On this episode of Molecular Moments, Lynn Kamen, Ph.D., and Michelle Miller, Ph.D. DABT discuss their very different journeys to
Though the modern world has progressed a lot in terms of medical interventions and biomedicine, there still are a vast number of diseases and disorders that demand a rather unconventional approach to treatment. Gene therapy has evolved as a technique that aims to address some of these diseases by introducing into a patient a normal copy of one or more defective genes responsible for the patient’s disease. By repairing the
Gene therapy is a powerful form of treatment that involves introducing genetic material into cells to replace defective genes resulting in the production of an essential protein. If a defective gene causes a required protein to be faulty or absent, gene therapy may be able to restore the protein’s function by introducing a “healthy” copy of the gene. Since gene therapy products work by introducing genetic material into the cell,
Cell and gene therapies have gone through a growth spurt in the last decade. According to data from the Alliance for Regenerative Medicine (ARM), during the first three quarters of 2020, regenerative medicine research and development broke records at nearly $16 billion in global financing. From the treatment of knee injuries to osteoarthritis, certain cancers and even muscular dystrophy, advances in gene and cell therapies have definitely caught the attention
ELISpot is a method commonly used to detect cellular immune responses to viral vector-based gene therapies. We take a closer look at how the industry is working to harmonize best practices for developing & validating ELISpot assays for gene therapy development.
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.