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By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Its a huge privilege to have the opportunity to point these powerful new tools at our disease.
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. Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. But a major hurdle has been finding a safe and efficient way to deliver those genes.
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.
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.
19, 2024 -- Parkinson's is a relentless disease for which few treatments, and no real cure, exists. Now, researchers say they are on the trail of a potential new therapy for the disabling neurological illness.It's early research. THURSDAY, Dec.
Researchers have developed a new gene switch that can be activated using a commercially available nitroglycerine patch applied to the skin. One day, researchers want to use switches of this kind to trigger cell therapies for various metabolic diseases.
By Count Me In Communications March 11, 2025 Count Me In , a patient-partnered research initiative led by the Broad Institute of MIT and Harvard and Dana-Farber Cancer Institute, is launching a new project focused on translocation renal cell carcinoma (tRCC), a rare and aggressive form of kidney cancer.
Like many of its cell therapy peers, Artiva, which originally sought an IPO in 2021, has shifted its strategy in hopes of riding a recent wave of investor interest in autoimmune diseaseresearch.
29, 2023 -- A new cellular therapy improved learning and memory in mice with Alzheimer's disease, researchers report.The therapy -- developed at the University of Nebraska Medical Center (UNMC) -- relies on both the immune system to. FRIDAY, Dec.
Researchers have demonstrated that a transplant surgical procedure (called 'needle trauma') triggers a profound immune response and causes the death of most grafted dopamine neurons. These findings suggest a path for the 'realistic' use of cell therapy to treat neurodegenerative disorders.
Researchers devised a process to convert a skin cell directly into a neuron, eliminating the need to generate induced pluripotent stem cells. Such neurons could be used to treat spinal cord injuries or diseases such as ALS.
Researchers have discovered the mechanism by which cholesterol in our diet is absorbed into our cells. This discovery opens up new opportunities for therapeutic intervention to control cholesterol uptake that could complement other therapies and potentially save lives.
The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.
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.
The research could revolutionize therapies for complex conditions like autoimmune disease and cancer. Bioengineers have developed a new construction kit for building custom sense-and-respond circuits in human cells.
Researchers have discovered several rare types of helper T cells that are associated with immune disorders such as multiple sclerosis, rheumatoid arthritis, and even asthma. The new T cell atlas is publicly available and should help in the development of new drug therapies for immune-mediated diseases.
The figure depicts the neuroprotective role of exendin-4 in Alzheimer's disease and Parkinson's disease. Due to the complex pathological processes underlying neurodegeneration, at present, there is no viable therapy available for neurodegenerative disorders.
The company’s haul is the latest sign brain drugs are recapturing the interest of investors after years of being passed over for other areas of research.
The advance could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene.
8, 2023 -- Therapies based on a hormone people make while exercising may be the next frontier in treating Alzheimer’s disease, according to a new study. Researchers have found that the exercise-induced hormone irisin may reduce both. FRIDAY, Sept.
What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? 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.
This Q&A explores how pre-clinical research is being used to identify potential therapies for Huntington’s disease, a devastating condition that currently lacks disease- modifying treatments. What makes the potential therapeutic for Huntington’s disease unique compared to other treatments?
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.
Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Our team of chemists then uses this information to develop novel therapies.
Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Because the vehicle binds to a well-studied protein in the blood-brain barrier, the scientists say it has a good chance at working in patients.
Diabetes and cardiac fibrosis are two conditions in dire need of new therapies,” said Todd Golub, director of the Broad Institute. For both patient populations, there are no safe and effective therapies for reversing disease. Florez is an institute member at the Broad, where he directs the Diabetes Research Group.
In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.
Allogene’s partnership with Arbor is designed to help the company stand out in what’s quickly become one of the industry’s hottest areas of drug research.
They found significant associations between the thinning of different retinal layers and increased risk of developing ocular, cardiac, pulmonary, metabolic, and neuropsychiatric diseases and identified genes that are associated with retinal layer thickness. Their findings are published in Science Translational Medicine. “We
Bayer and Broad Institute extend cancer therapyresearch collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapyresearch collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.
Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Fortunately, advances in clinical research are providing hope for better treatments and outcomes.
The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. Schwamborns research focuses on creating patient-specific brain organoids.
Led by Vivek Ramaswamy's brother, Kriya has now raised over $600 million to fund gene therapyresearch spanning ophthalmology, neurology and metabolic disease.
Their work focuses on creating ‘digital twin generators’ – AI-driven models that predict how a patients disease may progress over time. AI-powered digital twins A key component of Unlearns work is its focus on digital twin technology, which uses AI to create personalised models of disease progression for individual patients.
Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Goodsell, RCSB Protein Data Bank and Scripps Research. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Always free.
A personalised approach to obesity treatment Traditional obesity treatments have often followed a one-size-fits-all model, despite mounting evidence that obesity is not a singular disease but rather a collection of subtypes. This growing health challenge increases the risk of diabetes, heart disease, and other conditions.
The figure depicts the neuroprotective role of crocin in Parkinson's disease. Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects.
How can PDOs revolutionise drug discovery and deepen our understanding of disease? Patient-derived organoids (PDOs) are proliferative 3D cell structures derived from tissue samples of both healthy and diseased tissue. Improved translatability to the patient and the disease state.
Scientists link certain gut bacteria to lower heart disease risk By Allessandra DiCorato April 2, 2024 Breadcrumb Home Scientists link certain gut bacteria to lower heart disease risk Study finds several species of cholesterol-metabolizing bacteria in people with lower cholesterol levels.
In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare diseaseresearch and their hopes for 2025.
While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development.
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.
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