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

and Davidson have developed capsid engineering strategies to put new AAV models on the production line. There are two main approaches to capsid engineering. “If you know the rules, you engineer ...

Siren said proceeds will go toward investigational new drug (IND)-enabling animal studies and clinical-grade production of ...

Gene therapy represents one of the fastest growing biotherapeutics worldwide, with AAV at the forefront. This rapid growth is constantly challenging CMC approaches, from manufacturing through release ...

Risk factors for mortality in AAV-ILD included age, a history of smoking, a UIP pattern, acute exacerbations of ILD, and the microscopic polyangiitis subtype.

Finally, to validate their results, the researchers performed AAV-mediated genome editing in a transgenic mouse model with the GJB2 R75W mutation. Inner sulcus cells of the cochlea, which were ...