News & Events
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Protein expression biodistribution studies
Protein expression analysis is a critical process for researchers in the life sciences industry. The ability to accurately and reliably measure protein expression and distribution is essential for understanding the underlying biological mechanisms of disease and for developing effective therapeutic interventions...
Gene therapy developers can benefit from orphan drug and rare pediatric disease designations
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...
NICE recommends life-changing gene therapy for children with ultra-rare genetic disorder
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...
Biodistribution testing of cell and gene therapies
Cell and gene therapy drug products represent a rapidly growing field in the development of new treatments for a wide range of diseases. These therapies have the potential to revolutionize medicine by offering cures rather than just managing symptoms. However, with this potential comes unique challenges, particularly with regard to biodistribution testing...
Join Avance Biosciences™ at Gene Therapy for Rare Disorders – Mar 20-23 – Boston
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...
Gene editing of human stem cells to treat rare disease
Gene editing has the potential to transform the lives of patients with previously untreatable conditions, offering hope for a better future. As the technology continues to advance, it is likely that we will see more breakthroughs in this field in the coming years, bringing us closer to a world where rare diseases are...