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Enhancing Safety and Efficacy – Biodistribution Services for Advanced Cell and Gene Therapies
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...
A historic first for gene therapy
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 presents broad range of new data highlighting novel approaches across its cell therapies portfolio
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...
New preclinical data from Sana Biotechnology highlights the potential of CAR T cell therapy
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...
NGS for ID and characterization of AAV genomes
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...
Quality control for gene therapies
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.