Abstract
no abstract.
Highlights
THERE ARE AN estimated 7,000–10,000 different rare diseases that impact the human population.[1]
Platform Vector Gene Therapy (PaVe-GT) will aim to provide a useful blueprint for managing gene therapy product development and testing, sharing information, best practices, and lessons learned along the way with the public in an open forum that will help others to follow the path and decrease their burden to achieving gene therapy especially for rare diseases with small numbers of patients
Thousands of noncommercially viable rare diseases may remain ‘‘orphaned’’ despite the existence of tools with significant potential to meaningful alter the course of these diseases
Summary
THERE ARE AN estimated 7,000–10,000 different rare diseases that impact the human population.[1]. PaVe-GT will aim to provide a useful blueprint for managing gene therapy product development and testing, sharing information, best practices, and lessons learned along the way with the public in an open forum that will help others to follow the path and decrease their burden to achieving gene therapy especially for rare diseases with small numbers of patients. The NIH PaVe-GT team initially engaged FDA Center for Biologics Evaluation and Research (CBER) for nonbinding discussion of the general PaVe-GT concept, including the acceptability of developing and clinically testing four gene therapies for four diseases in a standardized process, exploring mechanisms for seamless information sharing between gene therapy applications, such as the use of master files, and hearing FDA’s concerns regarding maintaining highquality science in an expedited program. From our discussions to date, FDA has indicated that they are highly supportive of our approach and NCATS’ plan for information sharing
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