October 16, 2025

The need-to-know this morning

Praxis Precision Medicines said its experimental treatment for essential tremor, a neurological disease that causes involuntary shaking of the limbs, achieved the primary goals of two Phase 3 studies.

gene editing

Baby KJ's gene editing sparks a scaling of ambition

At the STAT Summit in Boston yesterday, Kyle and Nicole Muldoon shared the story of how their son, Baby KJ — once facing certain death from an ultra-rare liver disorder — became the first patient treated with a bespoke CRISPR-based gene-editing therapy that partially corrected his condition, STAT's Megan Molteni writes.

Developed by teams at the University of Pennsylvania and the Children's Hospital of Philadelphia, with help from top researchers across the country, the "n-of-1" treatment transformed KJ's prognosis and galvanized a push to make such precision medicine scalable.

"We need to be able to scale this," said Karen Ahrens-Nicklas, the physician who administered KJ's treatment. "In our country, to do that, you have to have an approval so that the drug product can be reimbursed and we can actually subsidize this to be able to help patients around the country. The technology is there, we just need the infrastructure to evaluate it and deliver it."

As KJ, now a lively toddler, charmed the crowd in his tuxedo, his doctors detailed their plan to launch an FDA-approved trial covering multiple urea cycle disorders — a potential first step toward making personalized gene editing a reality for more families.

pharma

GSK distances itself from FDA leucovorin push

GSK CEO Emma Walmsley said the company's involvement in the FDA's effort to update the prescribing label for leucovorin — a decades-old drug once marketed as Wellcovorin — is purely "administrative."

Speaking at the STAT Summit, Walmsley emphasized that GSK has "no commercial interest" in the effort, STAT's Elaine Chen writes, even though it has agreed to the FDA's request to submit an application for leucovorin as a treatment for cerebrate folate deficiency, a neurological condition that can have overlapping symptoms with autism. The approval of the application will allow generic manufacturers to update their labels as well.

The move follows internal FDA tensions over the push, which some agency scientists viewed as politically driven. "I would hope that we can stay grounded in science," added John Maraganore, co-CEO of Corsera Health, on the same panel.

immunotherapy

A researcher calls for renewed investment in immunotherapy

Mount Sinai oncologist Miriam Merad, speaking at the STAT Summit, called for renewed investment in basic science as she outlined how macrophages — the body's repair cells — could transform cancer treatment.

Merad described how these immune cells, often seen aiding tumors instead of fighting them, could be reprogrammed to deliver targeted therapies directly to damaged tissues. Her lab's work aims to overcome the limits of checkpoint inhibitors and CAR-T therapies by harnessing macrophages' precision. But progress, she warned, is slowing under funding cuts that have stripped funding from NIH grants.

"We feel so close to being able to reprogram those cells," she said. "I just don't understand why we as a society would reduce funding when we are so close."

rare disease

Novo strikes $2.1 billion deal with Omeros for rare disease drug

Novo Nordisk inked a licensing pact worth up to $2.1 billion with Omeros for zaltenibart, an experimental MASP-3 inhibitor targeting rare blood and kidney disorders.

Novo will pay $340 million upfront and in near-term milestones, Reuters writes, gaining global rights to develop and commercialize the drug, now in late-stage trials for paroxysmal nocturnal hemoglobinuria, a rare inherited blood disorder in which the immune system attacks red blood cells.

Zaltenibart has shown strong safety and potential advantages over other complement pathway inhibitors. Novo plans to launch a broader development program after the deal closes in late 2025, while Omeros continues pursuing approval of its separate therapy, narsoplimab, for transplant-related complications.

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