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UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms

UniQure’s highly promising Huntington’s disease gene therapy BLA (biologics license application) was rejected by the FDA — because UniQure used an external control rather than a surgical sham control. Yet the latter would put control group patients at additional risk, making it ethically problematic. Hopefully some agreement will be reached which circumvents these issues! For now, it is an educational story to watch unfold.

Abi-Saab said during uniQure’s earnings call that he wouldn’t count on the four-year data altering the FDA’s decision.

“We don’t believe that there’s any reason we have today to believe that this will change the FDA’s position regarding the Phase 1/2 trials,” he told investors.

H.C. Wainwright struck a different tone, however, in the Monday note. “While the FDA appears to be enforcing a full sham surgery-controlled Phase 3 trial in Huntington’s, we believe an alternative path forward may be negotiated given the strong AMT-130 data generated to-date,” the analysts wrote. The 4-year data “should further inform the durability and magnitude of effect observed to date.”

A humanoid robot sprints past the human half-marathon world record in Beijing race

The winner from Honor, a Chinese smartphone maker, completed the 21-kilometer (13-mile) race in 50 minutes and 26 seconds, according to a WeChat post by the Beijing Economic-Technological Development Area, also known as Beijing E-Town, where the race kicked off.

That was faster than the human world record holder, Uganda’s Jacob Kiplimo, who finished the same distance in about 57 minutes in March at the Lisbon road race.

The performance by the robot marked a significant step forward from last year’s inaugural race, during which the winning robot finished in 2 hours, 40 minutes and 42 seconds.

Systematic discovery of pro- and anti-HIV host factors in primary human CD4+ T cells

Now online! Genome-wide CRISPR activation and knockout screens in primary human CD4+ T cells systematically identify host proviral and antiviral factors modulating HIV infection, including the strongly antiviral factor PPID, which is shown to bind HIV capsid and reduce its nuclear import.

Stem cell gene editing to produce B cell protein factories

As a proof of concept, the team used CRISPR gene-editing tools to insert the genetic blueprint for producing rare, protective antibodies directly into hematopoietic stem and progenitor cells of mice. Once transplanted back into mice, the edited stem cells gave rise to B cells programmed to produce the engineered antibody. A conventional vaccination would then serve as the trigger.

It worked. Even when only a few dozen stem cells were edited, vaccination triggered rare cells to expand, mature into plasma cells, and produce large amounts of antibodies that persisted long-term and could be boosted if necessary. The engineered B cells behaved just like normal immune cells, and even provided protection from disease. Mice engineered to produce a broadly neutralizing influenza antibody were spared from an otherwise lethal influenza infection.

The team went on to demonstrate their novel platform’s versatility. Engineered B cells were able to secrete non-antibody proteins, pointing to potential applications in treating genetic diseases caused by missing enzymes or other essential proteins.

The researchers also showed that stem cells carrying different antibody instructions could be combined, enabling a single immune system to produce multiple antibodies at once—an approach that could limit viral escape and ultimately lead to functional cures for rapidly mutating pathogens such as HIV.

And the team showed that human stem cells edited using the same approach gave rise to functional immune cells, providing a key proof of feasibility that the platform could one day work in humans, as well. Science Mission sciencenewshighlights.

An innovative gene-editing strategy could establish a new way for the body to manufacture therapeutic proteins—including certain kinds of highly potent antibodies the are naturally difficult to produce—by reprogramming the immune system itself.

Continue reading “Stem cell gene editing to produce B cell protein factories” | >

Blood test detects aggressive brain tumors early and could reduce need for risky surgery

Researchers at the University of Sussex, in collaboration with scientists from different institutes worldwide, have identified a blood test capable of early diagnosis of the most aggressive form of brain tumor. The technology has the potential to save lives. Lead author Professor Georgios Giamas and his team have identified distinctive biomarkers (molecules that act as signs of normal processes, diseases, or responses to treatment) within patient blood samples, which could signal the presence of glioblastoma, one of the most aggressive forms of brain tumor.

The study published in Cell Reports Medicine investigated whether a simple blood test—analyzing the cargo of tiny particles called small extracellular vesicles (sEVs) that are released by cells into the bloodstream—could accurately detect and classify these tumors.

More than 11,000 people are diagnosed with a primary brain tumor in the U.K. each year. Glioblastoma is the most common high grade primary brain tumor in adults, which means it can grow and spread exceptionally quickly. Currently, diagnosing glioma often requires risky brain surgery.

Primary sclerosing cholangitis

Primary sclerosing cholangitis is a rare, chronic cholestatic liver disease characterised by biliary inflammation and fibrosis. Inflammatory bowel disease co-occurs in 50–80% of individuals with primary sclerosing cholangitis and there is an increased risk for hepatobiliary and colorectal cancers. Primary sclerosing cholangitis presentation is highly variable but there is usually a slowly progressive fibrosis of the bile ducts with strictures, development of liver fibrosis and cirrhosis, and eventually a need for liver transplantation, after which primary sclerosing cholangitis can reoccur.

Sinus MCs are enriched in burn pit–exposed military veterans with CRS and in mice exposed to environmental combustion-related compounds

Address correspondence to: Taylor A. Doherty, UCSD, 9,500 Gilman Drive, La Jolla, California 92093–0635, USA. Phone: 858.822.7563; Email: [email protected].

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¹VA San Diego Healthcare System, La Jolla, California, USA.

How Automation and AI Are Transforming Organoid Research

The life sciences are in the midst of a crucial shift, driven by the emergence of organoid-based models and the power of automation. Organoids—three-dimensional cell cultures that mimic human tissue architecture and function—are enabling researchers to ask and answer questions that were once beyond reach. Paired with advances in automation, robotics, and artificial intelligence (AI), these models are transforming drug discovery and preclinical testing, offering a more human-relevant alternative to outdated 2D cell cultures and animal models. This revolution is reshaping the pharmaceutical industry, while also holding the potential to accelerate progress in personalized medicine.

Beyond 2D: The Rise of Organoids

For decades, preclinical research has relied on 2D cell cultures, single-cell-type 3D spheroid models, and animal models, despite their limitations in replicating human biology. Organoids, which are derived from stem cells, offer a more accurate representation of human tissues, recapitulating complex biological processes such as organ-specific functionality and cellular interactions. These miniature self-organizing biological systems are being used to model diseases, test drug efficacy and toxicity, and even explore regenerative medicine.

What a Neutron Star Is Really Made Of

What happens to matter when it’s crushed beyond the point where atoms can exist? Inside a neutron star, the densest visible object in the universe, matter is compressed into states so extreme that physicists still don’t fully understand what’s there.

In this calm long-form space documentary, we take a journey layer by layer through the interior of a neutron star — from the crystalline crust where exotic nuclei form structures unlike anything on Earth, through the bizarre \.

Universal Quantum Computing as a Markov Chain

Let’s say you have a probabilistic computer with a single bit of memory. Some algorithms on the computer will stochastically flip the single bit of memory such that its new value will be uniformly distributed with a 50% chance of being 0 and a 50% chance of being 1. Other programs will place it into a degenerate distribution, meaning it either has 100% chance of being 0 every time you run the program, or other programs will produce 1 100% of the time.

A magician tells you to run one of the programs in one of the two categories of your choosing and then copy the computer’s memory state onto a thumb drive and hand it to him. You pick one, run the program, copy the bit of the memory to your thumb drive, then hand it to the magician. The magician then does something with the thumb drive you cannot see, then looks up at you and tell you exactly what category the program you ran to produce that bit came from.

Curious, you repeat this many times over: you run a program from one of the two categories (degenerate or uniform), copy the bit value produced from the algorithm, and then hand the thumb drive to the magician. Each and every time he always correctly guesses which category of program was ran to produce it.

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