New Gene Replacement Clinical Trial Launched for RP Test

Tuesday, September 3 2024 | 08 h 46 min | Vision Science

ViGeneron in Munich, Germany has launched a new Phase 1b clinical trial testing a novel gene replacement therapy in individuals affected by retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. Rod photoreceptor cells sense light and convert light signals to electric signals which are then passed to the brain to create vision. CNGA1 plays an important role in the conversion step and when CNGA1 is mutated this disrupts the ability of the rod cells to send visual signals and leads to vision loss.

Typically, retinal gene therapy has been delivered via a subretinal injection – this involves a surgical procedure that detaches the retina and delivers the treatment directly to the back of the eye close to photoreceptor cells.

This new therapy can instead be injected into the vitreous. Injections such as these are frequently done in an ophthalmologist’s office and do not require more advanced surgery.

This first-in-human trial will test if the gene therapy is safe and will also look at effectiveness.

About Fighting Blindness Canada

Fighting Blindness Canada (FBC) is the largest charitable funder of vision research in Canada.

Over our 49-year history, FBC has contributed critical funding for the development of sight-saving treatments and cures for blinding eye diseases. By raising and stewarding funds, FBC is helping drive forward research that supports our goal of understanding why vision loss occurs, how it can be slowed and how sight can be restored.

We are an invaluable resource for individuals and families impacted by blindness, providing accurate eye health information through our website and educational events, as well as engaging with government and other stakeholders to advance better vision health policies.

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