About the Center for Cell and Gene Therapy (CAGT)
The Gillson Longenbaugh Lab
The Center for Cell and Gene Therapy (CAGT) at Texas Children’s Cancer Center collaborates with Baylor College of Medicine to develop novel therapies to treat patients. The program provides an infrastructure to rapidly translate novel cell and gene therapy protocols from the laboratory to the clinic. Established in 1998, the Center has expanded to employ over 30 clinical and research faculty members and 300 staff members.
Shell Center for Gene Therapy
The Shell Center for Gene Therapy provides the environment for pre-clinical and basic science research. Optimizing the therapeutic potential of the cell and gene therapy requires a fundamental understanding of the molecular pathophysiology of disease as well as expertise in the gene transduction (transfer) techniques, transgene regulation, vector targeting, stem cell biology, cellular immunology, and stem cell transplantation. It is felt that advances in these areas will be critical for most cell and gene therapy strategies.
Baylor College of Medicine has made a significant long-term commitment to further enhance the basic research areas related to cell and gene therapy including the identification of genes, gene expression and regulation, target cell identification, development of animal models of disease, and vector development and large scale production.
In addition, The Vector Development Core consists of three investigators and seven additional scientific staff. The core will work with investigators throughout the institution to prepare a range of vectors expressing genes of interest, as well as improving the function of currently available vector systems. The core will import newly discovered vector systems and develop them for clinical use. The 12,000 square foot Shell Center for Gene Therapy is housed in the newly completed Alkek Building on the Baylor College of Medicine main campus.
Translational Research Labs and Protocol Monitoring
Texas Children’s Hospital houses both the Translational Research Labs, the pre-clinical research component, protocol monitoring functions, and over 12,000 square feet of Good Manufacturing Practice (GMP) Laboratories.
Located on the 12th floor of The Feigin Center at Texas Children’s Hospital, scientist and technical staff in these labs work to optimize the conditions and techniques for making the clinical protocol a reality. They address the issues involved in scaling up techniques to meet the volumes associated with clinical trials, developing a closed system for the techniques, and training the appropriate staff in the Clinical Applications Lab. The Translational Research Laboratory serves as a two-way transfer between basic science and patient care with the goal of better understanding diseases and to research new methods of treatment and prevention. Pre-clinical models are formulated, analyzed and tested by a team of researchers, physicians, and technical staff. Once a clinical trial is opened, the Translational Research labs monitor the efficacy of the trial. This staff does development and implementation of new techniques or processes, such as monoclonal antibody or plasmid production, into the GMP lab.
GMP Gene Vector Laboratory
The Gene Vector Lab produces clinical grade vectors for use with Phase I/II clinical trials. The laboratory is one of three National Gene Vector Laboratories (NGVL) and will produce clinical vectors free for NGVL sponsored investigators. The mission of the Center is to develop and implement novel cellular and gene-based treatments for a variety of diseases. Gene transfer vectors provide investigators important research tools in studies of gene function. They can make gene delivery easy and efficient in a number of models of disease. To these ends, the Center for Cell and Gene Therapy established a specialized Vector Development Laboratory (VDL). The VDL was established to function as a preclinical service and GLP manufacturing facility for gene therapy vectors for the CAGT. In addition, the CAGT also houses a Vector Production Facility which operates under current Good Manufacturing Practice (cGMP) guidelines.
The facility is designated National Gene Vector Laboratory for manufacturing adenovirus vectors. Designated areas of the lab produce therapeutic grade monoclonal antibodies and plasmids. The resources of the VDL are available to every investigator at Baylor/TMC as well as to outside investigators.
GMP Clinical Research Laboratory
The Clinical Research Laboratory performs cell separations, expansions, processing and transductions related to preparing patient components for clinical trials. Clinical grade monoclonal antibodies and plasmid DNA are also produced in the GMP Labs. The GMP Laboratories comply with all the Federal standards and have a separate QA/QC section with its own lab space for safety testing of products and audits of the facilities and records. The laboratory is a member of the Production Assistance for Cell Therapies (PACT) and will prepare cells for clinical use without charge for PACT sponsored investigators.
The Stem Cell Transplantation / Bone Marrow Transplant Program
The Bone Marrow Transplant Program houses a 16,200 square foot pediatric unit is located in the West Tower of Texas Children’s Hospital on the eighth floor.
The Protocol Office
The Protocol Office, located at Texas Children’s Hospital, coordinates protocol and IND submissions to the appropriate regulatory agencies. The office is also responsible for monitoring clinical trials and oversees annual reviews. The Director of Clinical Research and Regulatory Affairs is Bambi Grilley.
Learn more about the Cell and Gene Therapy (CAGT) Program at www.bcm.edu/genetherapy