ViGeneron launches clinical trial of gene therapy for RP caused by CNGA1 mutations 

Key Takeaways

• The first patient has been dosed in a phase 1b clinical trial for a gene therapy to treat retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. 
• The therapy, which does not require surgery for administration, uses a harmless virus to deliver healthy copies of the CNGA1 gene.

ViGeneron GmbH, a gene therapy development company in Munich, Germany, has dosed the first patient in a Phase 1b clinical trial for its gene therapy for people born with retinitis pigmentosa (RP) caused by mutations in the gene CNGA1. CNGA1 plays a critical role in phototransduction, the conversion of light into electrical signals, in rod photoreceptors, the retinal cells that provide night and peripheral vision. As a result, people with RP caused by CNGA1 mutations initially lose night and peripheral vision. As the condition progresses, patients lose cone photoreceptors and central vision.

The emerging gene therapy uses ViGeneron’s adeno-associated virus (AAV) gene-delivery technology known as vgAAV, which enables injection of the treatment into the vitreous, the soft gel in the middle of the eye. Intravitreal injections are minimally invasive and are frequently performed in a doctor’s office. It offers a good alternative to most AAVs, which require subretinal delivery via surgery. The company says the vgAAV can broadly deliver copies of the CNGA1 gene to photoreceptors, which are at the back of the eye.

For more information on this trial, visit https://clinicaltrials.gov/study/NCT06291935or contact Andrea Rindtorff at +49 7071 29 87747 or andrea.rindtorff@stz-eyetrial.de. Note that this trial is taking place in Germany.

Edited by Suditi Kedambadi and Miriam Kaplan, PhD

Source: Foundation Fighting Blindness, Eye on the Cure Research News, April 11, 2024; see source article