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The 5 steps of investigational gene therapy ONE METHOD OF GENE THERAPY CURRENTLY BEING EXPLORED IN CLINICAL TRIALS IS CALLED GENE TRANSFER. THIS APPROACH AIMS TO INTRODUCE A WORKING, OR FUNCTIONAL, GENE INTO THE BODY TO PRODUCE A NEEDED PROTEIN. © 2020 BioMarin Pharmaceutical Inc. All Rights Reserved. MMRC-GTH-0164 04/20 STEP 1 CREATING A WORKING GENE A working, or functional, copy of a mutated gene is created in a laboratory. STEP 2 BUILDING A THERAPEUTIC VECTOR STEP 3 DETERMINING ELIGIBILITY To protect and deliver the working gene, scientists place it inside an empty viral shell. Patients in clinical trials may meet with their care teams regularly for blood tests and to discuss their treatment regimen and lifestyle. This post-infusion monitoring is important to understanding the safety and impact of the gene therapy on the body. STEP 4 DELIVERING THE WORKING GENE STEP 5 MONITORING SAFETY AND EFFICACY A single, one-time infusion in an appropriate clinical setting delivers large numbers of therapeutic vectors into the body. Once in the body, the working gene is designed to provide instructions for the body to make the protein it needs on its own. Researchers closely monitor patients to evaluate safety and whether the body is creating any proteins. As part of gene therapy research, eligibility requirements may be considered, including age, gender, and organ health. In addition, some patients may have immunity to the therapeutic vector, which could be determined by a blood test. To learn more about ongoing gene therapy research and to sign up to receive new information as it becomes available, visit HemDifferently.com No gene therapies for hemophilia have been approved for use or determined to be safe or effective. =New proteins WORKING GENE DNA VIRAL SHELL THERAPEUTIC VECTOR
