Spotlight Therapeutics: In aiming to bypass CRISPR's delivery issues, one startup eyes the gene editing Spotlight

Much hype has been granted toward a cohort of CRISPR gene editing biotechs, including the eponymous startup founded by Nobel Prize winner Emmanuelle Charpentier. But one such company has stayed relatively quiet, albeit for a subtle series A emergence in November 2020. Now, the GV-backed startup is ready for the spotlight with a new round of financing. Yes, the biotech is named Spotlight Therapeutics, and the 28-employee company wants to skirt the delivery issues ingrained in the typical CRISPR path of viral and nanoparticle transportation. The biotech will have to create its own spotlight to edge into the gene-editing space via in vivo cell-targeted delivery. "The possibility when you leave behind packaging, it opens up all kinds of new doors in terms of limitations, and going to cell-selective system allows us to open up many new applications beyond what's possible today," Mary Haak-Frendscho, president and CEO, told Endpoints News. The 3.5-year-old biotech envisions a "step change" in the way gene editing is delivered, the CEO said, noting Spotlight plans to use antibodies to point gene-editing molecules toward desired cell types in vivo. The biotech wants to go the route of a "centrally manufactured and distributed biologic," which would eliminate many hurdles in the development process, Haak-Frendscho added. But the nascency of the technology put a wrinkle in securing funds, the CEO admitted, noting the industry's funding environment overall has been no walk in the park. "We get a lot of very positive reception but because this is so new, there are many folks that are also looking to see more de-risking, let's put it that way." First up on the R&D path is immuno-oncology. A development candidate will come into view in about a year and then a human trial entry "a little over a year beyond that," the CEO said. Further down the pipeline is work in ophthalmologic and hemoglobin areas, she said. The recent patent disputes over CRISPR/Cas9 gene editing shouldn't addle or impede Spotlight's work. "We've done a lot of our work with spike Cas9; however, we have also demonstrated we can put in Cas12a, we've done Cas nucleases from a lot of other bacterial species and we've started working with base editors, so we can swap out those modules to make bespoke molecules that are fit for purpose," Haak-Frendscho explained. The Hayward, CA biotech will expand its working space this year, add about 10 employees and look to ink biopharma partnerships. Haak-Frendscho has led the company since January 2019, after a two-year stint as a venture partner at Versant Ventures, where she led the VC shop's discovery unit to help incubate companies like Repare Therapeutics. GordonMD Global Investments and EPIQ Capital Group co-led the Series B, which came in at $36.5 million, a slight upgrade to the 2020 $30 million series A. The series A was meant to get the company to 2022, Haak-Frendscho told Endpoints at the time, and she delivered on that promise. Magnetic Ventures, GV, Emerson Collective and other investors also backed the company. Alongside the financing, Mary Janatpour was promoted to chief scientific officer.