Exciting advances are bringing hope to individuals living with rare inherited retinal diseases (IRDs). Retina South Africa’s latest update highlights several breakthroughs now moving from research to real-world treatment potential: 

Gene Replacement Therapy (GRT) 
Over 209 gene therapy trials have been launched worldwide, targeting single gene IRDs. One treatment for RPE65related LCA/RP is already approved in the US and Europe, offering lifechanging visual improvements.  

Gene Editing – CRISPRCas9 
“Cut and paste” gene editing is being used to remove faulty genetic material and replace it with healthy versions. A promising CRISPRCas9 clinical trial is currently underway, paving the way for mutation specific therapies in the near future. 

Stem Cell Therapies 
Researchers in Japan and the US are testing embryonic and induced pluripotent stem cells to regenerate retinal cells that normally cannot repair themselves. Some Phase I/II trials are already showing functional visual improvement and retinal integration, with potential conditional approvals expected by 2026–2027. 

Artificial Vision – The Prima System
A groundbreaking photovoltaic retinal implant is restoring partial central vision in people with advanced dry AMD. In recent trials, 27 of 32 participants regained the ability to read large print, marking a major milestone in artificial vision technologies. 

Optobionics: Repurposing Retinal cells
Dr. Sheila Nirenberg’s BSO1 optobionic molecule is being tested as a single intravitreal injection that confers light sensitivity onto retinal ganglion cells with no camera or external device required to boost the ambient light. This represents promising improvement of vision for Retinitis Pigmentosa and other retinal conditions. 
Additional advances are emerging from Gensight Biologics, AGTC, Nanoscope, Ray Therapeutics and Kiore. 
 

These innovations mean that treatments for rare retinal diseases are closer than ever before. 
Retina South Africa continues to support patients with genetic testing, counselling, clinical trial readiness, and up to date information on global research progress.