In a promising form of immunotherapy known as CAR T-cell (chimeric antigen receptor) therapy, the patient’s T cells are engineered to better recognize and attack antigens on the surface of cancer cells. In treatments currently approved for use in battling lymphoma and leukemia...
The U.S. Food and Drug Administration approved Lantidra, the first allogeneic (donor) pancreatic islet cellular therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes. Lantidra is approved for the treatment of adults with type 1 diabetes who are unable to approach target glycated hemoglobin (average blood glucose levels) because of current...
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...
Sarepta Therapeutics announces FDA approval of ELEVIDYS, the first gene therapy to treat Duchenne Muscular Dystrophy. ELEVIDYS addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as ELEVIDYS micro-dystrophin...
2seventy bio announced new data featuring novel approaches combining the company’s platform CAR T cell and T cell receptor technology and unique cell therapy engineering capabilities to potentially enhance treatment potency in a range of cancers...
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...
Adeno-associated virus (AAV) is a small, non-pathogenic, and non-enveloped virus that has become an important tool for gene therapy research and applications. AAV is a type of virus that requires a helper virus (such as adenovirus) to replicate, and it has a high degree of stability and safety, as it does not...
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.
Potency testing is an essential part of developing cellular and gene therapy (CGT) products. The US Food and Drug Administration (FDA) requires sponsors for Investigational New Drug (IND) and Biologics License Application (BLA) to provide in vitro or in vivo data to demonstrate the potency of the drug product...
