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.
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...
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...
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...
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...
Tecelra is the first FDA-approved T cell receptor (TCR) gene therapy. The product is an autologous T cell immunotherapy composed of a patient’s own T cells. T cells in Tecelra are modified to express a TCR that targets MAGE-A4, an antigen expressed by cancer cells in synovial sarcoma...
Avance Biosciences™ is excited to announce our participation in the American Society of Gene & Cell Therapy (ASGCT) conference, where we’ll be showcasing our advanced services and expertise to propel the future of gene and cell therapy. [...]
The guidance provides recommendations regarding information that should be provided in an investigational new drug (IND) application to assess the safety and quality of the investigational GE product, including information on product design, product manufacturing and testing, nonclinical safety...