• 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-11:00AM EDT (04:45PM-05:00PM CEST)

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) 25^th 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).

NOTES TO EDITORS:

About SparingVision

SparingVision is a genomic medicines company with a mission to translate pioneering science into vision saving treatments. Leveraging its unparalleled understanding of retinal diseases, SparingVision has built the world’s most compelling portfolio of synergistic cutting-edge gene therapy and genome editing treatments for inherited retinal diseases (IRDs). Both of its most advanced products, SPVN06 and SPVN20 look to go beyond single gene correction therapies to deliver new mutation agnostic treatments for Retinitis Pigmentosa (RP), a group of IRDs which are the leading cause of blindness globally. The Company also has a strategic collaboration with Intellia Therapeutics (NASDAQ:NTLA) to develop novel genome editing-based treatments for ocular diseases utilizing CRISPR-Cas9 technology.

SparingVision is backed by high-quality international investors including 4BIO Capital, AdBio Partners, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, Intellia Therapeutics, UPMC Enterprises, Jeito Capital, Ysios Capital.

Visit www.sparingvision.com for more and follow us on LinkedIn and Twitter @SparingVision.

About SPVN06:

SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress which, acting synergistically, aim at slowing or stopping the degeneration of cone photoreceptors, which inevitably leads to blindness in patients with rod-cone dystrophies (RCD). SparingVision’s primary disease target is Retinitis Pigmentosa (RP), one of the most common inherited retinal diseases that affects two million patients worldwide. There is currently no treatment approved to treat RP patients independently of their genetic background. This approach is potentially applicable to many more diseases where the loss of rods is known to be an early signal of the disease. First-in-man trials, with SPVN06 in patients with RP, will be commencing in H2 2022.

Miriam Cortes

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