May 27, 2025

The need-to-know this morning

Rocket Pharmaceuticals said a child with Danon disease, a severe, inherited heart condition, died after receiving treatment with the company's gene therapy in a clinical trial. The cause of death is still being investigated but Rocket said the patient experienced complications due to capillary leak syndrome, possibly tied to an immune-suppressing treatment used prior to the patient receiving the Rocket gene therapy, called RP-A501. The FDA placed the study on clinical hold.
Biogen and City Therapeutics, a privately held developer of RNA-based "trigger molecules," announced a partnership to develop a drug for an undisclosed central nervous system disease. The research is still in preclinical development. City was co-founded by veteran biotech executive John Maraganore.

gene editing

Baby's custom-built CRISPR treatment revitalizes gene therapy industry

The success of a custom-built CRISPR treatment for baby KJ Muldoon — who has avoided a liver transplant thanks to a therapy that targets his unique mutation — has reenergized a struggling gene editing field, STAT's Jason Mast writes. Yet while the case shows the lifesaving potential of n-of-1 therapies, it also highlights steep challenges: limited organ targets, unsustainable costs, murky regulatory pathways, and a lack of financial incentive.

Researchers hope KJ marks a turning point toward scalable, individualized medicine, but for now, bespoke gene editing remains more proof of concept than paradigm shift.

"This study highlights the imperative society now faces: science can benefit many patients who would otherwise suffer enormously or die young from serious genetic diseases," David Liu, the Broad Institute biochemist who originally designed the gene editing system used to treat KJ, told STAT. "So we have an obligation to minimize as many manmade barriers as possible, including economic challenges, that separate the science from these patients."

fundraisING

GlycoEra's global jobs underscore U.S. brain drain

From my colleague Allison DeAngelis: Just a few years ago, the chief executives at ex-U.S. biotech companies were swarming into Boston, opening offices near the biotech hub of Kendall Square. Well, the American political landscape and attacks on the scientific community have quieted that trend. And at least one biotech company is finding that their offices abroad are nabbing attention.

GlycoEra is fresh off of a $130 million fundraise, announced this morning, and has been recruiting clinical development experts as it prepares to launch its first clinical trial later this year. And it's finding that its dual locations in Massachusetts and Switzerland has become a draw. Job seekers, watching headlines pile up of new Trump administration policies, appreciate the option to relocate abroad, CEO Ganesh Kaundinya told STAT. Applicants who currently live outside of the U.S., meanwhile, are hesitating at the idea of moving stateside.

The torrent of federal funding, regulatory, and socioeconomic policy shifts have stirred concern. Some fear that it will cause a brain drain in the U.S., which might undermine the country's dominance in the sciences. So far, it's been more opaque just how the political landscape will affect drug industry recruiting and hiring.

GlycoEra is part of a wave of new autoimmune drug developers. The company is developing protein degraders that target IgG4 autoantibodies

gene editing

Unofficial ethics summit reframes global editing debate

Human dignity came to the forefront of an international genome editing summit in Cambridge, Mass. It brought together top gene-editing researchers, ethicists, artists, and policymakers who ultimately called for a global charter meant to guide emerging biotechnologies so they benefit the public good.

"Every time people feel that a technological frontier is breaking, there is this kind of excitement and suddenly the whole world is supposed to organize itself around this thing," Sheila Jasanoff, co-founder and director of the Global Observatory for Genome Editing, told STAT.

The first meeting of this ilk was in 2015, after the first reports that researchers in China were gene-editing non-viable human embryos. Last week's was the fourth, and though unofficial, it probed the value systems behind genetic innovation — raising urgent questions about power, governance, and equity in an era of geopolitical unrest and politicized science.

monoclonal antibodies

Prothena scraps birtamimab after trial failure

Ireland-based Prothena is halting development of birtamimab after the experimental antibody therapy failed to meet primary or secondary endpoints in a Phase 3 trial for AL amyloidosis. The company will shut down the open-label extension, it said in a release, and slash operating expenses — which will include a substantial workforce reduction. Shares dropped 24% in post-market activity Friday, to $5.01.

"This is not the outcome that we expected, and we are surprised and disappointed by these results for patients, their families and caregivers, and for the entire AL amyloidosis community," Prothena CEO Gene Kinney said in a statement. "With these results, we believe that the most appropriate action is to discontinue all development of birtamimab."

biohacking

Biohacking can't beat the randomness of biology

The booming biohacking movement — with all its obsessions with glucose monitors, supplements, and granular data — sells a dangerous illusion, internal medicine doctor and Atlas Venture entrepreneur-in-residence Alex Harding argues in a STAT First Opinion. It's the naive idea that we can control our health outcomes like programmers debugging code.

Biology is chaos, he writes. Even with pristine diets and disciplined regimens, disease often strikes due to randomness — genetic mutations, immune misfires, sheer bad luck.

"There is a growing industry of people and companies selling biohacking advice, tracking devices, and supplements," he writes. "In reality, they may be selling people guilt."