Biotech, in brief

HuidaGene, a Shanghai gene-editing company, has published its treatment's first trial in people. [1]

Four boys with Duchenne muscular dystrophy were given its gene-editing treatment. [1]

1 of the 4 boys died; he had been given a high dose. [1]

The treatment edited some of the target genes, but its benefit was limited. [1]

The results are now published in Cell, a science journal. [1]

Researchers say treatments like this urgently need safe doses worked out. [1]

Severe immune reactions, including deaths, have also hit other gene therapy trials for Duchenne. [1]

The death also put China's fast-track rules for clinical trials in the spotlight. [1]

Immunology studies the body's defences, which attack what looks foreign, even a harmless virus. [1]

The more virus arrives at once, the stronger that attack can be. [1]

The team gave immune-calming drugs and spaced out each boy's dose after a safety review. [1]

Yang says those steps were not enough. [1]

He says this kind of immune harm 'remains difficult to predict'. [1]

Whether the bigger dose caused the reaction is still unclear, he says. [1]

Woodcock says new kinds of AAV that enter muscle better could allow smaller doses. [1]

Or teams could carry the editor without AAV at all. [1]

Why a disease from one faulty gene can be treated at four different steps

A gene holds the instructions for one protein

A fault in the gene leaves too little protein, or a misshapen one

Without that protein a job goes undone: muscles weaken, nerves fail, an enzyme stalls

Medicine can step in at the gene: fix it, or add a working copy

Or further along: give the protein itself, or a drug that helps a faulty one work

Giving the protein is the safer step, but it never ends: Fabry patients need a drip every two weeks.

It worked on the gene itself, sending an editor into muscle, and the big virus dose needed to reach every muscle is suspected in one boy's death.

They add a working copy of the FXN gene without a virus, and in mice frataxin rose 18-fold in the liver.

Its new drug gives the faulty enzyme from outside as a monthly shot, while its pill Galafold helps a misshapen enzyme do its job.

It helps the CFTR protein work rather than replacing it, tried here in mice with lung scarring.

This fits diseases where one gene leaves the body short of one protein.

The same chain, from gene to protein to job, is running in every reader's body right now.

HuidaGene, a Shanghai company, gave a gene editor to four boys with Duchenne muscular dystrophy, and one died after a high dose that was set by his body weight. [1]

The company told the public about a year after the death, and its own co-founder says the safeguards it used were not enough. [1]

Doses were set by body weight, so as the heavier high-dose boy he received more of the virus-carried treatment. [1]

What set off the boy's fatal immune reaction.

He got a bigger dose because he weighed more for his height, but the authors say it is still unclear whether that triggered it. [1]

Ebola has now killed 5,026 people in Congo, out of 10,351 infected, government data show. [2]

Ebola is a deadly virus disease, caught by touching a sick person's blood or vomit. [3]

Only West Africa's outbreak of 2014 to 2016 killed more: about 11,000 people. [2]

This outbreak was declared in May and is caused by the Bundibugyo strain. [2]

There is no approved vaccine or medicine for that strain. [3]

Nearly half of those with confirmed Ebola have died: the death rate is 48.6%. [3]

It has reached 8 of Congo's 26 provinces. [2]

About 80% of new cases come from people nobody was tracking, Africa's disease agency says. [2]

A third of the dead died before any test, the World Health Organization says. [2]

Ituri and North Kivu, two eastern provinces at war, hold 9,782 of the cases. [2]

Fighting between the army and armed groups there limits where health workers can go. [2]

Epidemiologists, who study how diseases spread, stop outbreaks by tracing every patient's contacts. [2]

When most new cases come from untraced people, the tracing has fallen behind the virus. [2]

Health workers face attacks too: a nurse was killed answering an Ebola alert last week. [2]

On Friday, staff at an Ebola centre in Tchomia struck over 4 months' unpaid pay. [2]

Fighting between the army and armed groups keeps health workers out, and the two provinces hold 9,782 of the cases. [2]

How far Ebola is spreading in Congo without anyone seeing it.

About 80% of new cases come from people outside the watched contacts, and a third of the dead died before any test. [2]

2,788 people in Congo have recovered from Ebola since the outbreak was declared in May.

Nigeria's disease agency said on Saturday it is testing two suspected Ebola cases. [5]

The agency is the Nigeria Centre for Disease Control and Prevention, NCDC. [5]

The two are in north-east Nigeria and have been isolated. [5]

Officials are listing everyone the two met while laboratory tests run. [5]

Nigeria confirmed its first Ebola case in more than a decade last week. [5]

She was a Swiss woman who had travelled to Uganda, then to Abuja, Nigeria's capital. [5]

She is being treated at Geneva University Hospital, Swiss authorities say. [6]

Nigeria released 10 billion naira, about $6.7 million, in emergency funds after her case. [6]

If confirmed, the two cases could mean Ebola is spreading inside Nigeria. [5]

Epidemiologists keep 'suspected' and 'confirmed' apart: only a laboratory test confirms a case. [6]

Officials have not said whether the two are linked to the first case. [5]

NCDC says it will give more details when the results are in. [6]

Whether Nigeria's two suspected cases are Ebola, and how they got it.

Laboratory tests are still running, and officials have not said whether the two are linked to the Swiss woman who was the first case. [5]

A drug from GSK and Hansoh held back a returning bone cancer longer than chemotherapy did. [7]

Hansoh Pharma, GSK's partner in Shanghai, ran the final-stage trial in China. [7]

It enrolled 122 people aged 12 and over whose osteosarcoma, a bone cancer, had returned. [7]

They were picked at random to get the drug, ris-rez, or two chemotherapy drugs. [7]

On ris-rez, the typical patient went 5.5 months before the cancer grew or they died. [7]

On chemotherapy, the typical patient went 1.5 months. [7]

GSK puts it as a 67% cut in the risk of the cancer growing or death. [7]

Hansoh said in July that the trial had met its main goal, without giving data. [7]

Returning osteosarcoma has no approved drugs and no standard treatment. [7]

In bone cancer, GSK leads Daiichi Sankyo and Merck, whose rival drug is in early trials. [7]

Ris-rez is an antibody joined to a cancer-killing drug, aimed at a marker called B7-H3. [7]

Oncologists, cancer doctors, call this progression-free survival: time before the cancer grows. [7]

93% of patients on ris-rez had severe side effects, against 83% on chemotherapy. [7]

Patients were followed for a typical 7.1 months, and no survival figure was given. [7]

Its drug has a final-trial result in returning bone cancer while Daiichi Sankyo and Merck have only begun early trials there. [7]

Nearly all on the new drug had severe side effects, mostly blood problems, and 4% stopped treatment because of them. [7]

Whether ris-rez helps people with returning bone cancer live longer.

The trial reports how long the cancer was held back after a typical 7.1 months of follow-up, and gives no survival figure. [7]

In GSK's trial, people whose bone cancer had come back went a typical 5.5 months before it grew again on the new drug, against 1.5 months on chemotherapy.

BioMarin will pay $70 million up front for rights to a new Fabry disease drug. [8]

Up to $405 million more follows if the drug meets goals, plus a share of sales. [8]

The drug comes from GC Biopharma and Hanmi Pharm, two South Korean companies. [8]

BioMarin, which bought Amicus Therapeutics for $4.8 billion, already sells a Fabry pill, Galafold. [8]

Fabry disease comes from gene faults that leave people with a faulty enzyme. [8]

The approved drips, Sanofi's Fabrazyme and Chiesi's Elfabrio, are given every 2 weeks. [8]

The Korean drug is a monthly injection under the skin. [8]

It is in an early trial in the US, Argentina and South Korea, testing 3 doses. [8]

If it works, people with Fabry could swap drips every two weeks for monthly shots. [8]

It could also help people whose gene faults Galafold does not suit. [8]

Medicine can replace a faulty enzyme by giving the working protein from outside. [8]

Galafold works differently: it holds certain misshapen enzymes steady so they reach their place. [8]

BioMarin hopes Galafold alone will reach about $1.4 billion a year in sales by the mid-2030s. [8]

Whether the shot works is unknown: the trial measures kidney health for up to 48 weeks. [8]

Its new drug gives the faulty enzyme from outside as a monthly shot, while its pill Galafold helps a misshapen enzyme do its job.

BioMarin pays them $70 million now and up to $405 million later, and they keep the right to sell the drug in South Korea. [8]

A monthly shot would let it compete with Sanofi's and Chiesi's two-weekly drips, beside its own pill. [8]

The approved drips go into a vein every two weeks, and Galafold suits only some gene faults. [8]

Whether the monthly Fabry shot works in people.

It is in an early trial of three doses, with kidney results measured over up to 48 weeks. [8]

A Karolinska Institutet team in Stockholm raised frataxin, a protein patients lack, in mice. [9]

Frataxin runs low in Friedreich's ataxia, an inherited disease that slowly damages nerves. [9]

It is the most common inherited ataxia among Europeans, affecting about 1 in 29,000. [9]

Ataxia means losing control of movement; the disease harms the brain area that coordinates it. [9]

A repeated stretch of DNA in the FXN gene leaves cells with too little frataxin. [9]

Frataxin helps mitochondria, the cell's power units, make energy. [9]

The team used minicircles: small loops of DNA that carry a gene without a virus. [9]

In skin cells from patients, frataxin rose to the level seen in healthy cells. [9]

In mice, frataxin rose 18-fold in the liver and 3-fold in the heart. [9]

HuidaGene carried its editor in a virus, and the virus dose is suspected in a death. [1]

Minicircles carry no virus at all, and the authors call them safer than older DNA carriers. [9]

Gene therapy adds a working copy of a gene so cells can make the missing protein. [9]

This study treated cells and mice, not people. [9]

The mice got the DNA by a high-pressure injection into a tail vein. [9]

They add a working copy of the FXN gene without a virus, and in mice frataxin rose 18-fold in the liver.

In skin cells from people with Friedreich's ataxia, small loops of DNA raised the missing protein, frataxin, to the level of healthy cells.

Ivacaftor, a drug that helps the CFTR protein work, reduced lung scarring in mice. [10]

CFTR is a protein whose full name comes from cystic fibrosis. [10]

The disease studied is pulmonary fibrosis, a deadly illness in which the lungs scar. [10]

The team was at the University of Alabama at Birmingham, with Ningxia University in China. [10]

Lungs of people with the disease hold less CFTR, the authors note. [10]

Ageing of one kind of air-sac cell is a major driver of the scarring. [10]

In mice, the drug limited the build-up of collagen, a tough protein found in scars. [10]

It also restored the lungs' structure and how well they worked. [10]

When CFTR was removed from those cells, the drug worked less well. [10]

That points to the drug working through CFTR itself, the authors say. [10]

Pharmacologists call such a drug a potentiator: it helps a protein do its job better. [10]

The mice's scarring was caused on purpose by a drug, bleomycin, not by the disease. [10]

Grants from China's national science foundation paid for the work. [10]

It helps the CFTR protein work rather than replacing it, tried here in mice with lung scarring.

Drug-industry insiders feel better about the FDA, the US drug regulator, a survey of 569 found. [11]

Endpoints News, a drug-industry news site, ran the survey for July to September. [11]

Its score for regulation rose to 75, from 50 the quarter before. [11]

A year ago, it was 23. [11]

Survey researchers build such an index so 100 means neutral; below it, worriers outnumber the hopeful. [11]

It is the first survey since FDA head Marty Makary quit on 12 May. [11]

Vinay Prasad, the FDA's head of biological medicines, left in April. [11]

Among investors, 40% now call the climate more supportive than a year ago. [11]

But 58% of people at big drug companies still say regulation is getting worse. [11]

Kyle Diamantas has run the FDA as acting head since May. [11]

Heidi Overton, President Trump's pick to lead it, still awaits a US Senate committee vote. [11]

40% of scientists asked expect the rules to get worse within a year. [11]

Only 12% of them expect things to improve. [11]

The US Senate is away until 9 November, after the midterm elections. [11]

Who will lead the US drug regulator, and when.

Heidi Overton still waits for a US Senate committee vote, and the US Senate is away until 9 November. [11]

Anthropic bought Mithril Medicine, a start-up that looks for new drugs. [12]

Anthropic is the AI company that makes Claude. [13]

Mithril had one employee, who worked with AI tools and hired contractors. [12]

It was run by Cody Tranbarger, an entrepreneur-in-residence at the investor Atlas Venture. [12]

He posts on X under the name Professor Oak. [12]

In its share-sale filing on 26 October, Anthropic said it is building its own biology lab. [13]

Anthropic already works with the drugmakers Novo Nordisk and Bristol Myers Squibb. [13]

In business terms, Mithril outsourced: it used hired contractors instead of its own staff. [12]

Endpoints News asked whether drug start-ups will soon run with one employee or none. [12]

Anthropic's filing says drug development brings scientific, regulatory and operational challenges beyond its software business. [13]

Buying Mithril adds a drug-hunting start-up to the biology lab it says it is building. [12][13]

Pakistan has registered over 86,000 medicines on its Track and Trace System. [14]

A phone scan of a pack shows buyers its price and whether it is genuine. [14]

Health Minister Mustafa Kamal gave the figures in Karachi on Saturday. [14]

Registering a medicine once took years; it now takes 21 days, he said. [14]

The Drug Regulatory Authority of Pakistan has handled over 5,000 cases since going digital. [14]

Drugmakers want to raise medicine prices, and Pakistan's government is still considering it. [14]

A phone scan that checks a pack is genuine helps buyers spot fake medicines. [14]

Kamal said pricing 'requires serious attention'; the price request is still under consideration. [14]

With a phone scan they can now check a pack's price and whether it is real. [14]

Whether Pakistan will let medicine prices rise.

Drugmakers have asked for higher prices, and the health minister says the matter is still under consideration. [14]

Scientists in Poland mapped chemical tags on the DNA of lung cancers from 130 patients. [15]