All articles tagged with #gene therapy
A preclinical CNS-targeted gene therapy dampens pain signals in specific brain circuits to relieve chronic pain while avoiding the brain reward pathways tied to addiction, using AI-guided imaging and animal models, with an off switch for long-lasting relief and moving toward clinical trials to help the roughly 50 million Americans affected by chronic pain.
Gene therapies are moving from trials toward patient care, with breakthroughs like personalized CRISPR treatments and new gene-editing tools powering hope for many diseases; however, turning lab successes into approved, accessible medicines is hampered by regulatory, manufacturing, safety, and cost challenges, leaving access uneven despite progress.
Researchers suggest that targeting the Apoe gene, particularly its harmful variants Apoe3 and Apoe4, could potentially prevent most cases of Alzheimer's disease, although challenges remain due to the gene's vital functions and widespread presence in the population.
Krystal Biotech announced positive interim results from its Phase 1 trial of KB407, a gene therapy for cystic fibrosis, confirming successful lung delivery and expression of wild-type CFTR protein across diverse patients, paving the way for a larger, repeat-dosing study expected to start in 2026.
Scientists at UNSW Sydney have developed a new CRISPR-based epigenetic editing technique that can turn genes on without cutting DNA, by removing chemical methyl groups that silence genes. This approach could lead to safer gene therapies for conditions like Sickle Cell disease, as it avoids the risks associated with DNA strand breaks. The research demonstrates that methylation directly controls gene activity and opens new possibilities for treating genetic disorders by reactivating silenced genes.
The article previews ten significant clinical trials expected in the first half of 2026, focusing on Eli Lilly's obesity drug retatrutide, Merck's flu antiviral CD388, Regenxbio's gene therapy for Duchenne muscular dystrophy, Novartis' Lp(a) cardiovascular treatment, and other innovative therapies for rare diseases and cardiovascular conditions. Success in these trials could lead to major advancements and market approvals in their respective fields.
A five-year-old boy from Colchester with SMA has made remarkable progress after receiving the world's most expensive gene therapy, Zolgensma, which has transformed his mobility and quality of life, highlighting significant advances in treatment for rare genetic conditions.
A promising gene therapy for sickle cell disease, approved two years ago, remains largely inaccessible due to limited treatment centers, complex approval processes, and high costs, especially impacting rural and low-income populations despite recent federal initiatives to improve access.
A child undergoing experimental gene therapy with a new virus died shortly after the procedure, raising concerns about the safety of viral vectors used in brain gene therapies and potentially hindering progress in the field.
A mother was overjoyed to learn a drug could save her baby's life, but the $2 million cost of the treatment highlights the high prices of new gene therapies, raising concerns about affordability and the impact on the healthcare system.
Parents of children with SMA advocate for universal newborn screening to enable early diagnosis and treatment, highlighting the stark difference in outcomes between their children who were tested early and those who were not, with the UK considering expanding screening programs amid promising new treatments.
A pilot study demonstrates that a single gene-editing treatment targeting the ANGPTL3 gene can permanently reduce 'bad' cholesterol and triglycerides, potentially replacing lifelong medication for high cholesterol if further trials are successful.
Scientists have developed a potential gene therapy approach to enable the body to produce its own GLP-1 hormone, which could offer a one-time treatment for weight loss and related health benefits, reducing reliance on daily or weekly injections. Early tests in animals show promising results, but human applications are still years away and require careful safety evaluation.
Scientists at Mount Sinai have discovered that reactivating the Cyclin A2 (CCNA2) gene in adult hearts can promote self-healing after injuries like heart attacks, potentially revolutionizing treatment by enabling natural heart regeneration and reducing reliance on transplants or mechanical devices.
A long-term follow-up study shows that gene therapy for babies with severe combined immunodeficiency (SCID), known as 'Bubble Boy disease,' remains 95% effective in restoring immune function, with children living healthy, normal lives and the therapy nearing FDA approval.