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Forbes
3 days ago
- Health
- Forbes
Second Patient Death After Gene Therapy: Implications For The Future
As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics announced a second patient death following treatment with its gene therapy, Elevidys, for Duchenne muscular dystrophy. This news has sent shockwaves through the rare disease and biotech communities. It raises questions about the safety of adeno-associated virus-based gene therapies. The latest fatality, a 15-year-old, died from acute liver failure, a recognized but rare complication of adeno-associated virus-based gene transfer. This death follows a similar case reported in March. These recent tragedies have profound implications. Elevidys, Sarepta's gene therapy, was among the first to receive approval for the treatment of Duchenne muscular dystrophy. Yet, the deaths of two patients treated with Elevidys now force us to confront a difficult question. Do the potential benefits of this therapy truly outweigh the risks, particularly for those who can no longer walk and may be at higher risk of complications? Every advance in medicine brings new challenges, and gene therapy is no exception. The deaths associated with Elevidys have raised the regulatory bar for all investigational gene therapies that use adeno-associated virus vectors. This means more stringent safety monitoring, more careful patient selection, and better reporting of outcomes. This heightened scrutiny is not unwelcome, even if these measures may slow the pace of development in the short term. They are essential for ensuring safety and responsibility. Adeno-associated virus-based vectors have become the workhorse of gene therapy due to their efficiency and relatively low risk of complications. The field has long recognized that high-dose adeno-associated virus-based administration can trigger severe, sometimes fatal, immune-mediated toxicities. These primarily affect the liver. In 1999, Jesse Gelsinger died after receiving an adenoviral gene therapy. His tragic death marked a turning point in the field and led to decades of increased scrutiny and regulation. While adeno-associated viruses are generally less immunogenic than adenoviruses, high doses can activate the body's immune system. This leads to inflammation, liver injury, and, in rare cases, organ failure. Recent studies highlight that immune responses can contribute to toxicity, particularly in the liver. However, not all patients are impacted equally. Recent findings underscore that age and disease status can significantly influence a patient's response to adeno-associated virus-based gene therapy. Clinical studies reveal that individual factors, such as underlying infections, pre-existing liver disease, or genetic predispositions, can amplify the risks. For example, in the first reported Elevidys death, a concurrent infection may have played a role in the patient's rapid decline. Research from the University of Bristol indicates that in ocular gene therapy trials, older patients, particularly females, are more likely to experience severe inflammatory responses. However, in the Elevidys cases, both fatalities involved males who had lost the ability to walk due to advanced disease progression. This is a critical factor linked to heightened immune vulnerability. This underscores that risks are context-specific, depending on therapy type, delivery site, and patient health status. To address the pressing safety concerns, Sarepta is investigating several strategies. One approach under consideration is the addition of sirolimus, an immunosuppressant widely used in organ transplantation. While this aims to reduce immune-mediated liver damage, experience from liver transplant patients warns that sirolimus can nearly triple infection risk, even at low doses. Its use in gene therapy remains experimental and demands careful evaluation before it can be widely adopted. At the same time, non-viral delivery methods are emerging as a promising alternative. Lipid nanoparticles offer a way to bypass many of the immune challenges posed by viral vectors. Their ability to be tailored for precise tissue targeting and the absence of pre-existing immunity in patients make them particularly attractive for conditions like Duchenne muscular dystrophy, where repeated dosing may be necessary. Though still less efficient than viral vectors in some applications, the technology is advancing quickly. The second death following Elevidys for Duchenne muscular dystrophy is a sobering reminder of the risks inherent in adeno-associated virus-based gene transfer, particularly for older or more severely affected patients. Still, these tragedies are not new to the field. We have seen similar inflection points before. The history of gene therapy is marked by moments of triumph and tragedy. Each shapes the path forward. As we reflect on these recent events, it is clear that the field stands at another such crossroads—one that will require careful balancing of hope, caution, and scientific rigor to chart the best course for patients and the future of medicine. For a deeper exploration of these issues, including real-world patient stories and the latest scientific advances, see Destiny's Child No Longer: Rewriting Genetic Fate, which provides a detailed examination of this therapy and other gene therapies.
Hans India
11-06-2025
- Business
- Hans India
Kadapa student secures admission in University of Edinburgh
Kadapa: Kadapa native Poornima Nimmakayala has secured admission to the world-renowned University of Edinburgh in the United Kingdom for an MS in Mathematical Economics and Econometrics. Notably, she achieved this feat with an impressive IELTS score of 8, without any formal coaching. The university, ranked 27th in the QS World University Rankings 2025, has issued her student ID: 2858866, and her admission is scheduled for July 28. She also got offers from the University of Bristol and King's College London but chose Edinburgh. Poornima is the daughter of N Ravishankar Reddy, State Secretary of the Rayalaseema Communist Party, and K Anita of Adinimmayapalle village, Vallur Mandal. Her achievement has earned widespread appreciation.
Yahoo
11-06-2025
- Science
- Yahoo
'We've got a new mystery on our hands': Titan's weird wobble just got even stranger
When you buy through links on our articles, Future and its syndication partners may earn a commission. For years, scientists have been intrigued by a weird "wobble" in the atmosphere of Titan, Saturn's largest moon. Now, new research is revealing clues about Titan's strange atmospheric tilt, but it's also raising new mysteries. Data from the Cassini mission to Saturn has shown that, unlike Earth's atmosphere, Titan's atmosphere doesn't rotate in sync with its surface. Instead, it tilts and shifts like a spinning top that changes its orientation with the seasons. "The behaviour of Titan's atmospheric tilt is very strange," Lucy Wright, lead author of the new research and a postdoctoral researcher in the School of Earth Sciences at the University of Bristol in the U.K., said in a statement. "We think some event in the past may have knocked the atmosphere off its spin axis, causing it to wobble." Scientists thought the direction of the tilt would be influenced by either Saturn's gravity or the position of the sun, as is often the case in planetary systems — meaning it would change as Titan orbited Saturn and the sun. But observations show that the tilt direction doesn't move. Instead, it stays pointed the same way in space, as if unaffected by those external forces. This finding was unexpected. If solar heating or Saturn's gravity were controlling the tilt, it should move over time. Instead, the tilt seems locked in place, suggesting that some other, still-unknown process is at work, the researchers reported in a study published May 20 in The Planetary Science Journal. "That would've given us clues to the cause," Nick Teanby, co-author of the study and a planetary scientist at the University of Bristol, said in the statement. "Instead, we've got a new mystery on our hands." Titan is the only moon in the solar system known to have a substantial atmosphere. It is composed primarily of nitrogen and contains complex organic molecules, making it a scientifically important object for studying things like atmospheric processes and prebiotic chemistry. RELATED STORIES —Saturn's moon Titan may have a 6-mile-thick crust of methane ice — could life be under there? —There's liquid on Titan, Saturn's largest moon. But something's missing and scientists are confused —Alien life could exist on Saturn's big moon Titan — but finding it will be tough From 2004 to 2017, NASA's Cassini spacecraft closely observed Titan, revealing important changes in its atmosphere, like a steady tilt in the middle layers and the appearance and disappearance of swirling winter polar vortices. This new understanding of Titan's atmospheric wobble is important for NASA's upcoming Dragonfly mission, which is set to arrive in the 2030s. Because Titan's winds are much faster than its surface rotation, knowing how the atmosphere shifts with the seasons will help engineers more accurately plan Dragonfly's descent and landing. "Our work shows that there are still remarkable discoveries to be made in Cassini's archive," study co-author Conor Nixon, a planetary scientist at NASA's Goddard Space Flight Center, said in the statement. "This instrument, partly built in the U.K., journeyed across the Solar System and continues to give us valuable scientific returns. "The fact that Titan's atmosphere behaves like a spinning top disconnected from its surface raises fascinating questions — not just for Titan, but for understanding atmospheric physics more broadly, including on Earth," Nixon added.
Yahoo
10-06-2025
- Business
- Yahoo
Student funding to create generation of AI pioneers
A new scholarship aimed at developing the next generation of artificial intelligence "pioneers" will open to applicants in spring 2026. The Sparck AI scholarships, named after pioneering British computer scientist Karen Sparck Jones, will give master's degree university students access to industry-leading firms as the Government looks to boost the UK's AI credentials. The University of Bristol will be one of nine universities to offer the fully-funded Government scholarship, alongside faculties in Newcastle, Manchester and Edinburgh. The university said they "relish the opportunity," with the grant covering both students tuition and living costs. More news stories for Bristol Watch the latest Points West Listen to the latest news for Bristol The scheme has been developed in line with the Government's 'AI Opportunities Action Plan', with more than £17m of funding from Westminster. Alongside masters places, 100 scholars will receive placements in leading AI companies, as well as mentorship from industry experts. In 2024, Bristol was named 'AI University of the Year' and has developed the Isambard-AI computer - the most powerful of its kind in the country. Vice-chancellor, Evelyn Welch said the scholarships would give Bristol the chance to "explore bold new ideas and nurture exceptional talent." It is hoped the scholarships will give students "unparalleled access" to the fast-moving industry. Technology Secretary Peter Kyle MP said he believed the scheme would help students secure "highly skilled jobs" and build "a workforce fit for the future." Finn Stevenson, Co-founder and Chief Executive of Flok Health said they were "delighted" to be part of the scheme, adding attracting the "world's best talent" was vital in aiding AI advancement in the UK AI talent acquisition firm, Beamery said the scholarships would help their goal to create "equal access to work" and connect "talent to opportunity". Applications open in Spring 2026, with the first cohort beginning studies the following autumn. Follow BBC Bristol on Facebook, X and Instagram. Send your story ideas to us on email or via WhatsApp on 0800 313 4630.
Yahoo
09-06-2025
- General
- Yahoo
Iconic building's 100th anniversary celebrated
The 100th anniversary of one of Bristol's most iconic landmarks has been celebrated. The Wills Memorial Building was built as a memorial to the tobacco magnate Henry Overton Wills and opened by King George V and Queen Mary on June 9 1925 in a ceremony that saw thousands lining the streets. Construction of the building, seen by many as the heart of the university area of the city, began in 1915 but was delayed by the First World War. It was one of the last gothic buildings to be built in England and attracted national attention in the run-up to its grand opening. The University of Bristol marked the centenary with a special ringing of the tower's bell and commissioned a poem by Dr Lawrence Hoo that references the building's links to the transatlantic slave trade through the Wills family. "If law made it legal, does that make it right, would justice have weight, if it only wore white?" it reads. "We have newspaper cuttings from across the Bristol and national press showing pictures of the king and queen and events from the day - it just shows the level of interest there was across the country," said Rachel Gardner, Senior Archivist at the University of Bristol. "The stories focus on the famous people you could see at the procession but also on people like Granny Mary Jarrett, who was 104 years old and was given special permission to ride in a taxi as part of the procession, and met the king." "There's also a lot about how the citizens of Bristol were so well behaved and only a handful of wallets went missing that day so everyone should be very proud of themselves. "It was specifically requested by the king that there wasn't a costly decorations scheme but the mayor wrote in the press asking everyone to decorate as freely as possible - it really involved the whole city," she added. More news stories for Bristol Watch the latest Points West Listen to the latest news for Bristol The building narrowly avoided destruction just 15 years after its completion when incendiary bombs dropped during The Blitz caused the roof of the Great Hall to collapse - leaning burn marks on the floor that can still be seen today. Winston Churchill - who was chancellor of the university - inspected the damage the next day and insisted that a ceremony to award honorary degrees still went ahead. The Great Hall has long been repaired and now welcomes hundreds of students for graduation ceremonies every year. It has also welcomed some famous people to receive honorary degrees, including James Blunt and the former leader of the Soviet Union, Mikhail Gorbachev. The building's opening was marked by 21 chimes from Great George - the 9.5 tonne bell that sits at the top of the tower. To mark the anniversary the university organised a special manual ringing, as it would have been done in 1925. The bell is the third largest that can be manually swung in the country, and when that happens it can be heard much further than its normal two-mile radius. "When we get the bell ringing there is a lot more energy involved, and the speed the clapper hits is much higher," said Matthew Tosh, bell ringer with the University of Bristol Society of Change Ringers. "But getting the bell that high is really physically demanding and even with the strongest, heaviest big bell ringers we've never managed to get it more than half way up," he added. Great George is normally only rung manually for special state occasions such as royal deaths and coronations. Follow BBC Bristol on Facebook, X and Instagram. Send your story ideas to us on email or via WhatsApp on 0800 313 4630. No change for uni slave trade-named buildings Students want slave trader building to be renamed City's slavery links to be examined
