SparingVision’s lead product, SPVN06, is a breakthrough gene therapy approach targeting Rod-Cone Dystrophies (RCD). SPVN06 has the potential to slow or stop the degeneration of cone photoreceptors, which will ultimately lead to blindness. The primary disease target for SPVN06 is Retinitis Pigmentosa (RP), one of the most common inherited retinal diseases that affects two million patients worldwide. SPVN06 treatment is mutation agnostic, and therefore potentially capable of addressing more than 70 known genetic mutations of RP. SPVN06 expresses proprietary neurotrophic factors and is delivered via a single subretinal injection.
Unlike traditional gene therapies which replace a specific defective or missing gene in a given cell, SPVN06, SparingVision’sour lead drug product, acts independently of the causative gene mutation. SPVN06 introduces RdCVF and RdCVFL, two neurotrophic factors naturally produced by photoreceptors (rods and cones) in the eye in normal conditions, in patients experiencing a significant deterioration of their sight. photoreceptors (rods and cones). The DNA of the two distinct isoforms (RdCVF and RdCVFL) of the NXNL1 gene are supplied via an associated-adenovirus (AAV), a viral vector classically used in gene therapy. SPVN06 is delivered via the subretinal route to the cones and retinal pigmented epithelium (RPE). Such route of administration is already used in ophthalmology, for instance with Luxturna [Russel et al;, 2017). Upon delivery of SPVN06, the transgenes are mainly expressed in the RPE cells for RdCVF and in the cones for RdCVFL. SPVN06 is expected to provide a long-lasting neuroprotective effect to prevent the progression of RP in patients with moderate to severe pathology.
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 in 2020 and 2021.
The Company intends to start its first-in-human trial shortly after receiving a Clinical Trial Authorisation (CTA) and Investigational New Drug (IND). The first clinical trial will be a Phase 1b safety study aimed at testing the safety and tolerability of SPVN06 on a limited number of patients.
SparingVision’s goal is to bring SPVN06 to the market as quickly as possible, giving treatment options to the two million patients across the globe, who are currently living with this slowly progressing disease which will eventually lead to blindness.
Russel et al., 2017. https://pubmed.ncbi.nlm.nih.gov/28712537/