Sequencing of the human genome and numerous advances in molecular techniques have
launched the era of genetic medicine. Increasingly precise technologies for genetic …

Progress in understanding the molecular bases of human health and disease in recent
decades has flourished making it possible for the field of gene therapy (GT) to offer new …

With over 220 investigational new drug applications currently active, gene therapy
represents one of the fastest growing areas in biotherapeutic research. Initially conceived for …

Over the last few years, the transfer of therapeutic genes via gammaretro-or lentiviral vector
systems has proven its virtue as an alternative treatment for a series of genetic disorders …

The number of gene therapy (GTx) modality therapies in development has grown
significantly in the last few years. Adeno-associated virus (AAV)-based delivery approach …

Nonclinical development strategies for gene therapies are unique from other modalities. The
European Federation of Pharmaceutical Industries and Associates (EFPIA) Gene Therapy …

Introduction: With the burgeoning growth of the gene therapy industry, the Food and Drug
Administration (FDA) has produced various guidance documents intended to help gene …

The rapidly changing field of gene therapy promises a number of innovative treatments for
cancer patients. Advances in genetic modification of cancer and immune cells and the use of …

Introduction: We previously reported on the United States' regulatory environment evolving
to accommodate an emerging boom in gene therapy research. Several important …

Introduction: A dramatic increase in the number of clinical trials involving gene-modified cell
therapy and gene therapy is taking place. The field is on the verge of a boom, and the …