CRISPR-based Spotlight Therapeutics has shut down, former CEO Mary Haak-Frendscho, Ph.D., confirmed with Fierce Biotech.
The San Francisco-based company housed a cell-specific in vivo gene editing platform that was designed to have a similar structure to an antibody-drug conjugate but with a CRISPR-Cas payload. If successful, the biotech’s CRISPR therapies could’ve been delivered directly, skirting the need to use lipid nanoparticles or viral vectors.
Unfortunately, a therapy from Spotlight never made it to center stage.
“We explored a number of therapeutic areas beyond reprogramming the tumor microenvironment,” Haak-Frendscho told Fierce Biotech. “Spotlight's original IO approach was immunologic: knocking out ADAR in CD11a (+) immune cells in solid tumors. When seeking alternative applications, a key consideration was that the platform was not sufficiently matured to support systemic administration, which requires significantly greater in vivo stability.”
The company then determined ophthalmology would the best area in which to apply its technology after considering opportunities that would be conducive to local administration, plus areas of substantial unmet patient need with a compelling market opportunity, the CEO said.
In a preclinical mouse study, the company’s asset edited only about 7% of retinal cells when injected directly into the eye, according to a bioRxiv preprint published Dec. 31, 2024. When administered to minipigs, the rate dropped below 1%, according to the findings.
The biotech launched in 2020 with money from Alphabet’s venture capital arm GV (Google Ventures). The company was formed by Alex Marson, M.D., Ph.D., director of the genomic immunology institute at the University of California, San Francisco; Patrick Hsu, Ph.D., co-founder of the Arc Institute and assistant professor at the University of California, Berkeley; and Jacob Corn, a professor at ETH Zürich.
Both Marson and Corn declined to comment, while Hsu did not respond by time of publication.
Since 2025 began, at least three other biotechs—Kojin Therapeutics, Viracta Therapeutics and Velia Therapeutics—have closed their doors.