To generate successful gene therapies that are capable of delivering nucleic acids to specific cells and tissues, scientists must carefully design their products. They can employ either recombinant ...

Regenxbio’s gene therapy for Duchenne muscular dystrophy has smashed the primary endpoint of its pivotal trial, securing a ...

Viral vectors introduce genes and gene editing sequences into cells, and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in ...

To address the critical need for accelerated decision-making and de-risked progression from discovery to Chemistry, Manufacturing, and Controls (CMC) in cell and gene therapy programs, ProBio Inc. has ...

Safety issues linked to adeno-associated virus (AAV) gene therapies, including patient deaths tied to liver toxicity, are prompting developers to explore non-viral delivery methods. Alternatives such ...

Developers are increasingly exploring non-viral gene delivery methods as safety issues with viral vectors draw scrutiny. Advances in lipid nanoparticles, polymer carriers, and electro-transfection are ...