Impact of Pre-Existing Immunogenicity Against AAV on Gene Therapy Trials
Pre-existing antibodies against the adeno-associated virus (AAV) are a known challenge in gene therapy programs that use AAV-based viral vectors as their mode of gene...
Posts about:
Gene Therapy
CLIA Requirements for Gene and Cell Therapy Programs
It is a common misconception that all aspects of cell and gene therapy research and development are excluded from Clinical Laboratory Improvement Amendments (CLIA)...
CRISPR-Cas9 Milestones: The First In Vivo Clinical Trial Is Now Underway in Boston
For the first time in gene therapy history, CRISPR-Cas9 genome editing technology will be used in humans in an in vivo clinical trial in the Boston area that is set to...
Gene Therapy Trends: Conditional Approval of Zynteglo Brings Ex Vivo Gene Therapy Into Focus
In June, the European Medicines Agency (EMA) granted a conditional approval to the orphan drug Zynteglo, the first gene therapy approved for the treatment of a rare...
Your Questions About Immunogenicity Assessment of Gene Therapy Compounds Answered
By PD Dr. Arno Kromminga
Gene therapy has come a long way since it was first suggested as a potential treatment for genetic-based diseases in 1972. Completion of the ...
Immunogenicity Assessment of Gene Therapy Compounds
Gene therapy has experienced an exciting resurgence in the post-genomic era, with the efficacy and safety of this class of drugs now proving out and culminating most...
Supporting a Gene Therapy Clinical Trial with Assay to Measure NAbs Against the AAV9 Serotype
Gene therapy is experiencing an exciting resurgence with the development of new genome editing tools and new gene delivery vectors. Approximately 60% of gene therapy...
The FDA’s Response to an Increase in the Development of Gene and Cell Therapies: What It Means for Sponsors
In the last two decades, our understanding of the mechanisms behind human disease paired with significant innovation in complementary technologies has empowered our...