The NRG Oncology trial found that proton therapy did not improve overall survival or progression-free survival for patients with advanced hepatocellular carcinoma. However, fewer grade 3 or higher treatment-related adverse events were observed with proton therapy compared to photon therapy.
A large U.S. insurance claims analysis found that fewer than 30% of patients with hormone receptor-positive early-stage breast cancer remained adherent to endocrine therapy for five years after lumpectomy. Younger age and radiation therapy omission were associated with lower adherence rates.
A novel autologous gene therapy for Wiskott-Aldrich syndrome has demonstrated durable clinical benefits in a phase 3 study, with 96% survival at 1 and 5 years. The treatment, etu-cel, has also reduced severe infections and moderate-to-severe bleeding events.
A large Danish study found that current use of oral menopausal hormone therapy is associated with an increased risk of blood clots in legs/lungs, with no general increased risk seen with transdermal hormone therapy. Stroke and heart attack risks were limited to prolonged high-dose oral hormone therapy.
The FDA approved FAYUVI, a gene therapy treatment for pediatric patients with Sanfilippo Syndrome Type A, following years of research at the Abigail Wexner Research Institute. The treatment, developed by Nationwide Children's Hospital, is the fourth gene therapy to gain FDA approval from the institution.
A US study of 20 years of health data found that hormone therapy initiated during peri- or early postmenopause reduces cardiovascular disease risk by 22% compared to those who did not use hormone therapy. The protective effect was strongest among Black women and those who initiated therapy within 10 years of menopause onset.
Researchers at Murdoch Children's Research Institute have made a breakthrough in gene therapy, restoring heart function in lab-grown patient heart tissue and mouse models with genetic heart disease. The therapy may also correct other genetic heart diseases, including those caused by the MYH7 gene and TTN truncating variants.
Researchers at Dongguk University have developed an innovative gene switch that uses electromagnetic fields to control gene expression. The switch, which targets the Lgr4 gene, demonstrates precise activation with no detectable adverse effects, making it a promising platform for non-invasive gene therapies.
Researchers at UMass Chan Medical School have developed a microRNA-based gene therapy that suppresses mutant SOD1 production, delaying disease onset by 60 days and extending lifespan by 100 days in mice models of ALS. The therapy, delivered via adeno-associated virus (AAV) vector, preserves motor neurons and maintains neuromuscular con...
A new randomized clinical trial suggests that singing therapy provides greater short-term symptom relief and improved quality of life compared to diaphragmatic breathing exercises for people with supragastric belching. Structured singing therapy also resulted in higher response rates and more enjoyable experiences among patients.
The study demonstrated sustained improvements in disease features in mouse models with effects lasting throughout their lifespan. Genespire's approach has the potential to translate into human health as a single-administration treatment for patients with MMA.
Researchers developed a gene therapy that restored normal brain activity and improved behavior in mice with Fragile X syndrome by replacing the missing FMRP protein. The treatment administered during early development showed significant improvements in cognitive flexibility, social interactions, and probabilistic reversal learning.
This review provides a comprehensive overview of gene therapy tools and strategies for hereditary hearing loss. Advances in inner ear gene delivery platforms, including AAV vectors and cell-specific promoters, have improved the translational development of gene therapies.
A gene therapy developed at UAB prolongs health span and preserves the function of multiple organs in mice during aging, with sustained beneficial effects across multiple endpoints. The treatment shows improved metabolism and organ function, including weight management, insulin sensitivity, and increased energy expenditure.
Malcom Brenner, a renowned physician-scientist, will lead the new center. He brings expertise in cell and gene therapy to advance therapies for various diseases.
The center is the first in the region to offer TCR-T therapy, an FDA-approved treatment targeting proteins inside cancer cells. Patients with chemotherapy-resistant synovial sarcoma can now access this novel cellular therapy.
A new study by the American Society of Hematology finds that stem cell transplantation is the most cost-effective option for long-term care of sickle cell disease in adults. Gene therapy, while promising, comes with an astronomical cost and requires a significant price reduction to be competitive.
A new machine-learning model uses pre-therapy PET/CT scans to estimate radiation dose to tumors and healthy organs, improving patient selection and reducing toxicity risk. The study's findings suggest a promising ability to predict post-therapy dosimetry, which may optimize treatment plans for individual patients.
Odylia Therapeutics is advancing a gene therapy treatment for Usher Syndrome Type 1C (USH1C)-associated vision loss, a rare autosomal recessive disorder. The grant will support the development of an investigational USH1C gene therapy designed to restore or preserve vision.
Toloo Taghian, a UMass Chan Medical School assistant professor, has received a five-year, $3.2 million grant to develop a gene therapy for UBA5 disorder, a rare genetic disease that affects protein balance and leads to neurological impacts. The goal is to monitor disease progression and determine the efficacy of future clinical trials.
A new international study shows that gene therapy targeting the OTOF gene successfully restored hearing in most participants, with significant improvements in speech perception and language skills. The treatment remained safe and effective for up to 2.5 years, offering new hope for individuals with inherited deafness.
Researchers modeled gene drive dynamics in plants and found that seed banks can hinder the spread of genetic changes. The simulations suggest that proper consideration is needed to mitigate the impact of stored seeds on the gene drive's success.
Mohamed Abou-el-Enein, a USC physician-scientist, has received the Outstanding New Investigator Award and Best of Molecular Therapy Award from the American Society of Gene + Cell Therapy. His work focuses on translating scientific discoveries into clinical applications, with a particular emphasis on cell therapies.
A new study shows that PSMA therapy can delay the need for hormone therapy in prostate cancer patients by an average of 20 months. The therapy is effective in earlier stages of the disease, improving survival and quality of life for approximately two out of three patients.
The FDA has approved a gene therapy for severe leukocyte adhesion deficiency-I, a rare genetic condition that affects one in a million children globally. The therapy, Kresladi, has been shown to significantly reduce severe infections and improve immune function in treated patients.
The company's ATA-200 gene therapy has shown safety, pharmacodynamics, and efficacy results in the first patients treated, offering hope for children with LGMD-R5. The therapy delivers a normal copy of the γ-sarcoglycan gene and has been awarded Orphan Drug Designation in the US and Europe.
Researchers at the University of Basel have developed a gene therapy that can potentially treat LAMA2-related muscular dystrophy, a rare and fatal muscle disease in children. The therapy has been shown to stabilize muscles and nerves and halt disease progression in animal models, with a single treatment being sufficient.
A large Danish study found that menopausal hormone therapy is not associated with an increased risk of death in women with moderate to severe symptoms and no contraindications. The study also showed a significant survival benefit for women who had undergone bilateral oophorectomy after using hormone therapy.
UCLA researchers have developed a novel gene-editing approach using lipid nanoparticles to deliver a full-length CFTR gene into human airway cells. The study shows promise for treating cystic fibrosis by correcting the underlying genetic mutation, which could lead to more effective and long-term therapies.
A bibliometric analysis of global advances in cell and gene therapy reveals uneven progress, with US and China leading the field. Japan's contributions are significant but lack qualitative influence.
A new study by Mass General Brigham highlights the efficacy of proton therapy in achieving excellent tumor control rates while minimizing side effects compared to traditional radiotherapy. Proton therapy limits radiation exposure to healthy tissue, potentially preserving cognition and hormone function.
Researchers at the University of California - San Diego have developed a new method to improve gene therapy by increasing the efficacy of gene delivery while minimizing harmful side effects. The new workflow allows for increased control of nuclear DNA delivery, with greater than tenfold increase in nuclear DNA delivery observed.
Researchers developed AAVLINK, harnessing Cre/lox-mediated intermolecular DNA recombination to enable in vivo reassembly of large genes. The method achieves high-efficiency full-length gene reconstitution and significantly improves therapeutic outcomes in animal models.
The AAVLINK method harnesses Cre/lox-mediated intermolecular DNA recombination to enable efficient full-length gene reconstitution and robust expression of therapeutic genes. This approach overcomes the limited packaging capacity of adeno-associated viruses, offering a strategy for delivering large gene cargoes in gene therapy.
Pompe disease is a rare genetic disorder caused by a deficiency in the GAA enzyme, leading to glycogen accumulation in cells. Genethon's gene therapy approach has shown preclinical efficacy in animal studies, correcting glycogen accumulation and improving cardiac hypertrophy and muscular dysfunction.
A Phase III trial has demonstrated the survival benefit of proton therapy compared to traditional radiation therapy for oropharyngeal cancer patients, with improved overall survival at five years. Proton therapy also resulted in reduced toxicities and feeding tube dependence, highlighting its potential as a standard treatment option.
Waskyra, an ex vivo gene therapy, offers new hope for patients with Wiskott-Aldrich syndrome by reducing severe bleeding events and serious infections. The FDA approval confirms Fondazione Telethon's excellence in rare genetic disease research.
Fondazione Telethon's gene therapy Waskyra, treating Wiskott-Aldrich Syndrome, has received CHMP positive opinion and marketing authorisation in the European Union. The therapy offers new hope for patients affected by this rare genetic disorder.
Researchers at Weill Cornell Medicine have developed a powerful new gene-switch tool called Cyclone, which allows scientists to turn on or off target genes with precision. The tool uses a non-toxic molecule acyclovir to suppress gene activity, and has the potential to be adopted throughout biomedical research and gene therapies.
International experts have established guidelines for gene therapy in hereditary hearing loss, emphasizing patient-centered care and respect for diversity. The guidelines outline frameworks for clinical trials, including patient selection criteria, surgical procedures, and safety evaluations.
A landmark study published in the New England Journal of Medicine reports the long-term safety and efficacy of gene therapy for children with ADA-SCID, a rare immune disorder. The treatment resulted in a 100% survival rate and over 95% cure rate, with patients able to respond to routine childhood vaccinations.
A new gene therapy has restored and maintained immune system function in 59 of 62 children born with ADA-SCID, a rare genetic immune disorder. The treatment involves delivering a healthy copy of the ADA gene to patients' blood stem cells, allowing them to produce healthy immune cells capable of fighting infections.
Gene therapy offers new hope for hereditary deafness, while precision surgical techniques improve cancer outcomes. The AAO-HNSF 2025 Annual Meeting also highlighted access to specialized ENT care, with key strategies for workforce planning and efficient care delivery models.
Researchers developed a new gene therapy that reversed symptoms related to SYNGAP1-related disorders in mice, including intellectual disability, epilepsy, and risk-taking behaviors. The therapy successfully delivered a working copy of the SYNGAP1 gene into brain cells using an adeno-associated virus, offering hope for treatment in humans.
New research shows that music therapy significantly decreases heart rate, systolic blood pressure, and patient-ventilator asynchronies for patients admitted to the cardiac intensive care unit (ICU). Music therapy is a safe, low-cost, non-pharmacological intervention that complements conventional treatments.
A UCLA research team led by Dr. Donald Kohn has developed a one-time stem cell gene therapy treatment for alpha thalassemia major that could be curative. The therapy involves adding the missing alpha-globin gene to patient cells using a viral vector, enabling them to produce functional hemoglobin.
A KAIST research team has developed a universal technology that identifies gene control targets in altered cellular gene networks and restores them. By applying an algebraic approach, they can quickly and accurately calculate how the overall cellular response would change if a specific gene were controlled.
A study published in the Journal of Hepatology reveals that only 15-20% of neonatal liver cells are responsible for generating over 90% of the adult liver mass. This finding has major implications for pediatric gene therapy, allowing scientists to achieve more effective and durable correction of inherited liver diseases.
University of Iowa researchers have successfully tested a technique that stimulates a gene to prevent craniosynostosis, a condition causing infants' skulls to close prematurely. The treatment involved injecting nano-sized packets containing the miR-200a gene into newborn mice, allowing their brains to expand and preventing skull fusion.
A key contributor is the lab of Prof. Ayal Hendel at Bar-Ilan University's Goodman Faculty of Life Sciences, focusing on evaluating CRISPR-based gene editing precision and safety. The project aims to decentralize CAR-T cell therapy production, making it available to more patients by reducing costs and increasing accessibility.
Researchers used human stem cell-derived kidney organoids to test the safety of gene editing delivered by AAV, a common tool in clinical trials. The study found that AAV2 caused significant harm to kidney cells through the NFκB pathway, but an existing drug was able to prevent this damage without interfering with gene delivery.
Researchers developed a one-time gene editing treatment that restored hearing and balance in adult mice with DFNA41, a genetic form of progressive deafness. The therapy successfully disabled the harmful mutation while preserving the normal gene, leading to long-term hearing and balance restoration.
A Delphi exercise with 41 therapists identified 27 statements about therapy that can improve sessions, including the importance of teamwork, outside work, and discussing concerns with loved ones. An app called EveryStep is being trialed to prepare patients for therapy, offering daily activities to aid engagement.
Researchers at St. Jude Children's Research Hospital identified virtual memory T cells, a specialized group of immune cells, which provide nonspecific immunity for infants early in life. These cells are a bridge between innate and adaptive immunity, allowing the adaptive immune system time to develop true memory.
Researchers at the Korea Institute of Science and Technology (KIST) have developed a high-throughput multiplexed gene and cell doping analysis technique using CRISPR-Cas technology. This breakthrough offers superior performance in detecting exogenous genes, demonstrating potential applications in sports ethics and fairness, as well as ...
Genethon is launching a Phase 3 clinical trial in Europe for its low-dose microdystrophin gene therapy GNT0004, targeting boys aged 6 to 10 with retained walking ability. The trial aims to demonstrate efficacy and tolerance of the treatment.
A multicenter clinical trial found gene therapy safe and effective for both children and adults with hereditary deafness. The treatment restores hearing within one month of delivery, showing potential for fast results in addressing congenital deafness and other forms of hearing loss.
A new route of delivering gene therapy is being explored, potentially reducing invasiveness and increasing accessibility. Researchers will test a novel approach on non-human primates, aiming to expand access to office-based treatments.
Gene therapy relies on efficient and safe delivery of therapeutic genes to target cells. Macromolecular carriers, including synthetic and natural polymers, offer biocompatibility, controlled release, and targeted delivery. These systems have shown promise in treating genetic disorders and complex diseases like cancer.
A new study conducted at Karolinska Institutet reports that gene therapy improved hearing in all ten patients with congenital deafness or severe hearing impairment. The treatment was well-tolerated and showed remarkable results, with the majority of patients recovering some hearing after just one month.