Preclinical AMD Gene Therapy Shows Potential for Other Retinal Diseases


The new findings are consistent with previous research, in which ophNdi1 demonstrated benefit in AMD models.

This article was previously published on our sister site, Ophthalmology Times.

ophNdi1, a preclinical gene therapy intended to treat age related macular degeneration (AMD), may also be effective in treating other eye diseases, according to research published in the journal Pharmaceutics.

The research indicates that ophNdi1 improves mitochondrial performance in retinal ganglion cells, which are dysfunctional in glaucoma and other diseases.

The new findings are consistent with previous research, in which ophNdi1 demonstrated benefit in AMD models, and highlight the potential of the gene therapy to treat multiple eye diseases.

“Because a loss of retinal ganglion cells leads to sight loss in many conditions including inherited optic neuropathies and glaucoma, we are excited that this potential therapeutic approach could provide benefit to many patients in the future,” first author Namoi Chadderton, PhD, Trinity College Dublin, School of Genetics and Microbiology, stated. “Our study shows that ophNdi1 is protective in three models of mitochondrial dysfunction. Notably, the optimization of the therapy, which is outlined in the study, allows for use of a lower therapeutic dose.”

The college noted that mitochondria are known as the “powerhouses” of the cell because they manage the production of energy, however researchers had previously discovered that their performance decreases in the retina of people with eye diseases. This link to a deterioration in sight led them to investigate the potential of therapies to rescue struggling mitochondria.

ophNdi1 uses a virus to access the cells that are suffering and deliver the code needed to give mitochondria a lifeline, enabling them to generate extra energy and continue to function in supporting vision.

“Our work provides clear evidence supporting using this novel gene therapeutic approach for multiple eye disorders,” senior author Farrar Jane Farrar, PhD, professor, Trinity College Dublin, School of Genetics and Microbiology, added. “It also suggests that the ophNdi1 therapeutic platform targeting mitochondrial dysfunction could have applications for other devastating conditions beyond the eye in which mitochondrial dysfunction is in play.”

Further work will need to be undertaken before the therapy can be administered to patients, but the research team hopes that will become a reality in the years to come.

Naomi Chadderton; Arpad Palfi; Daniel M. Malone, Matthew Carrigan. Et al. Optimisation of AAV-NDI1 Significantly Enahances Its Therapeutic Value for Correcting Retinal Mitochondrial Dysfunction. Pharmaceutics. Published January 18, 2023. Accessed February 1, 2023. doi: 10.2290/pharmaceutics15020322
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