Biotech, in brief

China's Ministry of Commerce, which runs the country's trade, published a draft on Friday adding biotech methods to its list of technologies that cannot freely leave China. [1][2]

Foreign companies would be banned from buying Chinese platforms for cell and gene therapy and for siRNA, small pieces of RNA that switch off one gene. [1]

A platform here means a set of reusable tools that can produce many drugs, not just one. [1]

Platforms for gain-of-function work, which makes germs more dangerous in order to study them, would also be banned from sale, and China's commerce ministry gave national security as the reason. [1]

Selling abroad the full methods for finding and checking drug targets in these fields would need government approval. [1] A target is the molecule in the body that a drug acts on.

Licensing stays open: a Chinese company can still rent out the rights to a drug, or do research and manufacturing for foreign clients. [1][2][4]

China's drug regulators count $120 billion of foreign deals for Chinese biotech assets this year up to August, 36% more than a year earlier. [1]

Western buyers have almost always bought single drugs, or used a Chinese platform to find new ones, rather than buying the platform itself. [1] AstraZeneca's $1 billion purchase of Gracell, a Chinese maker of CAR-T cell therapy, at the end of 2023 is the kind the draft would likely stop. [1]

Roche, the Swiss drugmaker, has signed five deals with Chinese drug developers since late August, more than any other big drugmaker. [5]

One of them, on 13 October, rented the rights to an RNA-based obesity drug from Rona Therapeutics, built on Rona's own delivery platform. [6] Roche pays up to $100 million soon and up to $1.06 billion later. [6]

Aaron Gu of Han Kun, a Chinese law firm, called the draft significantly better than the market expected. [1] A list rumoured in May had also covered antibody-drug conjugates, antibodies that carry a drug to a cell, and other popular kinds of medicine. [1]

The same draft covers 40 technologies in all, including 23 kinds of AI such as large language models and chip designs. [1][3]

Two members of the US Congress have proposed a bill to put US deals for Chinese drugs under more checks, but it has not moved. [1]

Comments on the draft close on 15 November. [1] Gu says that until the list takes effect, exports in its fields must be registered with China's commerce ministry. [1]

Why it matters. Watch what the final list says after comments close on 15 November, and whether US Treasury rules also leave licensing of Chinese drugs open. [1]

The committee of the European Medicines Agency, the EU's medicines regulator, recommended approval on Friday of Foundayo, Eli Lilly's weight-loss pill. [7] Foundayo, also called orforglipron, is a GLP-1 pill. [8] GLP-1 drugs copy a gut hormone that makes people feel full. It is the second weight-loss pill to win the committee's backing, after Novo Nordisk's Wegovy pill. [7]

Why it matters. Eli Lilly, the US drugmaker, is trying to catch up with Novo Nordisk, its Danish rival, in pills. [8] A recommendation is not yet a licence: the final approval in the EU comes after it.

Kenya confirmed its third Ebola case on Friday, a 65-year-old woman who is a close relative of its first two patients. [9] She had been in quarantine since 6 October when she fell ill with fever and a sore throat. [9] Kenya's first patient, a 40-year-old businessman, died on 5 October after travelling from Congo through Uganda. [9][11] Officials say 127 of 130 known contacts are traced and quarantined. [9]

Why it matters. In Congo's Ituri province, 10 of 25 school pupils confirmed with Ebola have died since schools reopened. [10] This outbreak is the Bundibugyo strain, for which there is no specific vaccine or treatment. [9]

The FDA, the US medicines regulator, approved Roche's Enspryng on Friday for thyroid eye disease. [12] In this disease the immune system inflames the tissue around the eyes, which can bulge and see double. [12] Enspryng is an antibody that blocks a signal driving the swelling, and patients can inject it at home. [12] In one final trial, 52.9% on the drug had less bulging, against 23.4% on a dummy. [12] The other trial missed its main goal. [12]

Why it matters. It is the first treatment for the disease given as an injection under the skin rather than through a drip. [12] About 155 in every 100,000 people have the disease. [12]

A jury in Suffolk County, Massachusetts, awarded $6 million on Friday to David Sabatini, a former MIT professor and star biologist. [13] His career collapsed after Kristin Knouse, a younger scientist, said he pressured her into a relationship and ran a sexualised lab. [13] His lab was at the Whitehead Institute, a research centre in Cambridge. [13] The jury of seven women and six men found she had defamed him and must pay, plus interest. [13]

Why it matters. The three-week civil trial showed how much power lab heads hold over the young scientists who work for them. [14] The case turned on two scientists' opposite accounts and their text messages. [14]

The UK government said it will review the scheme that pays people injured or bereaved by vaccines. [15] Baroness Hallett, who chairs the official inquiry into Covid, had called the scheme not sufficiently supportive. [15] More than 24,000 people have claimed for Covid jabs, and just over 1% won the one-off award of 120,000 pounds. [15] Claimants must show they are at least 60% disabled. [15]

Why it matters. The campaign group Vaccine Injured and Bereaved UK says the UK government gave no timetable. [15] Running the scheme has cost more than double the 32 million pounds it has paid out. [15]

A study published on 12 October looked for blood cancers in adults with type 2 diabetes who started GLP-1 drugs such as semaglutide, the drug in Ozempic. [16] It matched 68,949 of them with similar people starting a different diabetes pill and followed them for up to five years. [16] Rates of seven kinds of blood cancer, including lymphoma and leukaemia, did not differ. [16] Against a third drug class, GLP-1 users had fewer of three blood cancers. [16]

Why it matters. The data came from TriNetX, a network of over 100 million health records, so the study can show a link or its absence but not prove a cause. [16]

A study in Nature Medicine on 16 October followed 8,127 women in the UK who started a weight-loss drug through an online programme. [17] Almost all, 99.8%, took tirzepatide. [17] After six months they had lost 15.5% of their starting weight on average, and 91.4% were still taking part. [17] The programme required blood tests and a video check-in every week. [17]

Why it matters. Staff of the two companies that ran it, eMed Healthcare UK and SheMed, are among the authors. [17] The women paid for themselves and there was no comparison group, which the authors list as limits. [17]

City Therapeutics raised $200 million on Thursday by selling 11.1 million shares at $18, more than it planned. [18] It began trading on Nasdaq on Friday. [18] It makes RNAi drugs, which silence a gene before it can make a harmful protein. [18] Its lead drug targets Factor XI, a clotting protein, and cut its level by up to 85% in early tests. [18] Iambic Therapeutics, which designs drugs with AI, saw its shares rise more than 50% after a $150 million listing. [19]

Why it matters. All eight approved RNAi medicines act in the liver, and City is trying to reach other parts of the body. [18] Earlier AI drug companies lost value after listing: Recursion's shares fell from $18 to under $4. [19]

Novartis, the Swiss drugmaker, ended a mid-stage trial of its pill Fabhalta in lupus nephritis, a kidney inflammation caused by the immune disease lupus. [20] It gave strategic considerations as the reason, in the US government's database of clinical trials. [20] Fabhalta is also called iptacopan. [20]

Why it matters. Novartis gave no results, so it is not known whether the drug worked in these patients.

Estuary Biotherapeutics, a California company backed by Biogen and run partly by its former staff, launched on Friday with $93 million. [21] It aims at ADPKD, an inherited disease in which cysts grow in the kidneys and often cause kidney failure. [21] In mice, restoring proteins called polycystins shrank the cysts. [21] Its two gene therapies are due to start human tests in 2027 and 2028. [21]

Why it matters. About 1 in 400 to 1,000 people have ADPKD, and only one drug, tolvaptan, is approved for it. [21] Estuary's chief executive says getting genes into the kidney is the hardest step left. [21]

Journals retracted four studies on how a mother's diabetes affects the neural tube, the early form of a baby's brain and spine. [22] The University of Maryland, Baltimore found copied images and other data problems, the retraction notices say. [22] All four list Peixin Yang as the lead author to contact. [22] Data sleuths have flagged 44 studies on which he is the only shared author. [22]

Why it matters. Yang's lawyer says three of the notices do not find that he committed misconduct. [22] Mary Loeken, a Harvard researcher, says her lab could not reproduce the team's results. [22]

Nature added an expression of concern, a public warning to readers, to a 2017 paper last week. [23] The paper linked a mother's gut bacteria to autism-like behaviour in her mouse pups, and has been cited more than 680 times. [23] The same values repeat across 10 of its figures. [23] The raw data were lost when the lab moved, so the numbers cannot be checked. [23]

Why it matters. Jun Huh, the Harvard immunologist who led it, says later studies support its findings. [23] Mu Yang, a Columbia scientist who checks research, says the repeated data are central to its conclusions. [23]

A documentary on Elizabeth Holmes, You Can See Everything, opened in cinemas on Friday. [25] Holmes ran Theranos, which claimed one drop of blood could run hundreds of tests. [24] She was sentenced to more than 11 years in prison for defrauding investors. [25] Her partner, Billy Evans, is building a new blood-testing company, Haemanthus, with Holmes listed as an adviser. [24]

Why it matters. John Ioannidis, a Stanford medical professor who questioned Theranos early, says start-ups now make claims without strong evidence more often. [24] A Case Western researcher says Theranos made funding harder to win for scientists who study blood tests. [24]

Four years after the FDA let adults buy hearing aids without a prescription, a study in JAMA, a leading US medical journal, weighed the result on Thursday. [25] Over-the-counter aids cost about $500, against about $3,400 for prescription ones, and most first-time buyers now choose them. [25] Only 25% of the hearing aids listed on Amazon could be matched to FDA records. [25]

Why it matters. Some of the first companies to sell them have already left the market. [25] It is not known how many people who need a hearing aid now buy one. [25]

Insilico Medicine, a drug company whose AI team works mainly in Abu Dhabi, says its pill ISM9528 eased pain better than morphine in animal tests. [26] It is not an opioid, the family of addictive painkillers that includes morphine. [26] It has not yet been tested in people. [26] Insilico says its AI lets it pick a drug candidate in 12 to 18 months, against about five years usually. [26]

Why it matters. Nine in ten drugs that reach human testing are never approved, so an animal result is only a first step. [26] The company says AI does not shorten human trials, which can take a decade. [26]

Scientists at Cold Spring Harbor Laboratory, a US research centre, reported in Nature Immunology this week that a protein called PPARD can help immune cells fight cancer. [27] Cancer cells use PPARD to grow, but turning it on in immune cells made them burn more fat and attack tumours harder. [27] In lab models of human colon cancer, CAR-T cells with PPARD switched on slowed or stopped tumour growth. [27]

Why it matters. CAR-T cells are a patient's own immune cells re-engineered to hunt cancer. [27] Colorectal cancer is now the leading cause of cancer death in people under 50, and this approach has not been tested in patients. [27]

Researchers at Tufts and Boston University grew small balls of breast tissue, called organoids, from cells given by 13 healthy young women. [28] Three common bisphenols, BPA, BPS and BPF, are chemicals that can interfere with the body's hormones. [28] Alone, each did little. [28] Mixed at levels found in people's blood and urine, they disrupted how the tissue developed in most donors' samples. [28] The work appeared in PNAS. [28]

Why it matters. The changes matched patterns seen in invasive lobular carcinoma, a breast cancer that a mammogram can miss. [28] The study does not show that the chemicals cause cancer. [28]

A study in Nature Communications on 17 October followed 238 adults in Lesotho with drug-resistant tuberculosis, a lung infection that the usual antibiotics no longer kill. [29] They took a shortened course of pills only, with no injections, a treatment the WHO backs. [29] Of 148 whose lab cultures showed the germ at the start, 142 cleared it within four months. [29] There were 27 poor outcomes, including 20 deaths. [29]

Why it matters. Most of the patients, 62%, also had HIV. [29] Scarring and holes in the lungs on the first X-ray pointed to who would do worse. [29]

Moderna's shares have risen more than 600% this year, against 23% for XBI, a fund that tracks biotech shares. [5] They fell to about $22 last year, near their 2018 debut price, after demand for its Covid vaccine dropped. [5] The rise took off in August, when its melanoma vaccine with Merck succeeded in a final-stage trial. [5] The vaccine uses messenger RNA, like Moderna's Covid shot. [25]

Why it matters. The full trial results are due at a medical meeting later this month. [5] RBC Capital Markets, an investment bank, calls that the biggest event for both companies for the rest of this year. [5]

The South African Pharmacy Council, which sets the rules for pharmacists, is working with the industry's regulator to tighten rules on pharmacies making their own copies of medicines, Reuters reported. [30] The copies include versions of the weight-loss and diabetes drugs Ozempic and Mounjaro. [30] The council's chief executive, Vincent Tlala, says some pharmacies now make large batches in advance, rather than for specific patients. [30] New guidelines are expected around March next year. [30]

Why it matters. Novo Nordisk won a court case in June against local pharmacies making unauthorised copies, and it and Eli Lilly are likely to welcome the change. [30] South Africa has Africa's highest adult obesity rate, 30%, and the branded drugs cost too much for most people. [30]