Gene therapy, a revolutionary approach to treating neurodegenerative diseases
Gene therapy is a revolutionary approach to treating and potentially curing genetic disorders by addressing the root cause - our DNA. Since most diseases, including cancer and neurodegenerative conditions, involve genetic factors, this cutting-edge technology works by introducing, modifying, or replacing genes to correct faulty information and restore normal function. It holds immense promise for conditions previously considered untreatable, offering hope for a healthier future.
But Blood-Brain-Barrier is actually a strong biological obstacle for gene therapy
The human brain has an almost impregnable fortress: the blood-brain barrier. While it protects it from external aggressions, it also blocks 98% of treatments. Delivering molecules and in particular gene therapies to the central nervous system remains a critical challenge.
We are on road to enable the delivery of gene therapy thanks to our innovative microbubbles.
Harmonix is developing innovative microbubbles to encapsulate and deliver gene therapy in a targeted, safe and non-invasive way in the brain. Our patented microbubbles now make it possible to deliver gene therapies in the central nervous system thanks to their activation by focused ultrasound in the blood-brain barrier.
Multiple
applications
We are able to deliver different types of molecules: from single stranded oligonucleotides, mRNA to larger double stranded plasmid DNA. Our technology enables repeated injections.
Microbubbles combined with focused ultrasound enable reproducible, transient, and safe opening of the Blood-Brain-Barrier.
Non invasive drug delivery
Targeted gene delivery
Fast, easy, and safe delivery
Reproductible BBB opening
Efficient nucleic acid delivery
Efficient gene expression
Various payloads, increasing load capacity
