SparingVision’s second product, SPVN 20, is a pioneering gene therapy product obtained through the acquisition of Gamut Therapeutics in 2021.

SPVN 20’s unique mutation-agnostic approach aims at restoring visual acuity and color vision in advanced and late-stage Retinitis Pigmentosa (RP) patients with “dormant cones”, independently of their genetic mutation.

About SPVN20

SparingVision Gamut MOA

Description

SPVN20 aims to reactivate patients’ “dormant cones”.

Dormant cones are viable cones with diminished outer segments that no longer respond to light, as such that the patients’ light response decrease 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.

Injection with SPVN20 provides dormant cone cell bodies with a channel protein that would allow to restore a short phototransduction cascade within the dormant cone, restoring electric signal and thereby visual acuity and color vision.

Development status

As of April 2021, SparingVision has made significant progress in the pharmaceutical development of SPVN06. The Company has successfully manufactured pilot runs and completed the first GMP production, which will provide SparingVision with the necessary clinical material for the Phase 1b clinical trial. Simultaneously, IND-enabling studies, to assess SPVN06 pharmacology, biodistribution and safety in rodent and non-rodent species, are ongoing.

The drug product and this mutation-agnostic approach are protected by several patent families.

SPVN06 has been granted Orphan Drug Designation in Europe and interactions with the US and EU regulators have been initiated with a view of a Clinical Trial Authorisation (CTA) and Investigational New Drug (IND) filing in the second half of 2021.

Development plan

The addition of SPVN20 in March 2021 broadened SparingVision’s novel genomic medicine pipeline. The Company is currently assessing the best development plan for this new product candidate, with a view to start pre-IND studies in 2022.

SparingVision also intends to evaluate the potential therapeutic synergy of the two products through the combination of SPVN06 and SPVN20 in a sequence of treatments and/or as a single construct to address a broader population of Retinitis Pigmentosa (RP) patients. Beyond RP, SparingVision will explore opportunities to evaluate SPVN20 in other Rod-Cone Dystrophies, for which its mechanism of action could be particularly relevant.


 Russel et al., 2017. https://pubmed.ncbi.nlm.nih.gov/28712537/