CBER Director’s Report Highlights Advances in Cellular and Gene Therapy, Transfusion Medicine

The Center for Biologics Evaluation and Research (CBER) issued a report last week detailing the agency’s 2024 efforts to advance public health and address emerging challenges in transfusion medicine and biotherapies. Authored by CBER Director Peter Marks, MD, PhD, the report includes several updates relevant to the AABB community.

In biotherapies, CBER issued 10 new cell and gene therapy approvals for rare diseases, doubling its approval rate following the 2023 reorganization of its Office of Therapeutic Products. CBER continued its focus on facilitating development of cell and gene therapies the publication of several guidance documents. The report also highlighted public meetings with patients and biotherapies stakeholders and noted progress in the Collaboration on Gene Therapies (CoGenT) Global Pilot, which is exploring concurrent, collaborative review of gene therapy applications with international regulators.

CBER also highlighted several 2024 safety communications. These address cybersecurity practices for blood establishments, emerging pathogen threats like Oropouche virus and the agency’s concerns related to “young donor plasma,” among others. CBER also cited expanded oversight of biologics manufacturing technologies and safety updates for chimeric antigen receptor (CAR) T-cell therapies.

AABB encourages members to read the entire report FDA’s website.