Tough times for CRISPR startups

Spotlight Therapeutics, a startup launched to develop a new class of in vivo gene editing therapies without the need for viral vectors or nanoparticle delivery, has shut down after disappointing preclinical results. The Bay Area biotech had raised $66 million from investors including GV (formerly Google Ventures) to deliver gene-editing biologics for undruggable disease targets by applying protein engineering to direct the nuclease to desired cell types. Spotlight was co-founded in 2018 by scientists from UC Berkeley and UCSF.

Even the first approved CRISPR–Cas9-edited gene therapy — Casgevy (exagamglogene autotemcel) for sickle cell disease — faces difficulties. Casgevy’s FDA approval in November 2023 was hailed as milestone for the technology and for co-developers CRISPR Therapeutics and Vertex Therapeutics. Soon after the approval, the biotech laid off 10% of its staff, citing challenging market conditions. In January 2025, another CRISPR gene-editing company, Intellia, reduced its workforce by a quarter and trimmed its pipeline, discontinuing its principal drug research programs on α1-antitrypsin deficiency treatment to prioritize its two most advanced therapeutics.

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