Exsilio develops genomic medicines that utilize lipid nanoparticles to deliver therapeutic genes into safe harbors within DNA, targeting genetic diseases, cancer, and autoimmune conditions. This approach enables precise gene insertion through RNA intermediate templates, providing a durable and safe treatment option for a wide range of diseases.
Funding
$82M 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.
NVFounders
Product
Problem
Many genetic diseases, cancers, and autoimmune conditions lack durable and safe treatment options. Current therapies often fail to address the underlying genetic cause or carry significant risks of off-target effects and immune responses. Precise and targeted gene insertion remains a challenge in genomic medicine.
Solution
Exsilio is developing genomic medicines that utilize lipid nanoparticles (LNPs) to deliver therapeutic genes into specific, pre-defined "safe harbor" locations within a patient's DNA. This targeted approach leverages RNA intermediate templates to enable precise gene insertion, minimizing the risk of disrupting other genes or triggering adverse immune reactions. By inserting therapeutic genes into safe harbors, Exsilio aims to provide durable and potentially curative treatments for a wide range of diseases with unmet medical needs. The LNP delivery system is designed to efficiently transport the therapeutic payload to the target cells, maximizing efficacy and minimizing off-target effects. This technology offers a novel approach to gene therapy, addressing the limitations of existing methods and paving the way for safer and more effective genomic medicines.
Target Audience
The primary target audience includes patients suffering from genetic diseases, cancers, and autoimmune conditions, as well as pharmaceutical companies and research institutions seeking innovative gene therapy solutions.
Features
- Lipid nanoparticle (LNP) delivery system for targeted gene delivery
- RNA intermediate templates for precise gene insertion into safe harbor locations
- Minimized risk of off-target effects and adverse immune responses
- Potential for durable and curative treatments