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Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If
Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.
Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.
• Face-to-face event, taking place in London, UK, 26-27th April, will feature two dedicated tracks: ‘Scientific’, focused on seven core therapy areas and ‘Life Sciences Business’ Programme features keynote from George Freeman MP, with an update on the UK Government Life Sciences VisionRegistration for BioTrinity 2022 now available online
Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?
The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.
They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.
50 years after founding, NIDA urges following science to move beyond stigma area Thu, 02/01/2024 - 11:20 Nora's Blog February 1, 2024 Image NIDA Image In 2024, NIDA celebrates its 50th anniversary. The knowledge generated by NIDA research has also led to a robust and rigorous science of drug use prevention.
Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.
Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.
The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. She passed away in 2013.
2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. These can include anti-tubulin compounds, potentially reducing side effects and improving quality of life.
While much attention has been given to the novel science behind cell and gene therapies (CGTs), the success of every precision medicine starts and ends with the patient. Cell and gene therapy patients often require support throughout their treatment journey. Learn key considerations for developing patient support programs.
A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.
Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. But a major hurdle has been finding a safe and efficient way to deliver those genes.
Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.
In the pursuit of a remedy for Alzheimer’s disease, a frontier in medical science is illuminating a glimmer of hope. Stem cell therapies have already demonstrated their prowess in treating diverse cancers and ailments linked to the blood and immune system.
In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.
Regenerative heart therapies involve transplanting cardiac muscle cells into damaged areas of the heart to recover lost function. However, the risk of arrhythmias following this procedure is reportedly high.
Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.
An experimental Alzheimer’s therapy from Cassava Sciences is still being tested in two Phase 3 studies, even as the company has come under regulatory scrutiny.
Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? Investors have historically taken a ‘guilty until proven innocent’ approach to investing in neuroscience therapies. All stakeholders have benefited from these innovations.
Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an international Phase III clinical trial.
A new clinical trial of five children with inherited deafness found administering gene therapy in both ears led to restored hearing and speech, and additional gains including sound source localization, ability to hear in noisy environments, and for two children, abillity to appreciate music.
Scientists engineer CRISPR enzymes that evade the immune system By Allessandra DiCorato January 9, 2025 Breadcrumb Home Scientists engineer CRISPR enzymes that evade the immune system The new genome-editing tools could lead to safer, more efficient gene therapies. Lisa Yang and Hock E. Tan Molecular Therapeutics Center at MIT.
elegans be applied to the development of targeted therapies for neuropsychiatric conditions like eating disorders, OCD, and PTSD? He is also a Professor of Biology at the University of Leuven, a member of EMBO and a Fellow of the Academy of Medical Sciences. How might the understanding of neuropeptide networks in C.
The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. In some cases, cell therapy can be curative for the patient.
The mucus in the airways is not as sticky, inflammation in the lungs significantly reduced: Triple combination therapy can achieve these positive, lasting effects in patients with cystic fibrosis (CF). According to new research, this form of medication improves the symptoms of CF in many patients.
Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?
Bill discusses specialty therapy access barriers, including payer utilization management techniques and “no coverage” policies. Today’s guest post comes from Bill Dupere, SVP of Product Development & Partnerships at Mercalis. Contact Mercalis to learn about their Patient Support Services. Read on for Bill’s insights.
A novel gene therapy for hearing loss was administered to six children in China in a clinical trial. Each child had an inherited deafness caused by mutations in the OTOF gene, called DFNB9.
In April, the FDA warned of risk of secondary cancers in people receiving CAR-T cell therapy. A large study now finds the risk is low and not related to the CAR-T cells.
Lynch, lead author, is now a research fellow in the Department of Veterinary and Animal Sciences. Pearson, co-author of the paper, is now a NEWVEC post-doctoral researcher at UMass Amherst. Credit: UMass Amherst […]
A broad new strategy could hold hope for treating virtually all blood cancers with CAR T cell therapy, which is currently approved for five subtypes of blood cancer. Scientists have demonstrated the potential efficacy of this approach a preclinical study.
A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.
With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.
The quality of science – and the ability to distinguish it from other approaches – was the entry ticket to serious engagement. The science presents an opportunity and a challenge for us at Sionna. The science presents an opportunity and a challenge for us at Sionna. That science resonates with investors.
A team of researchers from the Cancer Science Institute of Singapore (CSI Singapore) at the National University of Singapore (NUS), led by Assistant Professor Anand Jeyasekharan, has discovered a unique combination of oncogenes that could predict treatment resistance, and hence unfavourable outcomes, of patients with Diffuse large B cell lymphoma (DLBCL), (..)
They also found that co-transplantation of neuronal cell therapy with host regulatory T cells resulted in effective suppression of needle trauma and significant improvement in the survival and recovery of grafts. These findings suggest a path for the 'realistic' use of cell therapy to treat neurodegenerative disorders.
Researchers have found that two of the most frequently administered stem cell therapies, which are often used interchangeably, actually contain completely different types of cells.
There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. AAV-based therapies often can’t be dosed more than once due to concerns about an immune response. Molecular Therapy-Nucleic Acids. Human molecular genetics. 2014 Jan 1;3.
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