When it comes to genome editing, it seems there’s never a dull moment.
The CRISPR community is still buzzing about the recent milestone achieved by Intellia Therapeutics and Regeneron Pharmaceuticals when they reported the first positive clinical data on in vivo CRISPR genome editing in humans. The data on patients with with transthyretin amyloidosis (ATTR), published in The New England Journal of Medicine, appears to support the broad application of in vivo genome editing to treat a host of other rare and perhaps common diseases.
More recently, David R. Liu, PhD, of Harvard and the Broad Institute, successfully spun out Prime Medicine from his lab. The company, which applies a “search and replace” approach to gene editing, emerged from stealth mode earlier this month by raising more than $300 million in Series A financing.
On this episode of “GEN Edge Live”, three leading analysts who keenly follow developments within genome editing shared their insights on the significance of the latest CRISPR milestone, and discussed the opportunities and challenges arising within genome editing and related technologies.
Joining us on “GEN Edge Live” were: Michelle Gilson, a managing director with Canaccord Genuity focused on biotechnology; her colleague Arlinda Lee, PhD, a Canaccord Genuity analyst; and Geulah Livshits, PhD, senior research analyst with Chardan covering biotech companies, with a focus on gene editing and oncology.
Gilson, Lee, and Livshits assess the state of genome editing and its readiness to fulfill years of promise—sometimes bordering on hype—about delivering curative treatments for a range of diseases.
The three analysts also weigh in on the development of startups applying technologies beyond CRISPR, such as base editing and prime editing, as well as wave of the genome editing companies that have gone public in recent months. Caribou Biosciences, whose co-founders include Nobel laureate and CRISPR co-discoverer Jennifer Doudna, PhD, filed for an initial public offering (IPO) four months after raising $115 million in Series C financing. And in June, Graphite Bio raised $273.7 million in gross proceeds while Verve Therapeutics garnered $306.7 in gross proceeds through IPOs.