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Sparing gene therapy for Inherited ROd COne dystrophies

Periodic Reporting for period 3 - SIROCO (Sparing gene therapy for Inherited ROd COne dystrophies)

Reporting period: 2021-08-01 to 2022-07-31

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
August 2019-July 2021:
- Successful production of the cGMP Master Cell Bank & plasmids, the key raw materials to produce the clinical batch
- Analytical tests to perform the quality control of the GMP clinical batch qualified (one delayed to Oct 2020)
- Production of 1 confirmation and 2 pilot batches
- Review of the pilot data with GO decision for GMP production
- Completion of Phenorod1 (110 patients)
- Finalization of TPP
- Questionnaires KOL, payers and patients
- Completion of Forecast model for Market access
- cGMP HEH293 cell bank release
- Pilot batches certificates of analysis
- DPO appointed
- Website update
- Protocol Assistance and Pre-IND
- SIROCO Grant amendment
- cGMP Master Cell Bank 18-month stability
- Analytical tests of the GMP clinical batch
- GMP clinical batch produced and released
- Phenorod1 enrolment
- Novel development plan upon Regulatory feedback


Aug 2021-Jul 2022:
- Phenorod2
o Opening US site
o Enrollment completed, 86 included
o 1st interim analysis
o Oral presentation at ARVO 2022
- Update of the market access package
- Compliance upgrade of the process with DPO
- The 12-month stability control for the GMP DP passed
- For the first in human study
o Clinical study protocol
o ICF and all the patient related documents
o IMPD
o investigator brochure
o Pharmacy manual
o Laboratory manual
o DSMB members selected, and charter drafted
o GMO submission
o CTA submissions (part I and part II)
o IND done and submission to FDA
o IRB and IBC submissions. IBC approval granted and IRB final decision pending FDA
o TMF
o Vendors selected and contracted (monitoring, datamanagement, central lab, bioanalysis)
o Investigational sites qualification visits
o Contract negotiation with French site. Finalization on going
o Qualified Person and required audits performed
o IMP import license
o eCRF
Progression of retinitis pigmentosa