Adeno-associated viral (AAV) vectors have been reported to be a great promise in a large
number of clinical trials. Multiple AAV gene products will enter into early and late phase …

Adeno-associated virus (AAV) vectors are extensively used for gene therapy clinical trials.
Accurate and standardized titration methods are essential for characterizing and dosing AAV …

With the success of clinical trials using recombinant adeno-associated viral vectors (rAAV),
regulatory agencies ask for a more comprehensive characterization of process-and product …

Introduction Pompe disease is an autosomal recessive disorder caused by a deficiency of
acid-α-glucosidase (GAA), an enzyme responsible for hydrolyzing lysosomal glycogen. A …

Corneal blindness is the fourth leading cause of blindness worldwide. Since corneal
epithelium is constantly renewed, non-integrative gene transfer cannot be used to treat …

Recombinant vectors based on a non-pathogenic parvovirus, the adeno-associated virus
(AAV), have taken center stage in the past decade. The well-established safety of AAV …

In Nov. 2018, The National Institute for Innovation in Manufacturing Biopharmaceuticals
(NIIMBL) published technology roadmaps addressing needs and gaps in three key product …

Recombinant Adeno-Associated Virus (rAAV) is considered as one of the most successful
and widely used viral vectors for in vivo gene therapy. However, host immune responses to …

In the early years of gene therapy, the chemistry, manufacturing, and control (CMC) aspect
of gene therapy development was often viewed as a means to provide material to establish …

Recombinant adeno-associated viruses (rAAVs) are valuable viral vectors for in vivo gene
transfer, also having significant ex vivo therapeutic potential. Continued efforts have focused …