From The Stage

Vector Innovation to Improve Gene Therapy Safety: A Credit to Scientific Tenacity

Dr. Jim McNally
Posted by Dr. Jim McNally / / BioAgilytix Insight, Gene Therapy

A new clinical trial by St. Jude Children’s Research Hospital for the treatment of SCID-X1 highlights a major advancement in gene therapy safety over recent years, in this case spurred by scientists who were courageous enough to push forward the development of safer vectors to overcome serious adverse events (SAEs) that were resulting from the previous generation of gene therapies developed for SCID-X1.

Immunogenicity Assessment of Gene Therapy Compounds

Dr. Arno Kromminga
Posted by Dr. Arno Kromminga / / Gene Therapy, Industry Update

By Dr. Arno Kromminga and Dr. Lydia Michaut
Gene therapy has experienced an exciting resurgence in the post-genomic era, but unwanted immunogenicity is still a concern. Our latest blog gives insight into the current concepts for designing an immunogenicity assessment strategy fitting the specific needs of a given gene therapy compound.

The FDA’s Response to an Increase in the Development of Gene and Cell Therapies: What It Means for Sponsors

Jim Datin
Posted by Jim Datin / / Cell Therapy, Gene Therapy, Industry Update

The FDA has observed an unprecedented increase in the number of cell and gene therapy products entering the early stages of development. In a recent statement, the agency outlined new planned policies to help accelerate these innovations through the pipeline to clinical success. We discuss what this announcement means to sponsors.

Novel Gene Therapy Technique Creates a Breakthrough in Sickle Cell Anemia Treatment

Dr. Arno Kromminga
Posted by Dr. Arno Kromminga / / BioAgilytix Insight, Gene Therapy

Innovations in gene therapy solutions are being made every day, improving treatments and creating cures for many previously untreatable genetic diseases. In fact, According to a recent report on global trends in gene therapy, the number of gene therapy products in development doubled between 2012 and 2015. The exciting news is that, based on a recent study within the scientific community, sickle cell disease may soon be treatable with gene therapy. It is estimated that 50 million people worldwide carry the genetic mutation for sickle cell disease. This debilitating disease…