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.
Providing an overview of the submissions process and examples of U.S. Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help developers of gene therapies for rare genetic diseases...
A single dose gene therapy designed to correct the underlying genetic defect, eladocagene exuparvovec (also called Upstaza and made by PTC Therapeutics), is infused directly into the brain through a minimally invasive procedure – the first gene therapy to be administered in this way...
Non-clinical and clinical studies that evaluate pharmacokinetic (ADME) and toxicology characteristics are essential to determine cellular and gene therapy (CGT) product safety and efficacy. Studies on gene vector and transgene biodistribution (BD), gene expression, protein expression, viral shedding, vector integration...
Researchers report on the safety of a gene therapy to treat the common autosomal recessive hereditary disorder alpha 1-antitrypsin (AAT) deficiency in a new article in the peer-reviewed journal Human Gene Therapy. In ATT deficiency, neutrophil proteases destroy the lung parenchyma, the portion of the lungs...
Avance offers a broad range of assay development services, including assay design, assay validation, sample testing, and technology transfer under GLP compliance to support IND-enabling and clinical studies. With decades of experience helping gene and cell therapy companies, Avance Biosciences™ has the...
Twice a year, the Alliance for Regenerative Medicine (ARM) surveys the cell and gene therapy landscape and ARM’s initiatives to advance the sector and provide an update to their members, their stakeholders, and the public at large. While external conditions ebb and flow, the sector’s progress — in clinical milestones, regulatory developments, and patient impact — steadily advances...
bluebird bio has announced the the U.S. Food and Drug Administration (FDA) has approved ZYNTEGLO® (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying [...]
The genes in your body’s cells play a key role in your health. Indeed, a defective gene or genes can make you sick. Recognizing this, scientists have [...]
A paper from the Horae Gene Therapy Center at UMass Chan Medical School may settle a longstanding debate about how to best manufacture adeno-associated virus (AAV) vectors for gene [...]
The Center for Biologics Evaluation and Research (CBER) regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy. CBER uses both the [...]