Biotech, in brief

On 1 May the US drug regulator approved Veppanu, the first medicine that destroys a protein instead of blocking it. [2][1]

It was made by the biotech Arvinas with Pfizer, and it treats a form of advanced breast cancer after hormone treatment has stopped working. [2][1]

In its final-stage trial it cut the risk of the cancer worsening or the patient dying by 43%, against an older drug called fulvestrant. [1]

The drug has two ends. One end grips the faulty protein. The other grips the machinery a cell uses to shred proteins it has finished with. [1]

Holding the two together is the whole trick. The drug breaks nothing itself. The cell does the destroying. [1]

Craig Crews, a pharmacologist at Yale University in the United States, first proposed the idea more than twenty years ago. [1]

What he found was that two proteins that never evolved to touch each other can be forced to. That opened up the possibility of using induced proximity in many different ways, he says. [1]

More than 30 of these protein-destroying drugs are now being tested in people. [1]

Johnson & Johnson, one of the world's largest healthcare companies, paid $3.05bn in November 2025 for Halda Therapeutics, a Yale spin-out with no approved drug and one early trial. [1]

Halda's drug destroys nothing. It handcuffs a protein that prostate tumours carry in excess to BRD4, a protein cancer cells need to survive, and the pair kills the cell. [1]

In that early trial, 59% of men who took it for at least six weeks saw a marker of their prostate cancer fall by half or more. [1]

Thalidomide and drugs like it were later found to work this way by accident, as a glue between two proteins that would not otherwise meet. [1]

Nearly all of the molecular glues we know about were discovered by serendipity, says George Burslem, a biochemist at the University of Pennsylvania. Finding them on purpose is the open problem. [1]

Why it matters. Watch whether one of these drugs works outside cancer. An asthma drug from Kymera holds a US fast-track label, and that is the test of whether this is a cancer trick or a new way to make medicines. [1]

A new cut of trial data showed that ivonescimab, made by Akeso in China and licensed to Summit, helped people with lung cancer live longer than Merck's Keytruda. One analyst expects the full figures to show a 24% lower risk of death. Summit shares rose as much as 14%. [3]

Why it matters. Drugs like Keytruda transformed cancer care over the past decade, and drugmakers have largely failed to top them despite much trying. [3]

In a 438-patient final-stage trial published on 1 September, ivonescimab plus chemotherapy held lung cancer back for 6.8 months against 4.4 on chemotherapy alone. People lived a median 16.8 months against 14.0, a gap the trial could not separate from chance. [4]

Why it matters. Two results on the same drug in one week, pointing different ways on the only question that finally matters.

GSK agreed on 3 September to pay Hutchmed $110m now and up to $1.19bn later for most rights to HMPL-A830. It is an antibody that finds the protein EGFR and carries a small drug that blocks another, KRAS. [5]

Why it matters. Both proteins drive common cancers, and a great many KRAS-driven tumours still have no safe, lasting treatment. [5]

Researchers at the University of Oslo scanned healthy older adults for nearly twenty years, then looked back. Brain structure had already changed at least seven years before amyloid plaques showed up on the scan that is used to spot the disease earliest. [6]

Why it matters. Either something starts the plaques before they can be seen, or something else damages the brain first. The second would mean the drug hunt has been aimed too narrowly. [6]

Washington University researchers found that the immune cells which worsen tau damage in the brain are given their instructions by cells in lymph nodes outside it. Mice that lacked those instructing cells were protected from the damage. [7][8]

Why it matters. The harm is in the brain and the decision is made elsewhere, which is a place nobody had been treating. [8]

Stanford researchers found that ageing macrophages, the body's clean-up cells, stop clearing the roughly 100 billion worn-out white blood cells made each day. Blocking one receptor on them, called EP2, kept the brain, heart, liver, kidney and muscle of old mice more youthful. [9]

Why it matters. It points at chronic inflammation as a cause of ageing rather than a symptom, though this was mice and human cells, not people. [9]

Alumis said on 1 September that envudeucitinib failed both its main and its backup measures in a 408-person mid-stage lupus trial. The company is taking it into final-stage testing anyway, on the strength of one group of patients picked out in advance. Its shares fell 54%. [10]

Why it matters. About 60% of the trial's patients were in that group, against the 80% seen in other trials, and the company plans to enrol mainly those people next time. [10]

uniQure asked the US drug regulator on 2 September to approve its gene therapy for Huntington's disease, an inherited and fatal brain disorder with no treatment for its cause. [11]

Why it matters. The regulator had earlier refused; the filing follows its reversal. [11]

US overdose deaths dropped 27% in 2024 and fell again in 2025. Scotland was down 13% and Canada 9%. A Commonwealth Fund report published on 3 September asked officials in more than two dozen places, and every one said no single thing did it. [12]

Why it matters. The things named repeatedly were unglamorous: wider naloxone supply, treatment in and around prisons, and better data. [12]

Fifteen people with stubbornly high blood fats got a single infusion of CTX310, which uses CRISPR to switch off a liver gene called ANGPTL3. A year later the effect held: at the top dose, triglycerides down 48% and LDL cholesterol down 53%. [13][14]

Why it matters. It is a safety study of fifteen people, and switching a gene off permanently is not something you can undo if it turns out to be a mistake. [13]

Milvexian, from Bristol Myers Squibb and Johnson & Johnson, was meant to stop dangerous clots without the bleeding that clot drugs cause. In about 14,000 patients treated within a week of a sudden blockage in a heart artery, it did not increase serious bleeding, and it did not cut heart attacks, strokes or deaths either: 5.4% against 5.1%. [13]

Why it matters. The trial was stopped in November 2025 once it was clear it would not work; the full data explains why. Two more trials of the same drug are still running. [13]

Ten people with heart failure got SRD-002, a one-off gene therapy that adds a copy of the gene for SERCA2a, a calcium pump that lets heart muscle relax after each beat. At a year, eight of ten met a pre-set target for normal filling pressure. [13]

Why it matters. The same target and delivery method failed a 250-patient trial in a different kind of heart failure, so ten patients with no comparison group settles nothing. [13]

AstraZeneca's AZD5462 copies relaxin, a hormone that widens blood vessels during pregnancy. In 375 heart failure patients it significantly lowered the resistance the heart pumps against, in those whose pumping was only mildly reduced. Another measure narrowly missed. [13]

Why it matters. An injected version of the same hormone failed a final-stage trial years ago; this is the same biology in a tablet. [13]

Superluminal Medicines raised $60m to develop a daily tablet aimed at MC4R, a receptor that controls appetite and how much energy the body burns. Human testing should start by the end of the year. Nvidia and Eli Lilly joined the round. [15]

Why it matters. The existing drug for these rare genetic conditions is an injection, and several rivals aimed at the same problem have failed or been paused. [15]

Researchers used CRISPR to switch on genes that reprogram glioblastoma cells into cells resembling dendritic cells, the immune system's messengers, so the body attacks the tumour. Glioblastoma is the deadliest primary brain tumour and survival after it returns is very poor. [16]

Why it matters. It changes the tumour into something that calls for help, rather than sending a drug in after it.

China's State Council Order No. 818, a single framework for new biomedical technologies, came into force on 1 May. A Nature comment on 1 September set out how it works, noting the case of a six-year-old girl who died last year after an experimental gene-editing treatment aimed at her brain, in Shanghai. [17]

Why it matters. The death happened before the rules existed, and is the reason the authors say the rules are needed. [17]

Peking University researchers measured the stiffness of the part of the mouse hippocampus that makes new brain cells. It stiffened early in Alzheimer's mice, and stiffness alone was enough to stop new cells forming. An enzyme that softened it again brought them back. [18]

Why it matters. The same stiffening was found in brain tissue from people who had died with Alzheimer's, which makes it more than a mouse finding. [18]

Researchers put damaged tau into the locus coeruleus, a small brain area that supplies the chemical noradrenaline, in mice. Three months later the mice were anxious and repetitive, and had fewer sleep spindles, the short bursts of brain activity that punctuate sleep. No brain cells had died. [19]

Why it matters. People with Alzheimer's often become anxious or agitated years before they start forgetting, and this is where that may begin. [19]

A framework published on 3 September tries to make automated reading of Alzheimer's brain scans more trustworthy, by tying its answer to named brain regions rather than reading the image as a whole. [20]

Why it matters. The stages of the disease look alike on a scan, which is why these tools carry little confidence and travel badly to new data. [20]

The US House of Representatives voted 370 to 48 on 1 September for a stopgap bill funding the US federal government until 11 December. It temporarily blocks a plan by the Trump administration to give political appointees more say over federal research grants. [21]

Why it matters. The block expires on the same day the money does. [21]

Researchers at the US National Institutes of Health looking for a way to prevent malaria may instead have found a way to treat alpha-gal syndrome, the allergy to red meat caused by tick bites. The paper appeared in the Journal of Clinical Investigation. [21]

Why it matters. Nobody was looking for it, which is how a fair number of treatments arrive.

A review published on 2 September set out where optogenetic sight restoration stands. Viruses carry light-sensitive proteins into the retinal nerve cells that survive after the light-detecting cells have died, and the clinical pipeline is growing. [22]

Why it matters. It does not care which gene caused the blindness, which matters once there are no light-detecting cells left to fix. [22]

A reference review published on 3 September sets out what is known about varicella zoster virus, which infects more than 90% of people worldwide. It causes chickenpox, then hides in nerve cells for life, and can wake up decades later as shingles. [23]

Why it matters. Waking up is triggered by the immune system weakening with age or illness, which is why shingles is a disease of later life. [23]

Dementia affects more than 55 million people and is expected to triple by 2050. About 20% of cases are vascular dementia, caused by injured blood vessels, a leaking barrier around the brain and inflammation together. [24]

Why it matters. It is the second most common cause after Alzheimer's, and it is caused by things that damage blood vessels anywhere. [24]

A review published on 1 September finds obesity across life is linked to cognitive decline, with obesity in middle age carrying a particularly high risk. It acts through the hypothalamus and through inflammation and blood-vessel damage in the hippocampus. [25]

Why it matters. The measured risk is not evenly spread across a life; it is concentrated in the middle of it. [25]

A review on 2 September argues sleep and chronic stress feed Alzheimer's directly. Sleep runs the clearing systems that remove waste protein from the brain, and those systems are impaired in the disease; long-term stress adds inflammation. [26]

Why it matters. Both are things that can change, unlike the genes that make up most of the known risk. [26]

A review on 3 September finds people with autoimmune joint diseases catch more preventable infections, because of the disease, the drugs that damp their immune systems, and age. Their coverage for routine flu and pneumonia vaccines remains low. [27]

Why it matters. The same drugs that raise their risk also make a vaccine work less well, which is why the timing has to be planned. [27]

A review on 1 September collects the ways researchers try to make regulatory T cells, the immune cells that hold the rest back and stop the body attacking itself. The methods run from feeding them signals to engineering them. [28]

Why it matters. Most autoimmune treatment blunts the whole immune system; this would strengthen the part that already says stop. [28]

A review on 2 September finds that immune checkpoint proteins, the off-switches cancer drugs release to let the immune system attack tumours, also appear to shape obesity, diabetes and thyroid disease. [29]

Why it matters. Existing drugs already move these switches, which is why anyone is looking. [29]

A review published on 4 September sets out what is known about itaconate, a chemical immune cells make that acts as a brake on inflammation and on damage from oxygen. It shows up across infections, autoimmune disease and cancer. [30]

Why it matters. It is made by the cell's own energy machinery, which ties how a cell burns fuel to how hard it inflames. [30]

A paper in a Lancet journal on 3 September reports that recognition of the 2026 Bundibugyo virus outbreak was delayed by political circumstances and by problems getting people and equipment there. It describes qualifying diagnostic tests while the outbreak was still running. [31]

Why it matters. The tests were still being qualified while the outbreak was running. [31]

In an interview published on 3 September, Anthony Fauci, who ran the US government's infectious disease institute for decades, addressed the claim that many vaccines overwhelm a child's immune system. That is just scientifically not the case, he said. [32]

Why it matters. The claim is central to current US vaccine policy arguments, and he is the person it is usually aimed at.

Researchers testing rare human gene variants in Xenopus frogs identified ARHGAP10 as a gene behind congenital heart disease and ciliopathies, disorders of the tiny hairs cells use to sense and move fluid. [33]

Why it matters. It shows a route from a rare variant in one family to a mechanism, which is the step that usually stalls. [33]

A Cell Metabolism piece on 1 September sets out what an age-reversal claim should have to show. It notes that several ordinary things reverse multiple aspects of ageing, including rapamycin in laboratory mice and regular exercise. [34]

Why it matters. The bar matters commercially: without one, every intervention that improves anything can be sold as reversal. [34]

A viewpoint in a JAMA journal asks how doctors should use the new amyloid-clearing Alzheimer's drugs in patients who are likely to have a second brain disease alongside it. [35]

Why it matters. Most older brains carry more than one problem, and the trials were run in people chosen for having one. [35]