November 13, 2025

The need-to-know this morning

Day One Pharmaceuticals is acquiring Mersana Therapeutics in a deal valued at $129 million, and up to $285 million if certain post-close milestones are met. Day One markets a treatment for pediatric brain tumors, while Mersana is developing antibody-drug conjugates for cancer.

PERSONALIZED MEDICINE

FDA outlines pathway for bespoke gene editing

FDA Commissioner Marty Makary and biologics chief Vinay Prasad have sketched out the agency's first real blueprint for approving personalized gene-editing therapies, STAT's Jason Mast reports. It's a shift that could transform last year's one-off miracle for "Baby KJ" into a scalable model for ultra-rare diseases.

"Nearly 30 years after the sequencing of the human genome, bespoke therapies are close to reality," Makary and Prasad wrote in the New England Journal of Medicine.

Their proposed "plausible mechanism pathway" would allow companies or academic groups to treat a handful of patients with custom CRISPR editors or other tools and then win a broader authorization to keep crafting individualized versions for new mutations, bypassing traditional lab and animal testing.

The move was welcomed by gene-editing pioneers and rare-disease advocates — though thorny questions around manufacturing quality, guardrails, and payer willingness to cover million-dollar one-off treatments still loom large.

longevity

AbbVie ends pact with Google-backed Calico

AbbVie is pulling the plug on its decade-long Calico partnership, STAT's Damian Garde reports. The move underscores the ways in which AbbVie is steering away from small molecules and toward pricier, more complex biologics, RNA treatments, and cell therapies.

The breakup severs a $1.5 billion longevity alliance that never produced meaningful results — Calico's lead AbbVie-partnered ALS drug failed earlier this year — and follows the quiet exit of AbbVie's longtime chemistry chief, Phil Kym, who recently resurfaced at Calico.

AbbVie also laid off chemists working on discovering new drugs. The company's cuts in small-molecule research will affect more than 100 people, according to a person familiar with the matter.

gene therapy

Uniqure CEO blasts FDA turmoil behind closed doors

At a private dinner held as part of the Guggenheim Securities conference in Boston, Uniqure CEO Matt Kapusta vented to investors about mounting dysfunction inside the FDA, STAT's Adam Feuerstein writes. His frustration comes just a week after regulators told the company its clinical data are no longer adequate to support a submission of its gene therapy for Huntington's disease.

Kapusta also lamented the loss of FDA scientists and review staff, particularly those with deep subject matter expertise necessary to evaluate complicated therapies like gene therapies.

"Matt was more critical of the FDA than he's been in public," one of the dinner attendees told Adam. "He's clearly exasperated by the entire situation, but at the same time, he knows he can't antagonize the FDA too much, or muddy that relationship."

patents

USPTO policies may threaten patent challenges

The Patent and Trademark Office is rolling out policies that officials say will stabilize the patent system — but critics (including some drugmakers) argue they'll do the opposite, STAT's Ed Silverman writes.

One policy gives the agency director carte blanche to decide whether a contested patent should be challenged at all, rather than rely on an established agency procedure called inter partes reviews, or IPRs. IPRs rely on a patent office appeals board, a cheaper and faster way for generic rivals to challenge and invalidate patents than doing so in federal court.

"The PTO's proposed changes threaten to dismantle this vital mechanism, prolonging monopolies and driving up drug prices for patients and payers alike," the trade group Association for Accessible Medicines told STAT.

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