The concept of using pre-existing immunogenicity as an exclusion criteria for gene therapy clinical trials is changing. BioAgilytix’s Chief Scientific Officer, Dr. Jim McNally, explores why.
It's a common misconception that all aspects of cell & gene therapy research and development are excluded from CLIA oversight. We take a closer look at testing and when CLIA requirements apply.
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.
The first in-human in vivo clinical trial using CRISPR-Cas9 is now open for enrollment; we take a closer look at this genome editing technology to understand its promising benefits as well as the potential challenges of anti-Cas9 immunity.
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.
The webinar I recently hosted with my colleague Lydia Michaut explored key challenges and trends in the immunogenicity assessment of gene therapy compounds. This recap highlights the critical questions that were answered.
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.
Today we're featuring a case study on how BioAgilytix developed a cell-based assay to determine the prevalence of pre-existing antibodies against the AAV9 serotype in patients for a gene therapy clinical trial.
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.
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…
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