SparingVision Announces First Patient Dosed in NYRVANA Trial for Gene-Agnostic Therapy SPVN20 in Advanced Retinitis Pigmentosa

SPVN20 is a gene-independent therapy based on an intravitreal AAV vector, with the core goal of restoring vision and color perception in patients with retinal diseases.

It precisely targets "dormant cone cells" — photoreceptor cells in the retina that, although still alive, have lost their ability to respond to light due to disease. Through a single intravitreal injection, SPVN20 uses an AAV vector to deliver the gene encoding the human G protein-gated inwardly rectifying potassium (GIRK) channel, enabling dormant cone cells to regain the ability to convert light signals into visual electrical signals recognizable by the brain. This unique mechanism is not limited by the genetic cause of the disease, meaning it has broad applicability across various retinal degenerative diseases.

NYRVANA First-in-Human Trial is an open-label, multi-center, dose-escalation study focusing on patients with advanced retinitis pigmentosa (RP) who retain dormant cone photoreceptors.

The trial will evaluate the safety, tolerability, and preliminary efficacy of a single intravitreal injection of SPVN20 over six months, with a five-year long-term follow-up period. Safety and efficacy data will be collected in 2026 and 2027.

The trial has currently been launched in Belgium, and patient recruitment will subsequently expand to France and Ireland. Its Clinical Trial Application (CTA) is supported by comprehensive non-clinical data.Demonstrated SPVN20-mediated phototriggered reactions and functional rescue of cone photoreceptors in multiple in vitro, ex vivo, and in vivo models.Robust transgene expression targeting the central fovea and good safety profile in non-human primates after a single intravitreal injection.

Alongside another investigational therapy, SPVN06, SparingVision's two core clinical programs have established the first and most comprehensive gene-independent RP treatment system, with the potential to cover all patients — regardless of their genetic background or disease stage.

SparingVision CEO Stéphane Boissel: "The launch of NYRVANA is a key milestone for SparingVision, validating the therapeutic potential of our gene-independent gene therapy portfolio and providing a comprehensive solution for the treatment of various stages and types of retinal diseases. With two clinical programs progressing in parallel, we are steadily advancing towards our mission of transforming outcomes for patients with blinding retinal diseases."

Source: Rare Disease Information Network