As the field of advanced cell and gene therapies continues to grow exponentially, ensuring their safety and efficacy becomes paramount. Biodistribution studies, which evaluate the distribution of viral vectors and transgenes in the body, play a crucial role in pre-clinical pharmacokinetics and toxicity assessments...
Ensure the Success of Your Cell Therapy Drug Development - Avance Provides Assays to Support your Biopharmaceutical Development & Manufacturing - Avance Biosciences™ is a world-leading CRO specializing in GLP & GMP compliant biological assay development, assay validation, and [...]
Preclinical data demonstrates that hypoimmune-modified allogeneic iPSCs evade immune response and rejection without immunosuppression in a non-human primate model and long-term survival and immune evasion was at least equivalent to the survival of autologous iPSCs...
Strict quality requirements for gene therapies are designed to ensure the safety, efficacy, and consistency of these innovative treatments, and to minimize the risks associated with their use. These requirements are necessary to provide confidence to regulatory agencies, healthcare providers, and patients that gene therapies are safe and effective.
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...
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...
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...
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...
Ensure the Success of Your Biological Drug Development - Avance Provides Assays to Support your Biopharmaceutical Development & Manufacturing - Avance Biosciences™ is a world-leading CRO specializing in GLP & GMP compliant biological assay development, assay validation, and [...]
