SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal disease, announces that it has received FDA agreement for its plan to initiate a Phase II clinical trial of its lead gene-agnostic gene therapy program, SPVN06, in Geographic Atrophy (GA) secondary to dry age-related macular degeneration (dAMD). GA is a condition affecting millions of people globally and a leading cause of blindness in older adults.
The Company received positive Type C meeting feedback from the FDA, in which the agency confirmed that SparingVision’s data package – including preclinical studies and safety data from the ongoing PRODYGY trial in retinitis pigmentosa – is sufficient to support an application for a Phase II study in patients with GA secondary to dAMD. The FDA also agreed with the proposed study design, enabling the Company to move forward with plans to address a major unmet need in patients at risk of irreversible vision loss.
GA is an advanced and irreversible form of dAMD, characterized by the progressive loss of photoreceptors and retinal pigment epithelium. The mechanism of action of SPVN06, which aims at slowing or stopping the degeneration of cone photoreceptors, is considered by clinical experts to be particularly relevant to address the underlying pathology of GA.
Dr. Carl Regillo, MD, FACS, Chief, Retina Service at Wills Eye Hospital, Philadelphia, USA a leading expert in GA and GA Clinical Advisory Board member of SparingVision, commented: “The mechanism of action of SPVN06 is well suited to address the underlying biology of GA given the death of photoreceptors associated with the disease. The neuroprotective effect provided by RdCVF combined with the potent antioxidants provided by RdCVFl represents a totally novel approach that is clearly differentiated from current therapies regulating the complement cascade, and I look forward to seeing the start of this new clinical trial.”
Stéphane Boissel, CEO of SparingVision, also commented: “The FDA’s support of our strategy to expand into GA is a strong validation of our disease-independent, pipeline-in-a-product approach. By leveraging the data generated from RP, we can move with speed and confidence into a second indication, broadening the impact of our lead gene therapy program. Patients with GA urgently need treatment options that deliver real functional benefits with a better risk profile than existing therapies – we believe SPVN06 could be that breakthrough.”
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 a 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 a 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 disease utilizing CRISPR-Cas9 technology.
SparingVision is a spin-off from the Paris Vision Institute and backed by high-quality investors including 4BIO Capital, Adbio Partners, Bpifrance, Retinal Degeneration Fund, the venture arm of the Foundation Fighting Blindness, Fondation Voir & Entendre, Intellia Therapeutics, UPMC Enterprises, Jeito Capital and Ysios Capital.
Visit www.sparingvision.com for more and follow us on LinkedIn and X @SparingVision
About SPVN06
SPVN06 is a proprietary, mutation-agnostic, AAV vector based investigational gene therapy approach comprised of one neurotrophic factor (Rod derived Cone Viability Factor, RdCVF) and one enzyme reducing oxidative stress (Rod derived Cone Viability Factor Long form, RdCVFL). Acting synergistically, RdCVF and RdCVFL 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 an estimated 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, notably Geographic Atrophy (GA) secondary to dry Age-related Macular Degeneration (AMD). SPVN06 is the result of world-leading ophthalmology research by SparingVision founders José-Alain Sahel and Thierry Léveillard at the Paris Vision Institute.
About Geographic Atrophy
Geographic atrophy (GA) is an advanced and irreversible form of dry age-related macular degeneration (dAMD), a progressive retinal disease that affects the macula and leads to severe visual impairment. GA is characterized by the gradual loss of photoreceptors and retinal pigment epithelium (RPE), ultimately resulting in central vision loss. The disease is associated with secondary cone dysfunction caused by reduced trophic support following rod degeneration, as well as increased oxidative stress in retinal cells. There are currently no approved treatments that directly protect photoreceptors or improve visual function in GA.
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