Back to: FDA Regulatory Education for Industry (REdI) 2024 Conference – Biologics Track
Presenter
Gopa Raychaudhuri, PhD
Associate Director for Special Programs
Office of the Director (OD)
Center for Biologics Evaluation and Research (CBER)
US Food and Drug Administration (FDA)
Gopa Raychaudhuri, PhD is the Associate Director for Special Programs in the Office of the Director, Center for Biologics Evaluation and Research. Dr. Raychaudhuri is engaged in facilitating development of gene therapies for rare diseases including engagement in the public-private partnership – the Bespoke Gene Therapy Consortium (BGTC). Additionally, Dr. Raychaudhuri is CBER’s liaison to the World Health Organization (WHO) providing strategic direction and overseeing CBER’s scientific collaborations with the WHO. Dr. Raychaudhuri has a long-standing interest in the development of and access to essential medical products that promote national and global public health. She received her BSc and MSc degrees in Biochemistry from the University of Toronto and received a PhD in Microbiology from the University of Virginia. Her post-doctoral training at NIH/NIAID focused on vaccine development research. Since 2010, she has been in her current position implementing strategic programs that advance CBER priorities, including the development gene therapies for rare diseases.
Abstract
Gopa Raychaudhuri discusses efforts to facilitate the development of gene therapies for rare and very rare diseases. A rare disease affects fewer than 200,000 people in the U.S., representing over 10,000 different diseases impacting millions globally and a significant unmet medical need. Many are serious, life-threatening single gene disorders with potential for treatment via gene therapy. Gene therapy research and approvals show a notable increase in recent years. The presentation focuses on individualized or bespoke gene therapies, designed for a specific patient or small group, which pose unique challenges for manufacturing and clinical development outside traditional paradigms. CBER regulates types like directly administered gene therapy and genetically modified cellular therapies. Raychaudhuri highlights the Bespoke Gene Therapy Consortium (BGTC), a public-private partnership aiming to streamline development for very small populations (1-100 patients/year) where commercial interest is lacking. The BGTC works to standardize processes, testing, and clinical methods, focusing on AAV gene therapy. Regulatory streamlining opportunities are explored, including using master files and leveraging data across applications. Global collaboration is emphasized as crucial, given the small patient numbers in individual countries, making international harmonization beneficial. CBER actively engages with international regulators and the WHO to support this effort. The overall goal is to deliver safe and effective gene therapies to alleviate suffering globally, recognizing the need for innovative approaches outside standard processes for these conditions. Collaboration among stakeholders is vital for accelerating progress.