The road to treatment 12

Report of meeting with Abeona Therapeutics by Peter Makai.

Last summer we had a meeting with Abeona Therapeutics, a company working on a gene therapy to treat ADOA. In ADOA, there is a genetic error in the so-called OPA1 gene, which is necessary for the functioning of the mitochondria in the cells. If this is not done, the cells of the optic nerve and retinal ganglion cells will die, leading to a decrease in vision. Gene therapy holds the promise of halting this process and slowing the slow decline in vision that many people experience.

Abeona is working on several therapeutic solutions and they will ultimately select the best one that can be a possible treatment. The idea is to inject a mixture into the eye. A major advantage of gene therapy is that such an injection only needs to be given once. It is quite difficult to reach the actual optic nerve cells, but Abeona has developed a technology to do that. In animal studies, the gene therapy has replaced the function of OPA1 and improved the vision of mice with ADOA (mice are often genetically modified to study a disease or develop a drug).

At this point, Abeona must determine if the chosen mixture has any negative side effects and they must conduct animal studies to rule this out before giving it to patients. Although this is in the early stages of development, this is still an exciting development. We will monitor the progress of this study. We certainly hope that this study will go well and that it can be tested in patients in the future!