BioProcessing Journal Posts

A formulation for purified adenoviral vectors was developed that provides stability through freeze-thaw stress and long-term storage at non-frozen temperatures. To evaluate the various test conditions, a panel of stability indicating methods was assembled, which included laser light scattering, HPLC, and transgene expression assays. Preformulation studies were conducted, and the effects of buffer species, pH, cryoprotectants, and salts upon adenoviral vector stability were determined…

Viral Vectors

In orthopedic procedures, there is a need to form new bone to repair and fill defects arising from either trauma or degenerative disease. The current standard treatment utilizes an autograft, usually from the iliac crest, which results in a second surgical site, weakness in the harvest or donor harvest area, and additional patient morbidity. The goal of our research is to employ a local, ex vivo, gene therapy to obviate the need for autograft in spine fusion procedures. The LIM Mineralization Protein (LMP-1) is a novel intracellular protein capable of inducing bone formation in vitro and in vivo. In this article, I will outline a rapid protocol, whereby buffy coat cells, isolated from autologous peripheral blood, are transduced with an adenoviral vector encoding the LMP-1 gene. These transduced cells are then implanted on a collagen carrier to promote posterolateral arthodesis…

Cell & Gene Therapy

As you stand on the brink of finalizing your first Investigational New Drug (IND) application for a cellular therapy product, there is always the question looming in the back of your mind. “What did we forget?” Hopefully, the answer is “Nothing.” However, it is always good to undergo a review of the standard systems needed in order to transition from research to clinical manufacturing. This article describes an overview of the basic regulatory guidelines and quality systems necessary to begin clinical trials under the regulations of the Food and Drug Administration. However, this should only be considered a guideline, as it does not necessarily address the standards of other regulatory agencies. The investigational product, the clinical indication, and the manufacturing materials used in the investigational product can also change the regulatory requirements needed to proceed with the initiation of clinical trials…

Cell & Gene Therapy Manufacturing Regulatory