A new study in Human Gene Therapy describes a machine learning (ML) model that can be used as a surrogate for laborious in ...

Blackfinbio Ltd. has obtained IND clearance from the FDA for its novel AAV gene therapy, BFB-101, for hereditary spastic paraplegia type 47 (SPG47), which is caused by changes in the AP4B1 gene. A ...

This guide explores a faster, high-throughput approach to accurately evaluate AAV capsid content and help researchers improve efficiency and accelerate gene therapy development. Inside this technical ...

Streamline the Transition from AAV Vector Design to Comprehensive Gene Therapy QC Characterization Guangping Gao, PhD, Professor & Chair, Department of Genetic and Cellular Medicine Professor ...

With this information in hand, researchers who, for instance, are developing a gene therapy to treat a muscle condition, could use the atlas to identify AAV vectors that preferentially target the ...

and clinical developments that are driving today's explosion of gene therapy advances. Developing AAV capsids with improved yield, or fitness, is a key strategy for reducing manufacturing costs in ...