Biotech, in brief

The US Food and Drug Administration approved Fayuvi on 17 September, the first treatment for Sanfilippo syndrome type A. [1][4]

Sanfilippo type A is an inherited disease. Children lack an enzyme called sulfamidase, so a sugar molecule, heparan sulfate, builds up in the brain. [1][4]

Children develop normally at first, then lose thinking, speech and movement. Half of them die before about 15. [1][2]

It is sometimes called childhood Alzheimer's. [3]

Fayuvi is given once, into a vein. A harmless modified virus, AAV9, carries a working copy of the missing gene into the body's cells, including in the brain. [1][2]

There was no dummy-treatment group. 17 treated children were compared with 27 untreated children whose decline was recorded in an earlier study of the disease. [2]

Between ages 2 and 5, treated children scored 23.5 points higher on a standard thinking test than the untreated group. [2]

The FDA said treated children kept or improved their thinking, where the disease normally stalls it and then takes it away. [1]

It is approved only for children whose development is still mostly intact. [2]

Side effects included liver damage signs, low platelets, nausea and fever. Every child takes steroids from the day before the infusion for at least eight weeks. [1][2]

The FDA also warns of a rare clotting disorder in small blood vessels. It lists a possible long-term cancer risk if the inserted gene lands in the wrong place in the DNA. [1]

The FDA turned the therapy down last year over manufacturing concerns. [4]

The therapy began at Nationwide Children's Hospital in Ohio. Abeona Therapeutics held it, ran short of money and licensed it to Ultragenyx. [2]

Ultragenyx has not said what it will charge. [3] It expects to ship to specialist treatment centres within 30 to 60 days. [2]

Why it matters. Watch what Ultragenyx charges for Fayuvi and whether insurers pay for it, and whether treated children keep their abilities as follow-up passes eight years. [2][3]

Pennsylvania's health department asked the US Centers for Disease Control and Prevention (CDC), the national public-health agency, for its outbreak-support teams, a service called Epi-Aid. [18] It set one condition: the CDC must recognise four deaths the state links to measles. [18] US Health Secretary Robert F. Kennedy Jr. had told the agency to leave Pennsylvania's deaths out of the national count. [18] The state's outbreak has passed 700 confirmed cases across 37 counties. [18]

Why it matters. The state had refused federal help for weeks, saying it had the outbreak under control, and on 13 September the CDC's director said it had not taken up repeated offers. [18][19] A US health department spokesperson called the condition political blackmail and said the request had not come through normal channels. [18]

Africa CDC, the African Union's public-health agency, said on 17 September that the Ebola outbreak in the Democratic Republic of Congo is still serious. [22] Congo's health minister had said the peak came in mid-August and spread was slowing. [22] Africa CDC said the data could not confirm a peak, though its director praised a slight drop in Ituri province. [22] By 11 September Congo had counted 7,022 confirmed cases and 3,398 deaths, the largest outbreak in its history. [25]

Why it matters. The World Health Organization says shortages of trained staff, partners and money are already holding the response back. [25] Africa CDC's director said officials need to see a sustained fall in cases before saying the peak has passed. [22]

US health officials declared the country's largest recorded outbreak of cyclosporiasis over on 11 September. [20] Cyclospora is a tiny parasite spread in contaminated food or water, and it causes severe diarrhoea. [21] Iceberg lettuce from central Mexico, supplied by Taylor Farms, made 12,883 people ill in 21 states, put 570 in hospital and killed two. [20] At the peak more than 1,000 infections were reported a day; by August it was under two a day. [20]

Why it matters. The worst season before this one, in 2019, had about 4,700 illnesses. [20] The US Food and Drug Administration is still testing samples from the Mexican farms, so nobody yet knows how the parasite got into the lettuce. [20]

Roche, the Swiss drugmaker, is paying Dualitas Therapeutics $36.5 million upfront to screen more than 300,000 pairs of targets for new immune-disease drugs. [7][6] The drugs are bispecific antibodies, single molecules that grab two targets at once. [7] Dualitas finds the promising pairs and Roche develops the few it chooses. [7][6] Further payments, due only as programs pass set steps, could take the total to $1 billion, plus a share of sales. [6][7]

Why it matters. The upfront is under 4% of the headline figure, and the rest depends on drugs that do not exist yet. [7] Dualitas, founded a year ago with $65 million, will use the money to take its own arthritis drug into people in 2027. [6]

GSK, the British drugmaker, agreed to pay up to $750 million for a drug from Chimagen Biosciences, a company in China, against multiple myeloma, a cancer of the bone marrow. [5] The drug is a trispecific T-cell engager: one antibody that holds a cancer cell against an immune cell, using two cancer targets instead of one. [5] All four approved drugs of the older two-armed kind carry the strongest US warning for dangerous immune reactions. [5] GSK plans first human tests next year. [5]

Why it matters. It is GSK's fifth licensing deal with a China-based drugmaker since the start of 2025. [6] The reports give no upfront figure, and neither company disclosed which targets the drug hits. [5]

SK Biopharmaceuticals, a South Korean drugmaker, licensed an experimental Parkinson's pill called 1ST-104 from 1ST Biotherapeutics, another Korean company. [8] The pill blocks two enzymes, including LRRK2, which in excess stops brain cells growing the tiny antenna-like parts they use to receive signals. [8] 1ST Biotherapeutics gets $1.8 million upfront and the same again soon, out of a possible $314.8 million. [8] SK is also buying $2.2 million of its shares. [8]

Why it matters. Rival LRRK2 drugs from Neuron23 and Denali are further ahead, and Biogen gave up the Denali drug in May after a mid-stage failure. [8] SK's previous deal, $350 million upfront for an epilepsy drug, was hit days later when the US regulator paused that drug's studies. [8]

Eli Lilly, the US drugmaker, agreed to work with QurCan, a Toronto company, on genetic medicines for the brain and nerves. [9] QurCan carries genetic material in tiny particles instead of a virus. It says they can cross the blood-brain barrier, the lining that keeps most drugs out of the brain. [9] Lilly pays an undisclosed upfront sum and invests in the company. [9] QurCan could receive up to $237 million per program, plus royalties. [9]

Why it matters. Lilly already sells an Alzheimer's drug and is testing a gene therapy for Parkinson's, so one route into the brain could serve several of its programs. [9] QurCan says its particles can be given more than once and can reach targets in the brain and nerves without a virus. [9]

A modelling study in The Lancet looked at the Trump administration's most-favoured-nation plan. [19] The plan ties what Medicare, the US government's health insurance for older people, pays for some drugs to prices in other rich countries. [19] The Trump administration has also signed private deals with more than two dozen drug companies. [19] The study found those deals could cut the plan's benefit by as much as 80%. [19]

Why it matters. Because the deals are not public, nobody outside can judge what the plan delivers, said the lead author, Thomas Hwang of Brigham and Women's Hospital. [19] The rebate rules could also push companies to raise prices outside the US or to delay launching new drugs. [19]

US District Judge Brian Murphy paused the lawsuit that the American Academy of Pediatrics, the main US body for children's doctors, brought against Health Secretary Kennedy's vaccine changes. [19] The suit challenges his remaking of the Advisory Committee on Immunization Practices and changes to the childhood vaccine schedule. [19] In March the judge said the doctors were likely to win, and the US government appealed. [19] The appeals court hears arguments on 6 October. [19]

Why it matters. The committee helps the CDC set vaccine policy, and while the courts decide it has not met. [19] That leaves it silent as the flu, Covid and RSV season begins. [19]

Angelo de Claro now leads the Oncology Center of Excellence, the US Food and Drug Administration office that reviews cancer drugs. [23] He replaced Richard Pazdur, who ran the agency's cancer drug work for more than two decades. [23] In his first interview he said he wants to hire and keep staff, use AI tools in reviews and enrol more US patients in drug studies. [23] The office's work led to 17 new cancer drugs approved in 2025. [23]

Why it matters. In May he became the first senior drug-review leader confirmed in a permanent post while the agency's top ranks were being replaced. [23] His office approved a pancreatic cancer drug, Rasonque, more than six months ahead of its deadline. [23]

North Immunology, a private company started inside the hedge fund ADAR1 Capital, will merge with Aethlon Medical, a small company already listed on the Nasdaq exchange. [14][15] This is a reverse merger: a private firm takes over a listed one to reach the stock market without a public share sale. [13] Aethlon's shareholders will own 4.75% of the combined company. [14] Investors including Bain Capital are putting in $180 million, enough to run it into the second half of 2028. [13][14]

Why it matters. Its drug NOR-101, for atopic dermatitis, the commonest eczema, blocks two inflammation signals and has not yet been tested in people. [14][13] About two dozen biotech reverse mergers have been announced this year, against 10 in all of 2025. [24]

Lisata Therapeutics, a US biotech, will combine with Marea Therapeutics in a share swap that works like a reverse merger. [16] Lisata's current shareholders will own about 2.4% and Marea's investors 59.5%. New investors putting in $225 million will own the rest. [16] Lisata cut 72% of its staff after a planned merger with Kuva Labs collapsed last month, and it is suing Kuva. [16] Marea's two drugs, for very high blood fats and for acromegaly, a growth-hormone disorder, report results in the last three months of this year. [16]

Why it matters. The listed name survives, but the drugs, the money and nearly all the ownership come from the private side. [16] RA Capital, one of the busiest biotech investors this month, is among the new backers. [16][24]

Electra Therapeutics, a biotech near San Francisco, set terms for its first public sale of shares: 21.7 million shares at $14 to $16 each. [17] That could raise up to $341.9 million if extra shares are sold. [17] It plans to spend $220 million finishing a late-stage study of its drug ipsoprubart in secondary HLH, a rare and deadly immune overreaction that cancer or infection can set off. [17] In an earlier small study, all 12 patients were alive after eight weeks. [17]

Why it matters. Electra said last month it would go public, and this filing adds the numbers. [17] It is taking the traditional route in a year when about two dozen biotechs have announced reverse mergers instead. [24]

GE HealthCare, a US maker of medical scanners, is in talks to buy Sofie Biosciences for up to $1 billion, the Financial Times reported on 13 September. [11][12] Sofie, based in Virginia, makes radioactive chemicals that make certain cancers show up on PET scans, including stomach, gullet and pancreatic cancers. [11][12] Reuters could not verify the report, and neither company commented. [11] It would be GE HealthCare's second big purchase since General Electric split into three in 2024. [11]

Why it matters. It would add to GE HealthCare's business supplying scan chemicals, beside the MRI, CT and PET machines it already builds. [11][12] The company cut its profit forecast in April and is reviewing another of its units. [12]

Transcenta Therapeutics, a drug developer in Suzhou, China, licensed its HiCB manufacturing method to WuXi Biologics, a large Chinese contract drug manufacturer. [10] HiCB grows antibody-making cells continuously instead of in one batch at a time, which Transcenta says produces more for less money. [10] Transcenta gets $1.5 million upfront and further payments if set conditions are met. [10] WuXi also becomes its manufacturing partner for its own drugs. [10]

Why it matters. Transcenta has more than ten antibody drug candidates, for cancer, bone and kidney disease, that need a manufacturer. [10] Beyond the $1.5 million, every payment depends on conditions the companies have not made public. [10]