On this episode of Molecular Moments, Jim McNally, Ph.D., speaks with Adam Stasio, president of SciMed, whose mission is to
The third and final blog in our cell and gene therapy series will focus on PCR as a bioanalytical tool for gene and cell therapy. Be sure to read our first blog on bioanalytical support for gene therapy programs and the second blog on the safety and efficacy testing of cell therapies. Prevalence of PCR in Bioanalysis Polymerase Chain Reaction (PCR) is a laboratory method for detecting and quantitating molecular
An Overview of Cell Therapy: As we continue our blog series on cell and gene therapy, we will now dive into cell therapy programs. If you missed the first blog “Bioanalytical Support for Gene Therapy Programs,” you can find it here. Cell Therapy or Adoptive Cell Transfer are broad terms for the use of living cells to treat disease. This can include conventional treatments such as bone marrow and stem
An Introduction to Gene Therapy: As early as the 1970’s, the term “gene therapy” was used for the treatment of genetic disorders at the DNA level to replace the defective or missing proteins driving disease. By introducing functional gene copies to patients’ cells, the subsequent in vivo expression of protein leads to a therapeutic effect and may ultimately cure the associated disorder. Gene therapy has undergone several major paradigm shifts
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.