Center for Gene Therapy
Mission:
The Center for Gene Therapy (CGT) is dedicated to advancing the readiness for gene therapy clinical trials and developing infrastructure for rare disease research, including phenotyping and outcomes assessment. Our mission is to accelerate the early implementation of gene therapies in clinical settings by fostering collaboration among investigators, pharmaceutical companies, advocacy groups, scientists, and other key stakeholders. We aim to build multidisciplinary expertise in gene therapy and support the seamless execution of gene therapy clinical trials. CGT offers a comprehensive infrastructure to assist investigators, providing access to a skilled team of professionals including physicians, nurses, clinical research coordinators, and regulatory experts
Strengths
- Significant experience in conducting gene therapy trials across all phases and age groups
- Strong genetic research foundation, allowing many patients to undergo genetic testing
- Robust research infrastructure across departments that supports contracting, regulatory processes, and clinical trial execution
- CGT investigators are experienced with the rigor associated with dosing of intrathecal or systemic AAV and with complex delivery protocols and data collection.
- Inpatient observation for prior gene therapy trials has ranged from 24 hours to 8 days. The observation space is monitored by CTSI nurses experienced with gene therapy and is directly above the Emergency Department.