Latest news with #geneediting
Reuters
3 days ago
- Business
- Reuters
Nvidia, Dell to supply next US Department of Energy supercomputer
BERKELEY, California - May 29 (Reuters) - The U.S. Department of Energy on Thursday said its "Doudna" due in 2026 will use technology from Nvidia and Dell. The computer, named for Nobel Prize-winning scientist Jennifer Doudna who made key CRISPR gene-editing discoveries, will be housed at Lawrence Berkeley National Laboratory in Berkeley, California. At an event at the lab attended by Secretary of Energy Chris Wright, officials said that the system will use Nvidia's latest "Vera Rubin" chips built into liquid-cooled servers by Dell and will be used by 11,000 researchers.
Bloomberg
3 days ago
- Business
- Bloomberg
Intellia Plunges in Latest Example of Gene Therapy Troubles
Intellia Therapeutics Inc. fell Thursday after disclosing a patient experienced a severe side effect from its gene-editing treatment for a heart condition, the latest safety issue tied to DNA-targeting therapy. The patient, one of more than 200 who have received the Intellia treatment as part of a clinical trial, had elevated liver enzymes, 'which appear to be resolving without hospitalization or medical intervention,' the company said in a filing.
New York Times
4 days ago
- Health
- New York Times
Gene Editing: The Lessons of a Medical Breakthrough
To the Editor: Re 'Custom Gene-Editing Treatment Helps Baby in World's First Case' (front page, May 16): Your article highlighting the remarkable work of Dr. Rebecca Ahrens-Nicklas in developing a bespoke gene-editing therapy for KJ, a child with a rare disorder, is a powerful testament to translational research that bridges the clinic and the lab. It is no coincidence that Dr. Ahrens-Nicklas is a physician-scientist trained in both medicine and research through a program funded by the National Institutes of Health. Dr. Ahrens-Nicklas and I were classmates in the Tri-Institutional M.D.-Ph.D. Program, run jointly by Weill Cornell Medicine, Rockefeller University and Memorial Sloan Kettering Cancer Center. Our peers from this program are advancing our understanding of cancer, H.I.V.-AIDS and other illnesses, each drawing on the unique ability to connect patient care with scientific discovery. These dual-degree programs exist to train precisely the kind of visionary thinkers who can identify unmet clinical needs and then return to the lab to devise novel solutions. This is possible only when scientists understand disease at both the molecular and human level. Recent and proposed cuts to the National Institutes of Health threaten the pipeline that makes such breakthroughs possible. Without strong federal support, we risk losing a generation of physician-scientists — and with them, the kinds of lifesaving advances described in this incredible story.
ABC News
5 days ago
- Health
- ABC News
World-first CRISPR therapy could 'transform' treatment for rare genetic diseases, but key challenges lie ahead
It's been described as a revolutionary technology — and won its inventors a Nobel Prize. CRISPR gene-editing, often simply dubbed CRISPR, is a tool that allows scientists to precisely target and modify the human genome, making it possible to correct mutations and potentially treat genetic causes of disease. Earlier this month, scientists used CRISPR technology to achieve a significant milestone: re-write the DNA of a baby with a rare genetic disease. The patient, a now-10-month-old boy named KJ, is the first person in the world to successfully receive a personalised gene-editing therapy. The landmark case, led by scientists and doctors at the University of Pennsylvania and the Children's Hospital of Philadelphia, was published in the New England Journal of Medicine. Not long after he was born, KJ was diagnosed with a rare, life-threatening genetic disorder called CPS1 deficiency, which affects just one in 1.3 million babies. The disorder is caused by a mutation in a gene that affects a person's ability to properly metabolise protein, and results in toxic levels of ammonia to build up in the body. Unlike other CRISPR treatments, which were designed to be used in multiple people with the same disorder, KJ's therapy was customised to correct his specific disease-causing mutation. "This is a significant advance in our ability to modify human genes," said haematologist and gene therapy researcher John Rasko, who was not involved in the study. While it's too early to know whether the CRISPR treatment will work long-term, researchers say it could provide a blueprint for developing customised gene-editing therapies for others with rare diseases. "While KJ is just one patient, we hope he is the first of many to benefit from a methodology that can be scaled to fit an individual patient's needs,' said lead researcher Rebecca Ahrens-Nicklas from the Children's Hospital of Philadelphia. The high levels of ammonia caused by KJ's CPS1 deficiency can cause severe damage to the brain and liver and even prove to be fatal. The best available treatment for the condition is a liver transplant, but only about half of babies with CPS1 deficiency live long enough to receive one. Scientists at the University of Pennsylvania had been investigating gene-editing therapies for similar genetic disorders and when KJ was diagnosed, they quickly mobilised to create a treatment to fix his specific mutation. To do this, they used a "genetic engineering trick" called CRISPR base editing, a second-generation CRISPR tool, said Marco Herold, CEO and head of the Blood Cancer and Immunotherapy Lab at the Olivia Newton-John Cancer Research Institute. "The researchers identified through [genome] sequencing that this mutation was the result of a change in DNA bases," Professor Herold, who was not involved in the study, told The Health Report. DNA sequences are made up of four different "letters" which represent different chemical bases. The order of these letters or bases determines the genetic information carried in the DNA. "[CRISPR technology] scans the DNA and runs over all the letters until it encounters the wrong letter — it can be programmed to find this," Professor Herold said. Unlike traditional CRISPR medicines, which bind to the target DNA, cut it, and silence or repair a problematic gene, base editors convert target DNA from one letter into another. "In this case, the letter was an A and it had to be changed into a C … and that leads to the repair," said Professor Herold, whose own research focuses on CRISPR screening and editing. KJ, who had been on a highly restrictive diet since birth, as well as medication to remove ammonia from his blood, was given a small first dose of the novel gene-editing therapy at seven months of age. Over the next two months, he received two more infusions at higher levels. Since the treatment, he's been able to eat a full protein diet and take just half his usual medication — a sign the therapy has, at least partially, reversed his disease. 'While KJ will need to be monitored carefully for the rest of his life, our initial findings are quite promising,' Dr Ahrens-Nicklas said. Professor Rasko, chair of the federal government's advisory committee on gene technology, said the speed at which the drug was developed was "extraordinary". But he stressed that longer follow up was needed to assess its safety and efficacy, and determine whether additional doses would be necessary. "These are very early days," Professor Rasko said. "Everything is looking great but let's wait a year and see what's going on." It's estimated there are more than 5,000 genetic diseases, which, while rare individually, affect hundreds of millions of people worldwide. In Australia, around two million people — or 8 per cent of the population — live with a rare disease, 80 per cent of which have a genetic cause. But the lack of economic incentive for pharmaceutical companies to develop drugs for extremely rare conditions has led to a scarcity of effective treatments, Professor Rasko said. "Of the 5,000 plus rare genetic diseases, we have a treatment that's specific for less than 5 per cent," he said. Peter Marks, who until recently was responsible for overseeing gene-therapy regulation at the US Food and Drug Administration, described KJ's therapy as potentially "transformational" for the treatment of rare genetic diseases. "Although not all rare diseases may be eligible for a gene-editing approach … there could be hundreds to thousands of diseases that could be treated through an approach similar to the one described," he wrote in an editorial for the New England Journal of Medicine. While KJ's treatment was targeted to his specific mutation, Dr Marks said the same technology could be adapted and "customised" to correct other rare genetic mutations, reducing the cost and complexity of developing new drugs. Professor Herold agreed the same approach could be taken to treat other illnesses caused by a single mutation, with only the CRISPR instructions needing to be changed. "But if you have multiple mutations … there are a lot of diseases that are made up of four, five, six different mutations, then it becomes difficult," he said. "We are not there yet, but we're working at this." Despite the promising results seen in KJ's case, there are several key challenges that need to be addressed before personalised gene-editing could be scaled up and expanded. For one, developing treatments that can successfully reach parts of the body other than the liver — where KJ's mutation occurred — is more difficult, and will require further research. "Because the liver is like a big sieve that processes poisons, toxins, and manufactures hormones and other proteins … the lipid nanoparticles [which encase the gene-editing products] get taken up there," Professor Rasko said. Even though KJ's treatment was a "breathtakingly impressive" proof of concept, replicating and adapting it for other patients would still be resource intensive, he added. "Every time we do this, we have to alter the guide DNA and the technology has to change. It has to be quality-controlled, it has to pass some form of regulatory approval … it's not just a one size fits all." A more established therapeutic approach called gene addition therapy, which involves introducing a working copy of a gene (rather than correcting one), had been a "scientific and medical success" but "an economic failure" to date, Professor Rasko said. "Companies that have been valued at billions of dollars have had to walk away because they can't recoup their costs without charging millions of dollars a pop for these genetic therapies," he said. But, he said, the rate of development and innovation in the field of gene editing — which may help to solve some of the challenges — was "awesome". "You just can't keep up." Listen to the full story on Radio National and subscribe to the Health Report podcast for more.
Associated Press
5 days ago
- Business
- Associated Press
Advancing access to clinical trials for cardiomyopathy
( NewMediaWire ) - May 27, 2025 - DALLAS — Despite scientific advances in cardiovascular care, people in living in rural areas and other communities with long-term economic or social challenges still face barriers to cutting-edge therapies such as gene editing, according to a 2020 American Heart Association presidential advisory. To address gaps in care, the Association, devoted to changing the future of health for all, is using its Get With The Guidelines(R) data to support patient populations that may be underrepresented or overlooked in clinical trials. Get With the Guidelines programs connect hospitals with current evidence-based guidelines and accurate measurement tools to improve care quality and industry practices. As part of this work, the Association today announced an initiative to improve education, outreach and access to clinical trials for gene editing therapies for transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a progressive and often underdiagnosed condition that disproportionately affects older adults and certain racial and ethnic groups. In ATTR-CM, a misshapen form of the protein transthyretin builds up in the heart, preventing the left ventricle from relaxing and filling properly. Over time, this can impair the heart's ability to pump blood, leading to heart failure. This new effort builds on the American Heart Association's ongoing commitment to improving diagnosis, care and outcomes for people with cardiomyopathy. The objectives are to close knowledge gaps, raise awareness and improve access to early diagnosis and emerging treatments for cardiac amyloidosis — no matter where people live — through research, clinical education and public outreach. The nationwide initiative, conducted with financial support from Intellia Therapeutics, aims to elevate understanding of gene editing, advance research and support clinical trial opportunities among populations often excluded from research. 'Too many people remain unaware of or disconnected from lifesaving cardiovascular clinical trials,' said Michelle Kittleson, M.D., PhD, American Heart Association volunteer, professor of medicine at Cedars-Sinai and director of education, heart failure and transplantation at Smidt Heart Institute. 'This effort is designed to help close that gap, ensuring that medical innovation is matched with education, trust and opportunity for all.' The initiative includes a multi-pronged education and research initiative to assess current ATTR-CM awareness and develop new insights related to gene editing and cardiovascular disease. These observations will inform new educational materials, a series of national webinars and outreach strategies designed for patients, families and clinicians. The first webinar, Understanding Amyloidosis & Emerging Therapeutic Frontiers, will be held June 18, and will feature experts in cardiology and gene therapy. Additional components of the initiative focus on enhancing patient identification for access to emerging therapies and clinical research opportunities. This includes activating a referral network of non-trial sites, supporting multidisciplinary provider education and developing tools that leverage clinical data to help identify potentially eligible participants. Additional Resources: ### About the American Heart Association The American Heart Association is a relentless force for a world of longer, healthier lives. Dedicated to ensuring equitable health in all communities, the organization has been a leading source of health information for more than one hundred years. Supported by more than 35 million volunteers globally, we fund groundbreaking research, advocate for the public's health, and provide critical resources to save and improve lives affected by cardiovascular disease and stroke. By driving breakthroughs and implementing proven solutions in science, policy, and care, we work tirelessly to advance health and transform lives every day. Connect with us on Facebook, X or by calling 1-800-AHA-USA1. For Media Inquiries: 214-706-1173 Michelle Rosenfeld: 214-706-1099; [email protected] For Public Inquiries: 1-800-AHA-USA1 (242-8721) and
