Retinitis pigmentosa (RP) is a rare, genetic disorder characterized by progressive vision loss which involves breakdown and loss of photoreceptors in the retina. Symptoms appear in young adults, but sometime during childhood. RP is progressive as patients first lose night vision and can experience photophobia due to rod degeneration. The visual field is restricted to a “tunnel vision”. The latest stage can lead to blindness. With corrective gene therapy only 1 out of 65 mutations is treated, a small group eligible.
The main factor leading to blindness is loss of a protein, rod-derived cone viability factor (RdCVF) which stimulates cone's aerobic glycolysis essential for cone's renewal. Another protein RdCVFL also secreted in rods and cones protects cones against oxidative stress.
SPVN06, introduces DNAs coding for RdCVF and RdCVFL factors. With a single injection under retina, SPVN06 can treat all RP, potentially treat dry AMD.
SparingVision ambition is to be one of the first companies bringing to market a disease-modifier treatment addressing RP patients regardless of mutations.
RdCVF serves as neuroprotector and anti-oxidative and is necessary to maintain day vision. SPVN06 provides protection to the cones by stimulating the glucose metabolism and repairing the oxidative damages. In SparingVisions proof of concept SPVN06 was injected to mice and provided almost immediate protection and repair to the cones and the progression to blindness stopped.
Launch of first FDA-approved corrective gene therapy for one mutation fulfils unmet need for a curative pharmacotherapy. Approval of additional stem cell therapy therapies will provide more options for patients and stimulate competition. Providing a product treating all the RPs is a huge opportunity for the patients and a challenge for SparingVision.
The objectives for SparingVision in SIROCO were defined in work packages to ensure a successful path to phaseIII and commercialization
WP 1: Produce two pilot and a clinical batches
WP 2: Further secure the clinical development of the gene therapy, SparingVision extend Phenorod1 by conducting a prospective study
Phenorod1&2 objectives
• further describe the natural history of retinal degeneration in RP;
• assess inclusion and efficacy criteria notably a mobility test
• identify patients for Phase I
WP 3: Phase I to assess safety, tolerability and efficacy in RP adult patients. Phase I will be in France where the majority of the patients should be included, and US to complete the cohort.
WP 4: Fulfil regulatory requirements, secure clinical with agency consultations to ensure patient safety. The regulatory workpackage aims to secure obtention of the market authorisations within the best timelines
WP 5: Prepare market access, communicate the results to the opinion leaders and the public, increase media coverage and dissemination results in congresses publications and interviews
