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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...
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...
Avance Biosciences™ licenses SeQure Dx’s GUIDE-seq off-target NGS analysis technology
Avance Biosciences™ (Avance), a leading CRO that provides GLP/CGMP-compliant biological testing services that aid drug development and manufacturing, announced today that it has signed a licensing agreement with SeQure Dx, a cutting-edge gene-editing diagnostics company...
Quality Assurance of E. coli Cell Banks used in Biopharmaceutical Production
The use of E. coli cell banks poses some challenges and requires careful attention to quality control and quality assurance to ensure that the cells are of the highest quality and free from contamination or genetic mutations. Testing may include identity, purity, viability, characterization, and stability testing...