Partnership eyes up gene-editing approaches to vision loss

Macquarie University’s Vision Science Group has announced a research partnership with biotechnology company CasBio Therapeutics focused on developing next-generation gene-editing therapies for glaucoma — one of the leading causes of irreversible blindness worldwide.

The partnership will build on ophthalmic research by Associate Professor Vivek Gupta, Professor Stuart Graham and colleagues at Macquarie into mechanisms that damage nerve cells connecting the retina to the brain, leading to irreversible blindness.

“We have identified several genes and proteins that play either a direct pathological role or are otherwise involved in glaucoma and other related diseases of the retina,” Gupta said. “This collaboration creates exciting new opportunities to take the findings from that research and use the expertise of CasBio Therapeutics to help translate them to retinal neuroprotection and regeneration.”

The retina also provides a unique opportunity to study neurodegeneration more generally, making it a critical model for investigating neuroprotection and disease-modifying treatments in other conditions including Alzheimer’s disease, Parkinson’s disease and multiple sclerosis, Gupta explained.

“We believe this collaboration is especially significant because it addresses the critical need for effective treatments for glaucoma and other neurodegenerative diseases that pose a substantial burden on global health,” Gupta said.

The CRISPR Vision partnership team at Macquarie University. L–R: Dr Shahab Mirshahvaladi (Research Fellow, Macquarie University and CasBio Therapeutics), Professor Ghasem Hosseini Salekdeh (CEO, CasBio Therapeutics), Professor Stuart Graham (Ophthalmology and Visual Science, Macquarie University) and Associate Professor Vivek Gupta (Vision Sciences Group, Macquarie University). Source: Macquarie University

CasBio Therapeutics is an Australian subsidiary of a Singapore-based biotechnology company and specialises in developing novel and highly efficient CRISPR-Cas systems for gene therapy and next-generation diagnostics. CRISPR-Cas gene-editing technology is regarded as highly adaptable for clinical use as it facilitates seamless translation to potential therapeutic applications, Macquarie said.

The work of the partnership will initially focus on combining CasBio’s advanced CRISPR-Cas technology with targeted nanodelivery systems to enable precise delivery of gene-editing tools to retinal ganglion cells (RGCs), which are critically affected in glaucoma.

Researchers will evaluate targeted nanocarrier-based delivery of CRISPR-Cas gene-editing systems directly to RGCs in preclinical glaucoma models and assess their impact in terms of neuroprotection and disease modification to support RGC survival. Successful outcomes could pave the way for advanced gene-editing-based treatments for neurodegenerative diseases, Macquarie said.

Professor Ghasem Hosseini Salekdeh, CEO of CasBio Therapeutics and an Honorary Professor at Macquarie, said the collaboration reflects the importance of interdisciplinary partnerships in addressing complex health challenges.

“By bringing together expertise from academia and industry, this initiative provides a platform to explore how gene-editing technologies can be translated into approaches that may ultimately benefit patients,” Salekdeh said. “It is an important step towards connecting scientific discovery with real-world impact.”

Top image credit: iStock.com/Lan Zhang