January 9, 2026

The need-to-know this morning

UniQure said this morning a meeting with the FDA had been scheduled to discuss the status of its application for its Huntington's disease gene therapy, though it did not specify when the meeting would be held. The agency recently raised doubts about the data package underlying the therapy, which has been seen as a promising treatment.

rare disease

Aurora bets on personalized CRISPR medicines

Born from a 2024 pizza-fueled conversation in Berkeley, a new startup focused on personalized CRISPR treatments for rare and mutation-driven diseases has just launched with $16 million from Menlo Ventures. Co-founded by CRISPR pioneer Jennifer Doudna and gene-editing researcher Fyodor Urnov, the 11-person startup Aurora Therapeutics is capitalizing on momentum from last year's rapid, bespoke treatment of infant KJ Muldoon.

New signals from the FDA outline a potential approval pathway for custom gene editors via "basket trials," STAT's Jason Mast and Allison DeAngelis write.

Aurora plans to start with phenylketonuria, a rare liver disease driven by as many as 2,000 mutations, even as academic groups and other biotechs explore similar ground. The company says recent regulatory clarity and manufacturing advances make the model feasible — but it will still need more capital, and proof of profitability, to turn personalized gene editing into a sustainable treatment modality.

"The motto is let there be light," Urnov said. "We are passionate about charting a path others can use."

glp-1 drugs

Lilly tests Zepbound add-on in psoriatic arthritis

Eli Lilly said a trial combining its weight loss drug Zepbound with the autoimmune therapy Taltz helped obese patients with psoriatic arthritis more than Taltz alone, hinting that GLP-1 drugs could have anti-inflammatory benefits beyond weight loss.

In an open-label Phase 3 study, 32% of patients on the combination achieved both meaningful symptom relief and at least 10% weight loss, versus less than 1% on Taltz alone, though the primary endpoint was tilted toward the combo since Taltz does not cause weight loss.

On a more conventional measure of joint improvement, results favored the combination but were less impressive than Taltz's own pivotal trials, raising doubts about whether the data will shift prescribing amid stiff competition from rivals like UCB's Bimzelx and AbbVie's Skyrizi.

drug pricing

Appeals court blocks Trump-era 340B rebate pilot

A federal appeals court has blocked the Trump administration from moving forward with a pilot that would have shifted the 340B drug discount program from upfront price reductions to a rebate-based system, STAT's Ed Silverman writes. The court sided with hospitals that argued the government failed to consider how the pilot might harm so-called safety-net providers.

Upholding a lower court ruling, a panel of three judges said the Health Resources and Services Administration did not adequately weigh hospitals' reliance on the existing 340B structure, or how these rebates could create new administrative hurdles and financial strain.

The halted pilot was initially planned to begin Jan 1.

The Readout Loud

JPM to bring more deals and happy CEOs

What news happened over the holidays? Why will pharma CEOs be greeted warmly in San Francisco next week? And who will buy Revolution Medicines?

We discuss all that and more on this week's episode of "The Readout LOUD," STAT's weekly biotech podcast. We bring on former co-host Damian Garde, who's now a reporter at large at STAT, to preview the upcoming J.P. Morgan Healthcare Conference.

We also discuss the approval of Novo Nordisk's Wegovy pill, the federal government's move to slash the number of recommended pediatric immunizations, and the resurgence of M&A deals.

Listen here.

C-Suite

Former NIH genomics director Eric Green to join Illumina as CMO

From my colleague Jonathan Wosen: DNA-sequencing juggernaut Illumina announced yesterday that Eric Green, former head of the National Human Genome Research Institute, will serve as its next chief medical officer starting Feb. 2. The news comes nearly 10 months after Green was forced out of the National Institutes of Health last March when his appointment was not renewed.

Green was the first (though far from the last) ousted institute director, and in a subsequent interview with STAT he said he was never told why he was pushed out, and who ordered it.

His new appointment, which was first reported by Endpoints News, comes as Illumina looks to maintain control over the genomics market against its many competitors and drive the use of sequencing in clinical care.

"I am excited to join the company at a time when genomic information is becoming increasingly important in clinical care," said Green in a press release. "Illumina sits at the center of the growing omics ecosystem, and the company is uniquely positioned to help shape the next phase of genomic medicine."

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