On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …

Immunogenicity has imposed a challenge to efficacy and safety evaluation of adeno-
associated virus (AAV) vector-based gene therapies. Mild to severe adverse events …

Adeno-associated virus (AAV)-based gene therapies, exemplified by the approved therapy
for spinal muscular atrophy, have the potential to deliver disease-course-altering treatments …

Gene therapy has seen a transformation from a proof-of-concept approach to a clinical
reality over the past several decades, with adeno-associated virus (AAV)-mediated gene …

Nucleic acid therapeutics have emerged as one of the very advanced and efficacious
treatment approaches for debilitating health conditions, including those diseases affecting …

A number of adeno-associated virus (AAV)-based gene therapy products have entered
clinical development, with a few also reaching marketing approval. However, as our …

Given the recent success of gene therapy modalities and the growing number of cell and
gene-based therapies in clinical development across many different therapeutic areas, it is …

Microphysiological systems (MPS), also known as miniaturized physiological environments,
have been engineered to create and study functional tissue units capable of replicating …

Dystrophic cardiomyopathy is a significant feature of Duchenne muscular dystrophy (DMD).
Increased cardiomyocyte cytosolic calcium (Ca 2+) and interstitial fibrosis are major …

The Central Nervous System (CNS) is vulnerable to a range of diseases, including
neurodegenerative and oncological conditions, which present significant treatment …