Biotech, in brief

On Tuesday 27 October the FDA, the US drug regulator, published draft rules for gene-editing treatments built for each patient's own faulty gene. [1][2]

Gene editing changes DNA at a chosen spot inside a person's cells. A short molecule called a guide RNA steers the editing tool to that spot. [1]

Many inherited diseases come from different faults in the same gene, and some faults are found in only one patient or a handful. [1]

The draft says one licence can cover many versions of a treatment, each aimed at a different fault but sharing the same core design. [1]

A company would set limits in advance on what may change, such as the guide's sequence. The dose, the formula and the package that carries the editor into the body stay fixed. [1]

A new version inside those limits would count as a manufacturing change, not a new medicine, if lab tests predict it is safe and active. [1]

That lets a company add versions on lab data without new clinical trials, the FDA wrote. [1]

The limits themselves must still rest on data from patients. The draft does not say how large the first trials must be, and the FDA plans separate guidance on that. [1]

Rare disease groups welcomed it. A PKD Foundation official called it putting a flag in the ground. [2]

It came out on the second day of a rare disease summit run by NORD, a US patient group, where the heads of the FDA and NIH were due to speak. [2][3]

The FDA is run by an acting head, Kyle Diamantas, since its commissioner Marty Makary resigned in May. [4]

The draft is open for public comment and is not yet in force. [1]

Why it matters. The draft now takes public comment, and the FDA still owes separate guidance on how much patient data the first version needs; that second document decides how much cheaper this route really is. [1]

Artiva Biotherapeutics, a San Diego biotech, stopped work on Tuesday on AlloNK, a treatment made from donors' natural killer cells, a kind of immune cell. [5][6] It was being tested in rheumatoid arthritis and other immune diseases. [6] One patient died of brain damage that a safety review linked to fludarabine, a chemotherapy drug given first to make room for the new cells. [6][8] A second died suddenly about two months after starting, cause unknown. [5] The effects also faded faster than hoped, and Artiva is cutting 94% of its staff. [6][7]

Why it matters. Artiva is the third company to report deaths in a cell treatment for immune diseases, after Novartis and Bristol Myers Squibb. [5] Fludarabine is given before many cell treatments, so Artiva says it will publish the case for others to study. [5]

Novartis, the Swiss drugmaker, took a $1.9 billion charge on del-desiran, a drug for myotonic dystrophy type 1, a rare disease that wastes muscles. [9] The drug failed its final trial last month. [9] It came with Novartis's $12 billion purchase of Avidity Biosciences a year ago. [9] On a results call on Tuesday, chief executive Vas Narasimhan called the failure a bump. [9] He said the charge reflects the extra time and money needed for another final trial. [9]

Why it matters. Two more drugs from the same purchase are still moving: one in a final trial for another muscle disease, and one under a fast FDA review for Duchenne muscular dystrophy. [9]

The FDA approved Pfizer's talazoparib, sold as Talzenna, with the hormone-blocking drug enzalutamide on 26 October. [10] It is for prostate cancer that has spread and carries faults in genes that repair DNA, found by an approved test. [10] In a trial of 599 men, adding talazoparib cut the risk of the cancer growing or death by 52%. [10] Data on how long the men live are not yet mature. [10]

Why it matters. It is approved for men whose cancer has not yet had, or still responds to, drugs that block male hormones. [10] Australia's and Canada's regulators are reviewing it alongside the FDA. [10]

Jennifer Shuford, deputy director and chief medical officer of the CDC, the US disease-control agency, is leaving. [11][12] President Trump appointed her in April, after she ran Texas's health department, and she started in June. [11] Her last day is 12 November. [11][12] She said she wants more time with her family. [12] Sources told the Washington Post she had clashed over changes to vaccine advice. [11] Satish Pillai will act in her place. [12]

Why it matters. The CDC has lost a third of its workforce and is facing 48 measles outbreaks, the Guardian reports. [12] Only half of people in the US trust its health advice, a recent survey found. [11]

Nabilone, a drug used to ease pain in multiple sclerosis, is out of stock at wholesalers across the UK, Northern Ireland's health department said. [13] In MS the immune system attacks the coating around nerves. [13] The 1mg capsules ran short in July and the 0.25mg ones more recently, and supply is not expected until December. [13] Jill Kenning, who has taken it for 17 years, has spent most days in bed since running out. [13]

Why it matters. The department would not say why supply stopped, calling it commercially sensitive. [13] Medicines in short supply in the UK reached 157 this week, up from 107 in January, by the MIMS tracker. [13]

A US federal judge, Zahid Quraishi, refused on Monday to block Eli Lilly's adverts comparing its Zepbound with Novo Nordisk's Wegovy. [14] Novo, the Danish maker of Wegovy, said the ads leaned on an older head-to-head trial and ignored a newer, higher dose of Wegovy. [14] The judge found Wegovy prescriptions did not fall while the ads ran. [14] He noted Lilly's newer ads mention the higher dose, and he also faulted Novo's own Ozempic ads. [14]

Why it matters. The ruling only refuses an early ban, so Novo's claim that the ads mislead is still undecided. [14] Analysts expect sales of weight-loss drugs to pass $100 billion a year by 2030. [14]

Researchers asked five AI chatbots, including GPT-5.4 and Claude Sonnet 4.6, to recommend medicines for 177,120 made-up patients, in a study in Nature Health on 26 October. [15] Each case named a disease and a medicine that several approved products contain. [15] On average one product took 97.5% of the chatbots' first choices, whatever the patient's background. [15] The picks differed from what US doctors actually prescribe and often cost more. [15]

Why it matters. About a fifth of US adults already use such chatbots to look up health information, the authors note. [15] When told to prefer the cheapest or generic product, the chatbots often switched to a generic one. [15]

Kevin Strauss, a doctor who treats Amish and Mennonite families in Pennsylvania, is building a biotech company by and for them, STAT reports. [3] These communities are hit by an unusual set of rare diseases. [3] The company aims to revive treatments that drugmakers dropped or left in university freezers, often because too few patients would make a profit. [3] Strauss's team gave a gene treatment to a calf with a cattle form of maple syrup urine disease, and she is now a healthy five-year-old heifer. [3][20] None of his patients has had it yet, even in a trial. [20]

Why it matters. The FDA draft in today's lead lets new versions of a treatment skip new trials. [1] This company tries a second route: owners who will pay for a medicine that would never make money. [3]

Ferring Pharmaceuticals, a privately held Swiss maker of drugs for IVF, the treatment that joins egg and sperm outside the body, signed a $1.4 billion deal on Tuesday to buy Gameto. [16] Gameto, a biotech, is best known for a product meant to improve egg freezing and fertility for women. [16] Ferring plans to sell it through its reach into fertility clinics around the world. [16]

Why it matters. The product still needs approval from the FDA, so the price is a bet on an approval that has not happened. [16]

Researchers isolated a compound from a marine bacterium that killed several strains of Naegleria fowleri faster than the current treatment, in lab tests. [3] N. fowleri is a tiny parasite that lives in warm fresh water and can infect the brain. [3] Infections are hard to treat and nearly always fatal. [3]

Why it matters. The team's next step is to test whether the compound can cross from the blood into the brain. [3] Much work remains before it could become a medicine. [3]

Researchers in Beijing found that pantethine, a form of vitamin B5 sold as a supplement, calmed the immune cells that drive psoriasis, a skin disease. [17] It stopped psoriasis developing in mice. [17] In a 12-week trial of 24 patients, all used a standard skin ointment and half also took pantethine by mouth. [17] Those on pantethine improved more, especially if they started with high cholesterol. [17] The study is in the Journal of Experimental Medicine. [17]

Why it matters. The lead researcher calls the trial a sign the idea can be tested, not proof of how it works. [17] Pantethine is already sold widely as a supplement to lower cholesterol. [17]

A study from one hospital looked back at 496 adults with acute myeloid leukaemia, a fast-growing blood cancer. [18] Some had been given gemtuzumab ozogamicin, an antibody that carries a poison into cancer cells, in three small doses with chemotherapy. [18] Where the cancer had a change in a gene called NPM1, 57.9% of those on the drug were alive without relapse at two years, against 36.0% without it. [18] Liver damage was no more common. [18]

Why it matters. The patients were not split at random, and those who got the drug were younger with less risky cancers. [18] So the gap may partly come from who received it, which only a randomised trial can settle.

Liposuction fell 11.4% worldwide in 2025, after a 12.6% fall the year before, in a survey by the International Society of Aesthetic Plastic Surgery. [19] It asked more than 2,900 surgeons in 51 countries. [19] Cosmetic procedures fell 14% for men and 7.7% for women. [19] Surgeons in Dubai told The National that weight-loss drugs are a big reason. [19]

Why it matters. One Dubai surgeon said the drugs are also creating a new group of patients, who want their face and body reshaped after losing weight. [19]