A company developing a cell and gene therapy that is a combination product should submit which type of application?

When developing a cell and gene therapy that qualifies as a combination product, the appropriate regulatory pathway involves the submission of a Biologics License Application to the Center for Biologics Evaluation and Research (CBER). This is due to the nature of the combination product, which typically involves biological components and may incorporate elements from both drugs and devices.

Cell and gene therapies are classified as biologics because they involve the use of living cells or microorganisms, which are regulated under the Public Health Service Act as well as the Federal Food, Drug, and Cosmetic Act. CBER has the authority to regulate products that contain biologics, including those used in the context of cell and gene therapy. The Biologics License Application process provides the framework to evaluate the safety, purity, and potency of these biologic products, ensuring compliance with regulatory standards.

While the other options may seem relevant, they do not align with the specific requirements for cell and gene therapies. New Drug Applications typically address small molecule drugs, and Combination Product Applications may be related to devices or other applications, rather than the biologics-focused framework required for cell and gene therapies. Therefore, the Biologics License Application to CBER is the most suitable choice for the regulatory submission in this case.