Description

In RP, the outer segment of cone photoreceptors atrophies, losing the ability to respond to light.

While these cone receptors remain present in the retina, they become non-functional or “dormant”. As the natural phototransduction cascade (which allows for normal vision) occurs in the outer segment of the cones, these dormant cones are no longer capable of converting light into an electric signal, leaving patients with tunnel vision and, as the disease progresses, ultimately blindness. Despite this damage, opsins are still present on the membrane of dormant cones and can be activated in ambient, natural light.

SPVN20’s unique mechanism of action allows to restore visual acuity and color vision in patients with advanced and late-stage retinitis pigmentosa (RP) with “dormant cones”, independently of their genetic mutation by delivering a native human ion channel – G-protein inwardly rectifying potassium (GIRK1) – that leverages endogenous opsins. This creates an alternative pathway that generates electrical signals adapting to changes in brightness. SPVN20 aims at restoring cone function by engaging the retina’s intrinsic pathway developed to function under normal lighting conditions without external stimulation devices.

SPVN20 is delivered by a single intravitreal injection, an established and minimally invasive route of administration.

Development plan

SPVN20 has recently started enrollment in NYRVANA (NCT07807111), a Phase I/II clinical trial, aimed at testing the safety, tolerability, preliminary efficacy and quality of life of SPVN20 in up to 27 participants with late-stage RP.

RCD is a rare disease, and only limited information is available about its natural history, with no published prospective or retrospective natural history studies focused on patients with advanced RCD. In parallel to NYRVANA, SparingVision will therefore conduct ECSTASI (NCT07854899), a proprietary natural history study of advanced RCD in parallel with the NYRVANA clinical trial, as part of the overall clinical development plan for SPVN20. ECSTASI will be used as an external control for NYRVANA and provide information regarding endpoints for the whole clinical development program of SPVN20 in advanced RCD.

Beyond RP, SparingVision will explore opportunities to evaluate SPVN20 in other blinding retinal diseases, where its mechanism of action could be particularly relevant.

Disclaimer

This investigational gene therapy has not been approved by any regulatory authority and is not available for commercial sale. The information presented on this page relates to an investigational product currently under clinical evaluation. Statements regarding its potential safety, efficacy, mechanism of action, or therapeutic benefit are based on non-clinical data and/or ongoing clinical research and should not be interpreted as established clinical outcomes.