top of page

This just in: substantial developments in the advancement of optic mitochondrial disease treatment.

Advancements in Optic Mitochondrial Disease Treatment

As reported by Mitochondrial Disease News, a potential treatment for optic mitochondrial diseases has been deemed “safe and well tolerated” in initial clinical trials conducted by GenSight Biologic.

GenSight Biologic is a biotechnology company committed to finding treatments and cures for mitochondrial and neurodegenerative diseases. The company is currently conducting the first phases of a clinical trial to test the feasibility of using GS010 gene therapy as a treatment option for a rare mitochondrial disease called Leber’s Hereditary Optic Neuropathy (LHON). After two years of testing and following up with subjects during Phase I/II of the clinical trial, the GenSight Biologic researchers have deemed GS010 safe and tolerable.

This is incredible news for the Autosomal Dominant Optic Atrophy (ADOA) community. LHON is one of the most closely associated mitochondrial diseases to ADOA, as both diseases affect the DNA of the retinal ganglion cells in the optic nerve, causing rapid loss of vision. If researchers continue to see these positive outcomes from the GS010 clinical trial, it could potentially result in support for similar trials conducted on those affected by ADOA.

Luckily, this is only the beginning of GenSight Biologic’s research into the use of GS010 gene therapy to treat LHON. Topline results are expected next month from the company’s Phase III clinical studies, titled RESCUE and REVERSE. Whereas the Phase I/II trial was designed to study the physical reactions to GS010 injections, the RESCUE and REVERSE trials are explicitly studying the effectiveness of GS010 as a treatment option for LHON.

You can help get involved with future research as well. The Autosomal Dominant Optic Atrophy Association is dedicated to finding a cure for ADOA, but clinical trials and medical research require a substantial amount of funding. If you share our passion and want find a cure for ADOA, please join us and get involved.

To review the full, published results of GenSight Biologic’s Phase I/II clinical trial, visit their article in the Journal of the American Academy of Ophthalmology.


bottom of page