SparingVision’s second product, SPVN20, is a is a gene agnostic gene therapy aiming to restore vision for people with blinding retinal disease.
Its unique mechanism of action allows to restore visual acuity and color vision in advanced and late-stage Retinitis Pigmentosa (RP) patients with “dormant cones”, independently of their genetic mutation.
Description
SPVN20 aims to reactivate patients’ “dormant cones”.
As part of the course of disease in RP, the outer segment of cone photoreceptors atrophies, losing the ability to respond to light. Cone photoreceptors are still present but they become non-functional or “dormant”. Patients’ light response decreases and they become unable to see. Since the 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. However opsins are still present on the membrane of dormant cones and can be activated in ambient, natural light.
SPVN20 unique mechanism of action introduces the GIRK (G-protein inwardly rectifying potassium) channel into dormant cones, which would allow to create an alternative phototransduction cascade into dormant cones – making them functional again.
Development plan
The addition of SPVN20 in March 2021 broadened SparingVision’s novel genomic medicine pipeline. SPVN20 is expected to enter the clinic in 2025. Beyond RP, SparingVision will explore opportunities to evaluate SPVN20 in other IRDs, where its mechanism of action could be particularly relevant.
SPVN30
SparingVision also intends to evaluate the potential therapeutic synergy of its two lead products through the combination of SPVN06 and SPVN20 as a single construct, called SPVN30, to address a broader population of Retinitis Pigmentosa (RP) patients.
Publications
Reactivating the phototransduction cascade with a mutation agnostic gene therapy preserves vision in Rod-Cone Dystrophies
Simon, C.J.; Khabou, H; …Sahel, J.A; Audo, I; Herlitze,S; Deniz Dalkara (2025)
iScience,112106,2589-0042.
Vertebrate cone opsins enable sustained and highly sensitive rapid control of gi/o signaling in anxiety circuitry
Masseck, O. A., Spoida, K., Dalkara, D., Maejima, T., Rubelowski, J. M., Wallhorn, L., …Herlitze, S. (2014).
Neuron, 81(6), 1263–1273.
Crystal structure of the mammalian GIRK2 K +channel and gating regulation by G proteins, PIP 2, and sodium
Whorton, M. R., & MacKinnon, R. (2011)
Cell, 147(1), 199–208.
X-ray structure of the mammalian GIRK2-βγ Gprotein complex
Whorton, M. R., & MacKinnon, R. (2013)
Nature, 498(7453), 190–197.
PARTNERS
