Biotech & Longevity · Friday, 18 September 2026

01 Briefing what happened

The first treatment for Sanfilippo syndrome, a disease that takes speech and thinking from young children, is approved in the US. It was tested on 17 children.

Biotech & Longevity 25 sources

The US drug regulator approved Ultragenyx's Fayuvi, a one-time gene therapy, after treated children scored higher on thinking tests than untreated children from medical records; the rest of the day was drug deals that pay little up front, a measles standoff in Pennsylvania and the end of a record US parasite outbreak.

23.5 points

higher on a thinking test for treated children than for untreated children from records

17 treated against 27 untreated, measured between ages 2 and 5 [2]

15 years

the median life expectancy of a child with Sanfilippo syndrome type A

until 17 September there was no approved treatment, only care for symptoms [1][2]

3,000 to 5,000

patients Ultragenyx estimates in the countries where it can sell

follow-up on treated children now reaches nearly eight years [2]

The lead story — what happened

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]

Who is involved

Ultragenyx

a US company that makes medicines for rare diseases; it won the approval and has not yet said what Fayuvi will sell for
The US Food and Drug Administration (FDA)

the US drug regulator; it refused the therapy last year over manufacturing and approved it on 17 September
Abeona Therapeutics

a US gene therapy company that held the drug; it licensed it to Ultragenyx when money ran short
Nationwide Children's Hospital

a children's hospital in Ohio where the virus carrier was first built; its doctor Kevin Flanigan led the study
Cure Sanfilippo Foundation

a group started by parents of children with the disease; it says families used to be sent home with nothing to try

How it unfolded

Over a decade ago the virus carrier is built at Nationwide Children's Hospital in Ohio; Abeona later licenses the drug to Ultragenyx when its money runs short [2]
2025 the FDA declines to approve it, citing manufacturing concerns [4]
17 September the FDA approves Fayuvi; Ultragenyx shares rise nearly 6% [1][4]
Within 30 to 60 days first shipments to specialist treatment centres [2]

Where this points

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]

What is pushing on the whole day

The bar and the word are our reading of how hard each one is pushing today. The arrow is where it is heading. The evidence is in the stories below.

Big promises, small first payments High↑

Roche paid Dualitas $36.5 million on signing a deal that could reach $1 billion. [7] SK Biopharmaceuticals paid 1ST Biotherapeutics $1.8 million of a possible $314.8 million. [8] Transcenta got $1.5 million upfront from WuXi Biologics. [10]

Joining the stock market through a shell Building↑

North Immunology is taking over Aethlon Medical, a listed company whose owners keep 4.75%. [14] Lisata's shareholders will keep about 2.4% after it combines with Marea. [16] About two dozen biotech deals of this kind were announced this year, against 10 in 2025. [24]

Getting medicines into the brain Building↑

Fayuvi reached children's brains from a single infusion into a vein. [1] Lilly signed with QurCan, whose tiny particles are meant to carry gene medicines past the brain's protective lining. [9] SK licensed a Parkinson's pill that blocks an enzyme which, in excess, disrupts parts of brain cells. [8]

Officials disputing the death count High→

Pennsylvania will accept federal measles help only if the CDC counts four deaths the state links to measles. [18] Africa's public-health agency said Congo's own data cannot show that its Ebola outbreak has peaked. [22]

The rest of the day

15 more stories on this beat.

Each with its own sources. None of these is a link to the story above.

02
Pennsylvania asks for federal measles help, with a condition

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]
03
Africa's health agency says Ebola has not peaked

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]
04
The largest US cyclospora outbreak is over

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]
05
Roche pays $36.5m toward a $1bn search

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]
06
GSK bets up to $750m on a Chinese myeloma drug

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]
07
$1.8m now for a $315m Parkinson's pill

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]
08
Lilly signs for a way to carry gene medicines into the brain

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]
09
Secret price deals may shrink Medicare's savings

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]
10
A judge pauses the doctors' case over US vaccine policy

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]
11
The new US cancer-drug chief sets out his plans

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]
12
An eczema startup joins the stock market through a shell

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]
13
Lisata's owners keep 2.4% in a rescue merger

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]
14
Electra sets a price for its share sale

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]
15
GE HealthCare reported in talks for Sofie

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]
16
A Chinese biotech licenses its factory method to WuXi

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]

02 Lesson why it matters

Why a billion-dollar drug deal pays so little on the day it is signed

Most of the big number in a drug deal is paid only if the drug keeps passing tests, so the small company selling it carries most of the risk.

The twist

Roche's deal with Dualitas is reported as $1 billion. Dualitas gets $36.5 million now, and the rest arrives only if drugs that do not yet exist keep passing tests.

How it works

A small company has an early drug or tool and not enough money to test it in people
A big drugmaker pays a small sum on signing, often a few percent of the headline figure
It promises far larger payments later, each due only when the drug passes a set step
Most early drugs fail a step, and the later payments stop there
So the headline number is the seller's best case, and the upfront is the only money it can count on

The same force, elsewhere today

Where this chain is also running, in today's other stories.

SK's $315m Parkinson's pill

1ST Biotherapeutics gets $1.8 million now and the same again soon, and the other $311 million waits on a pill that has not reached patients.
Transcenta's factory method

WuXi pays $1.5 million upfront, and the rest comes only if the method meets conditions set in the deal.
Lilly's brain delivery deal

QurCan's up to $237 million is counted per program, so it is paid only for each medicine that gets through its steps.
Fayuvi, the lead story

Abeona held a drug with good early results but ran short of money to finish it, which is the first step of this chain.

Where you've seen this

Football transfers

a fee reported as up to 100 million often includes bonuses paid only if the player makes enough appearances or the club wins trophies

Book deals

an author gets a smaller advance now and royalties only if the book sells

Buying a startup

the founders get part of the price at the sale and the rest only if the business hits targets over the next years

The catch

A buyer can usually hand the drug back at any step, so the seller keeps the upfront and the drug but loses the rest of the headline sum.

And the whole of it

A family waiting for a Parkinson's or myeloma drug reads the same $1 billion headline as everyone else. Whether that money is ever paid gets decided years later, in test results that neither the buyer nor the seller can see today.

03 Truth what's really going on

What is really going on

Most of the biotech money announced this week was promised, not paid: Roche paid Dualitas $36.5 million on a deal billed at $1 billion, and SK paid 1ST Biotherapeutics $1.8 million of a possible $314.8 million. [7][8] The one drug that did reach patients, Ultragenyx's Fayuvi, was approved by comparing 17 treated children with 27 untreated children from an earlier record of the disease. [2]

Why it works on us — A deal is reported at its largest possible total, and a round number like $1 billion sticks in the mind while the few million actually paid does not.

Who gains

Ultragenyx — It gets its second gene therapy approval and a priority review voucher, a fast-review pass it can sell to another drugmaker. [2]
Roche — It gets first pick of 300,000 screened drug pairs for $36.5 million now, and pays more only for the ones that work. [7][6]
SK Biopharmaceuticals — It controls a Parkinson's pill for $1.8 million upfront and a near-term payment of the same amount. [8]
Private investors behind North Immunology and Marea — They reach the Nasdaq without a public share sale, while the listed companies' old shareholders keep 4.75% and about 2.4%. [13][14][16]
Drug companies with secret Medicare price deals — A Lancet model says the deals could cut the plan's benefit by up to 80%, and the rebate rules reward raising prices outside the US. [19]

Who pays

Families of children with Sanfilippo type A — The therapy ships in 30 to 60 days, at a price nobody has named, and only to children still mostly developing normally. [2][3]
Small drug developers selling rights — 1ST Biotherapeutics and Transcenta get under $2 million each upfront, and the rest depends on steps they may never reach. [8][10]
Aethlon and Lisata shareholders — Their stakes fall to 4.75% and about 2.4% of the combined companies. [14][16]
People in Pennsylvania's measles outbreak — More than 700 cases across 37 counties, while federal help waits on an argument over the death count. [18]
People made ill by the lettuce — 12,883 people in 21 states fell ill, 570 went to hospital and two died in Michigan. [20][21]

What nobody knows yet

Open questions from across today’s stories — ours included.

01

What Fayuvi will cost, and who will pay for it.

Ultragenyx did not release a price with the approval, and families depend on insurers agreeing to cover it. [3][2]
02

How long the benefit lasts, and whether it helps children who have already lost abilities.

Follow-up reaches nearly eight years, and the approval covers only children whose development is still mostly intact. [2]
03

Whether the inserted gene causes cancer years later.

The FDA lists it as a potential long-term risk of this kind of gene therapy, and the longest follow-up so far is nearly eight years. [1][2]
04

How many measles deaths Pennsylvania has had, by the national count.

The state reports four measles-associated deaths, and the CDC has not recognised them on the health secretary's instruction. [18]
05

Whether Congo's Ebola outbreak has passed its peak.

Congo's health minister says it peaked in mid-August, and Africa CDC says the data cannot confirm that. [22]
06

How the cyclospora parasite got into the lettuce.

The FDA inspected the farms and processing plant in Mexico, and the sample results are still pending. [20]
07

How much GSK is paying Chimagen now, and for what.

The reports give only the $750 million ceiling, with no upfront figure, and the drug's targets were not disclosed. [5][6]
08

What Medicare's most-favoured-nation plan will actually save.

The deals with more than two dozen drug companies are secret, and one model says they could cut the benefit by up to 80%. [19]
09

Whether GE HealthCare buys Sofie, and for how much money.

The only report is the Financial Times citing unnamed people, which Reuters could not verify. [11][12]

04 Hope carry this

Children with Sanfilippo syndrome type A now have an approved treatment for the first time. In the study behind it, treated children scored 23.5 points higher on a thinking test, measured between ages 2 and 5, than untreated children with the same disease.

Also true today

The largest cyclospora outbreak ever recorded in the US is over. At its peak more than 1,000 people fell ill each day, and by August it was fewer than two.