yet been able to reach the kidney. The biggest challenge is delivering therapeutic agents to the kidney safely and in a targeted way, and that is exactly what our proprietary delivery technology is built to solve
via Mint
The three of us have spent a significant amount of time in the West, and we saw what advanced therapies could really do—the promise of it. The idea was to grow that sort of innovation in India
via Mint
Rohini KalvakuntlaHelex chief business officer
The motivation was to have top-class medicine and science, but it has to be accessible and affordable for patients in India and worldwide
via Mint