Helex develops genomic medicines utilizing proprietary drug design and lipid nanoparticle delivery platforms to treat genetic kidney diseases. The company focuses on non-viral targeted delivery to the kidney to unlock novel therapeutics for conditions like Autosomal Dominant Polycystic Kidney Disease. Their technology aims to provide safer and more efficient gene editing-based treatments for patients with significant unmet medical needs.
Funding
$3.2M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Chronic kidney diseases affect a significant portion of the global population, with a substantial percentage having a genetic origin. Many patients progress to end-stage renal failure, requiring frequent dialysis or kidney transplantation, interventions that severely impact their quality of life. There is a large unmet need for curative therapies that address the underlying genetic causes of these diseases.
Solution
Helex is developing targeted gene editing therapies for genetic kidney diseases using its proprietary EPIC-Cure™ technology and lipid nanoparticle (LNP) delivery systems. The company's approach focuses on precision drug design based on the 3D genome structure and epigenome to enhance the safety and efficacy of in vivo editing. Their LNP technology enables targeted delivery of genetic medicines to the kidney, aiming to correct the underlying genetic defects responsible for chronic kidney diseases. By leveraging data and machine learning, Helex seeks to accelerate drug development and reduce the trial-and-error associated with designing gene editing-based drugs.
Target Audience
The primary target audience includes individuals suffering from chronic kidney disease with a genetic origin, specifically Autosomal Dominant Polycystic Kidney Disease (ADPKD), and the healthcare providers who treat them.
Features
- EPIC-Cure™ technology for 3D genome-based drug design, enhancing precision in gene editing.
- Proprietary lipid nanoparticle (LNP) technology for targeted delivery of genetic medicines to the kidney.
- Focus on avoiding unwanted editing in non-target cells and tissues to improve safety.
- Comprehensive short-term and long-term impact estimation of editing on the genome and cell functioning.
- Capabilities for proprietary target identification and rational gene editing drug design suitable for multiple editing nucleases.