GenSight Biologics’ proprietary MTS platform is designed to address defects in the mitochondrial genome by extending the reach of gene therapy beyond the cell nucleus. This technology, currently being applied to vision loss, may open a new therapeutic pathway for mitochondrial dysfunction.
Mitochondrial-Targeted Gene Therapy
The Challenge:
Mutations affecting the mitochondrial genome impair the function of the mitochondria, the organelles essential to cellular energy production. Because mitochondrial DNA is distinct from nuclear DNA, these mutations escape conventional gene therapy approaches, which are primarily designed to target the cell nucleus.
The Solution:
GenSight’s gene therapy delivers a gene to the cell nucleus, where it is transcribed into messenger RNA (mRNA). Our MTS technology shuttles the mRNA to the mitochondrial surface, where the functional protein needed to restore cellular energy is synthesized and translocated into the mitochondria.
GS010: Our Lead MTS Program
GS010 is GenSight Biologics’ MTS-enhanced product candidate targeting ND4-LHON, the most common form of Leber Hereditary Optic Neuropathy (LHON), a severe inherited blinding disease and the world’s most prevalent mitochondrial condition. Caused by a mutation in the mitochondrial ND4 gene, the disease leads to the degeneration of retinal ganglion cells and, consequently, to rapid and irreversible vision loss. GS010 aims to restore mitochondrial function and rescue vision.
GS010/LUMEVOQ® has not been granted marketing authorization in any country and is not commercially available.
The excerpts below are drawn from third-party sources and reflect the views of their authors alone.
Potential Applications
Mitochondrial Diseases
Central Nervous System Disorders
Neurodegenerative Disorders
Pipeline
Learn more about the clinical development of GS010,
our MTS-enhanced product candidate
