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FDA Policy Changes will Accelerate Cell and Gene Therapy Approvals

Feb 21, 2019 10:02:00 AM / by Nancy Andon, MSc

FDA-logo (002)The U.S. Food and Drug Administration (FDA) has announced that they are adding staff and rolling out policy changes aimed at advancing the development of safe and effective cell and gene therapies. [1] The announcement came in the form of a press release on January 15th, citing that the new policies are a response to the current surge in cell and gene therapy products that the agency is handling. Based on the number of investigational new drug (IND) applications being submitted, the FDA projects a significant rise in the number of therapies that will be approved over the next few years:

We anticipate that by 2020 we will be receiving more than 200 INDs per year, building upon our total of more than 800 active cell-based or directly administered gene therapy INDs currently on file with the FDA. By 2025, we predict that the FDA will be approving 10 to 20 cell and gene therapy products a year based on an assessment of the current pipeline and the clinical success rates of these products.”
- FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research.

The FDA specifically recognizes the revolutionary impact cell and gene therapies are expected to have on medicine as a whole, likening it to the impact of antibody-based drugs in the late 1990’s. Then, as now, the dramatic adoption of the new technology was a function of widespread implementation of a major innovation; in that case the development of fully human monoclonal antibodies. Today, the innovative game changer is the development of safe and effective vectors whereby defined genetic changes can be introduced. The advent of this technology has been enormously important to CAR-T cell therapy, one of the first new cell therapy products to be approved.

The anticipation of a dramatic increase in the number of cell and gene therapy product approvals necessitates regulatory and manufacturing changes to keep pace with the influx of new therapies. For the FDA, this means increasing clinical review staff, and rolling out new policies that support more efficient development and fast-tracked approval of qualified therapies. [2]

A similar transformation needs to take place in the cell therapy starting material supply chain. The availability of high-quality GMP starting material must be scaled up to meet demand. Scale-up of GMP products as complex as cell and gene therapies will require improvements that so far are just in the fledgling stage. To make these products safe, reliable, and cost-effective, the industry needs to expand donor access, quality control systems, and GMP processing capability. Newly expanded GMP-compliant workspace [3] is a laudable step in the right direction. HemaCare’s expanded capabilities are designed to support industry requirements through expedited access to GMP products and initial processing services, as well as increase downstream manufacturing throughput by offering more refined and consistent starting material.

Cell and gene-based therapies hold great promise to treat or even cure life-threatening diseases that have so far been intractable to treatment. We hope to continue to see new guidance and innovation supporting the safe and efficient introduction of these novel therapies. 


  1. S. Food and Drug Administration. Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. Jan. 2019.
  2. BioLife Solutions. New Policies to Advance Development of Cell and Gene Therapies Announced by FDA. Jan. 2019.
  3. HemaCare Details Expanded GMP Capabilities at CAR-TCR Summit in Boston, MA. Market Watch (press release). Sept. 2018.

Topics: Gene Therapy, Cell Therapy, GMP Cellular Therapeutics

Nancy Andon, MSc

Written by Nancy Andon, MSc

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