From The Stage

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

Dr. Corinna Fiorotti
Posted by Dr. Corinna Fiorotti / / 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.

Gene Therapy Trends: Conditional Approval of Zynteglo Brings Ex Vivo Gene Therapy Into Focus

Dr. Lydia Michaut
Posted by Dr. Lydia Michaut / / Gene Therapy, Industry Update

The recent approval of Zynteglo further builds credibility to the specific and emerging class of cell therapies engineered via “ex vivo” gene therapy. We explain how these cell-based gene therapies work, and how they lend themselves to a host of translatable techniques for future therapeutic candidates.

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.