Advances in genetics and bioengineering have enabled the development of
gene therapy to treat diseases caused by recessive gene disorders,
acquired genetic diseases, and some viral infections. Technologies for
the manufacture and analysis of gene the...
Knowing the genome packaging efficiency during adeno-associated virus
(AAV) manufacturing is a crucial step toward the successful clinical
application of next-generation biotherapeutics. In recent years,
recombinant AAV production has been steadily o...
Production of recombinant adeno-associated viruses (AAVs) for the
purpose of gene therapy is challenging and requires various analytical
assays to monitor product quality attributes (PQAs) such as product
titer. Due to low production yields, it’s imp...
This is the fourth blog post in the Biopharmaceutical Basics series.
Over the past 30 years, noteworthy progress has been made in the field
of oligonucleotide therapeutics, beginning with antisense
oligonucleotides (ASOs) and aptamers, followed by si...
This is the third blog post in the Biopharmaceutical Basics series.
Sometimes the whole or part of a gene is defective or missing from
birth. This is typically referred to as a genetically inherited
mutation. In addition, healthy genes can change (mu...