Legend Biotech emerged as a rare market winner

Shares of Legend Biotech soared over 40% yesterday after the company disclosed early data on its in vivo CAR-T therapy that showed promise in Non-Hodgkin's lymphoma.

In an ongoing Phase 1 trial, all six patients so far on the highest dose responded and five experienced a complete response, after a median follow-up of 2.2 months.

Traditional CAR-T therapies are manufactured through an arduous process in which cells are removed, genetically altered, and then infused back into the body. In vivo therapies would bypass that process, since cells would be transformed within the body.

Legend was an outlier among biotech stocks yesterday. Shares of companies including Abivax, Celcuity, and Praxis Precision Medicines tanked as investors reacted to new data. The XBI fell over 4%.

regulation

FDA proposes new guidance for gene therapy developers

From my colleague Lizzy Lawrence: The FDA yesterday introduced draft guidance explaining how gene therapy companies can leverage existing knowledge in their drug approval applications. That existing knowledge might be data from a specific manufacturing platform, and other public information. The goal is to increase efficiency and advance innovation.

Carl June, a cell therapy researcher at the University of Pennsylvania, said the guidance is a step in the right direction but the impact will depend on how reviewers interpret it. Fyodor Urnov, a gene-editing researcher at the University of California, Berkeley, said the guidance is important and timely.

“We need to stop redoing studies that, so to speak, recertify the pizza oven when all we changed is one topping,” Urnov said.

financing

Longevity startup raises its third round in a year

Biotech firm NewLimit announced a staggering $435 million Series C round as it prepares to launch its first clinical trial of a liver medicine.

This is the third time in the past year that the company has disclosed a financing — first, a $130 million Series B round in May 2025, followed by another $45 million in October.

NewLimit, co-founded by Coinbase CEO Brian Armstrong, is among the startups focused on aging-related diseases that have captured the attention of Silicon Valley investors.

Read more from STAT's Allison DeAngelis.

pharma

Lilly signs deal for RNA-editing kidney drugs

Ascidian Therapeutics said today that it will license to Eli Lilly a class of drugs called RNA exon editors for undisclosed kidney disease targets.

Ascidian is eligible to receive up to $1.9 billion and will lead discovery and certain preclinical work, while Lilly will be in charge of remaining development and commercialization.

RNA exon editors are designed to trick cells into replacing large sections of glitchy RNA with genetic instructions for making functional proteins.

Lilly, flush with cash from its booming GLP-1 business, has been on a spree of acquisitions and licensing deals. It's shown particular interest in genetic medicines.

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