CBER recognizes that the development of AAV gene therapies in rare disease can introduce unique challenges related to unknown safety profiles, complex manufacturing processes, new technologies and equipment, incorporation of innovative devices, and the use of cutting-edge testing methodologies that can benefit from early FDA input.
An INTERACT meeting can provide great value for you in:
Assisting your early product characterization and design of pre-clinical proof-of-concept studies
Identifying critical issues around proof-of-concept studies, manufacturing-related questions, or other deficiencies you can address early in development, before approaching the Pre-IND
This feedback is a step towards de-risking and accelerating your drug development process. [1]
Center for Biologics Evaluation and Research (CBER) - The Center within FDA that regulates biological products for human use under applicable federal laws, including the Public Health Service Act and the Federal Food, Drug and Cosmetic Act. CBER protects and advances the public health by ensuring that biological products are safe and effective and available to those who need them. CBER also provides the public with information to promote the safe and appropriate use of biological products.
Adeno-associated virus (AAV) - A member of the parvovirus family of single-stranded small DNA viruses that require a helper virus such as adenovirus or herpes simplex virus for replication. AAV vectors are the leading platform for gene delivery for the treatment of a variety of human diseases.
Food and Drug Administration is a U.S. government agency responsible for regulating food, pharmaceuticals, cosmetics, and medical devices