Gene Therapy News
Gene therapy is rapidly advancing, offering new hope for treating rare diseases, cancers, and genetic disorders. This page provides a curated collection of the most important news in gene therapy drug development, from clinical trial milestones to regulatory updates worldwide. Our goal is simple: to keep you informed on the latest breakthroughs and developments shaping the future of gene-based medicine.
Gene Therapy News
Gene therapy is rapidly advancing, offering new hope for treating rare diseases, cancers, and genetic disorders. This page provides a curated collection of the most important news in gene therapy drug development, from clinical trial milestones to regulatory updates worldwide. Our goal is simple: to keep you informed on the latest breakthroughs and developments shaping the future of gene-based medicine.
Inconsistent assay performance is a common but often overlooked cause of batch failures. Robust, reproducible analytical and bioanalytical assays help reduce risk, improve decision-making, and support reliable batch release across drug development and manufacturing...
Avance Biosciences now offers PacBio HiFi long-read sequencing, providing highly accurate, full-length plasmid analysis. This technology ensures precise detection of variants, contaminants, and structural anomalies for reliable plasmid QC...
Explore the FDA’s new Plausible Mechanism Pathway and how it compares to accelerated approval, expedited programs, and other regulatory routes for rare disease therapies.
The FDA released three new draft guidances to streamline cell and gene therapy development. The documents outline innovative trial designs for small populations, expedited pathways for serious conditions, and improved postapproval methods for monitoring long-term safety and efficacy...
Avance Biosciences’ NGS Center of Excellence delivers comprehensive sequencing solutions, including short-read, long-read, and advanced single-cell platforms. Our regulatory-compliant workflows support biologics, gene, and cell therapy programs with high-quality data, enabling informed development...
An NIH-supported team has delivered the first personalized CRISPR-based gene therapy to an infant with CPS1 deficiency, offering new hope for rare disease treatment. The customized approach shows early success and may pave the way for future individualized therapies...
Avance Biosciences is excited to exhibit at the ASGCT 2025 conference in New Orleans, LA, from May 13–17, 2025. Join us to explore our expert bioanalytical solutions for gene and cell therapy development. Visit booth #750 to learn how we can support your therapeutic advancements...
MIT engineers have developed a gene circuit that precisely controls gene expression, offering potential breakthroughs in gene therapy for disorders like fragile X syndrome. This advancement helps ensure therapeutic genes are expressed at optimal levels, minimizing risks and paving the way for more effective treatments...
A groundbreaking gene therapy using rAAV8.hRKp.AIPL1 has restored vision in 11 children with AIPL1-related retinal dystrophy (LCA4), who were blind at birth. Published in The Lancet, the study shows significant improvements in visual acuity and retinal preservation, supporting expedited approval...
Patients in England with severe beta-thalassaemia will be amongst the first in Europe to benefit from one-time gene therapy exagamglogene autotemcel. Exa-cel is the world’s first CRISPR-based gene therapy and the first gene therapy available in Europe for treating severe beta-thalassaemia...