SparingVision Presents Additional Data on its Breakthrough Gene Therapy Approach SPVN06 at ASGCT 2022
- Data from the 1-month pilot tolerability and biodistribution study in non-human primates show that SparingVision’s gene independent program SPVN06 is well tolerated. In addition, pharmacology study shows that SPVN06 dramatically reduces vision loss in a murine model of retinitis pigmentosa.
- Findings provide additional preclinical validation ahead of IND/CTA submission planned in Q3 2022
- Oral presentation will take place today at 10:45AM-11:00 AM EDT (04:45PM-05:00PM CET)
Paris, May 19, 2022 – SparingVision (“the Company”), a genomic medicine company developing vision saving treatments for ocular diseases, announces that it will present additional positive preclinical findings for its lead gene independent program SPVN06, a breakthrough gene therapy approach targeting Inherited Retinal Diseases (IRDs), in an oral presentation today at 10:45AM-11:00AM EDT at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting taking place in Washington D.C.
Dr. Florence Lorget, Chief Development Sciences Officer at SparingVision, will share positive pharmacology data demonstrating a highly significant protection of visual function and structure loss following subretinal administration of SPVN06 in the rd10 mouse model. Data from a 1-month tolerability and biodistribution study further shows that SPVN06 is well tolerated in non-human primates (NHP). These results provide robust preclinical validation ahead of the Company’s Clinical Trial Authorisation (CTA) submission and Investigational New Drug (IND) application, which are both expected in Q3 this year.
Stéphane Boissel, President and Chief Executive Officer of SparingVision, said: “The additional data from our IND-enabling studies reassert the safety and efficacy potential of SPVN06 in treating retinitis pigmentosa, one of the most common inherited retinal diseases with significant unmet medical need and the lead indication for our product. The GLP toxicology study, in its finalization stage, marks the final step in the path ahead of our CTA and IND submissions. We’re at a pivotal stage of growth and look forward to bringing our unique, gene-agnostic, and disease-independent gene therapy approach to the clinic, with a first-in-human trial expected to commence by the end of 2022.”
More details of the presentation can be found below:
Oral Presentation: SPVN06, a Novel Mutation-Independent AAV-Based Gene Therapy, Dramatically Reduces Vision Loss in the rd10 Mouse Model of Rod-Cone Dystrophy and is Well Tolerated in a 1-Month Pilot Safety Monkey Study
Presenter: Dr. Florence Lorget, Chief Development Sciences Officer at SparingVision
- Positive pharmacology data in a murine model showed significant preservation of visual acuity at all timepoints (P32, P38, and P45), and greater cone density at P48 in the SPVN06-treated retinas compared to controls.
- Safety data from a 1-month tolerability and biodistribution study in non-human primates (NHPs) showed that SPVN06 administration was well tolerated at dose 7E10 vg/eye with no test article related systemic effects, macroscopic or microscopic observations in the eye, brain, or liver. SPVN06 cell transduction and transgene expression occurred with high expression of both transgenes in the photoreceptor layer and retinal pigment epithelium (RPE).