From The Editor | January 3, 2023

The Next Big Thing

By Ben Comer, Chief Editor, Life Science Leader

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This month, a large swath of the biopharma industry will pack into the Westin St. Francis in San Francisco for the 41st annual J.P. Morgan Healthcare conference. JPM is expecting 400 public and private companies to give presentations to more than 8,000 attendees this year, according to event organizers, and that doesn’t count the numerous company executives, interested parties, and hangers-on joining the melee in an uncredentialed capacity.

In my experience, the conference is a starting gun for the new year; it helps to set the industry agenda by highlighting business topics, clinical advances, and therapeutic areas in vogue, while providing an in-person platform for introducing new executive leaders or catching up with biopharma’s old guard. The question underlying much of the conference activity boils down to this: What is the next big thing?

A strong case can be made for genetic medicines, which are proliferating across industry development pipelines and expanding to target conditions beyond cancer and rare diseases. At the Galien Forum in October, George Yancopoulos, president and chief science officer at Regeneron, noted that biologics and antibodies represented the most exciting developments over the last 10 years, but that “genetic medicines are the next thing.” This month’s cover story explores how Cofounder and CEO Sekar Kathiresan secured the funding and technology to launch Verve Therapeutics, a “base editing” gene therapy company focused on a big goal: preventing heart attacks with a one-time treatment that turns off a gene associated with high LDL cholesterol levels.

Despite productive interactions with the FDA, Verve’s lead candidate IND application was placed on clinical hold in early November, even as human trials testing the same product were greenlit previously by regulators in the U.K. and New Zealand. Beam Therapeutics, which developed the base editing technology in-licensed by Verve for its lead candidate, also received an IND clinical hold on one of its development candidates in August, an allogeneic CAR-T therapy targeting blood cancers. Delivering the next big thing in medicine is never easy — or without complications.

Peter Marks, CBER director and effectively the FDA’s chief regulator of gene and cell therapies, told me in an interview (page 18) that aside from managing an inordinate amount of vaccine applications this year, advancing gene and cell therapies is a top priority in 2023. In truth, the biopharma industry is too broad and diverse for a single next big thing; every one of this year’s JPM presenters will be prepared to make the case for his or her company’s strategy and pipeline. If you spot me squeezing through the hallways of the Westin St. Francis, say hello, and tell me about your next big thing.