SparingVision raises $52M to kick off long journey for a next-gen gene therapy that goes much, much broader than Luxturna
Until Spark Therapeutics’ pioneering gene therapy, Luxturna, came along, patients with retinitis pigmentosa had few treatment options. Even after it was approved, though, the majority were left with the exact same options.
Because it’s targeting mutations in a specific gene known as RPE65, Luxturna can only address 2 to 3% of the entire RP population, Stephane Boissel told Endpoints News.
Boissel is the newly-minted CEO of SparingVision, a French biotech co-founded by José-Alain Sahel and Thierry Léveillard at the Institut de la Vision. They have their sight set on a new kind of AAV construct — a next-generation gene therapy if you will — that can treat all patients of RP independent of genetic mutations.
And they have $52 million-plus (€44.5 million) to realize that vision steadily over the next four or five years, with plans to complete both first- and second-in-human trials.
What the founding scientists pinpointed, instead of a gene, was a neurotrophic factor secreted by rod photoreceptors that appears to protect the cones, which explains why RP patients see their cones die even in the absence of genetic abnormalities. By wrapping RdCVF, or rod-derived cone viability factor, and an oxidative stress reducing enzyme into a viral vector, they came up with SPVN06 in hopes of replacing one key function that RP patients lose alongside their rods.
It won’t reverse the disease, but SparingVision is hoping it can slow or stop its progression.
The approach paves a path that reaches all 2 million RP patients — a kind of “medium rare” indication and broader application of gene therapy favored by 4BIO Capital, said director of investment Owen Smith. 4BIO co-led the round with UPMC Enterprises, the venture arm of Pittsburgh-based health system UPMC, where Sahel chairs the Department of Ophthalmology.
“One of the alternatives is developing a Luxturna in every single genotype, and we haven’t even fully characterized the disease on a genetic level,” he said.
Currently focused on a single asset, SparingVision now operates virtually without labs or manufacturing — everything is outsourced and will be for a while — with all 12 of its staffers currently in France. Having recently moved to Paris from the Bay Area, where he was working on corporate strategy for Sangamo following the sale of TxCell two years ago, Boissel also plans to build a US-based team to start preparing for that second-in-human efficacy trial.
“It’s easier to find talents who have done it in the US than in Europe, and I wanted people who have done it,” he said. “So our CMO, our CTO, our head of translational science will be based in the US.”
He envisions recruiting a lean team of very senior people, building out to 20 or 25 assuming SparingVision sticks only to SPVN06. With that program alone there’s potential to go into dry AMD, where the neuroprotective mechanism could be relevant. There is also a possibility of bringing in new products or technologies that would fit under genomic medicines for the eye, from gene replacement to editing to regulation. But it’s early days, and Boissel said the syndicate — which also features Jeito Capital, Ysios Capital, Bpifrance and Foundation Fighting Blindness — isn’t expecting a quick flip to the market.
It’s taken the company four years to get to where they are. The first clinical trial should begin next year in Europe, while the efficacy trial is planned for 2023.
“That is a very unique setting in a world where things are very often going too fast,” he said.