Multiple gene therapies will be approved for marketing, and the FDA plans to release 6 new guidelines in July.


With the end of the BIO International Convention in 2018, Jim Greenwood, President and CEO of Biotechnology Innovation Organization (Bio), interviewed Scott Gottlieb, Director of the US Food and Drug Administration (FDA).

Gottlieb said: “There are currently hundreds of clinical new drugs for cell and gene-related treatments being applied for, and the FDA is expected to approve 40 gene therapies by 2022. There is no doubt that people will be able to see it in the near future. A variety of curative therapies. If I was the director five years ago and said that we can cure some genetic blood-borne diseases like sickle cell anemia, I think this would be irresponsible, but from Now, we can do it in the next ten years.”

Then, Gottlieb did admit that any treatment is at risk, but he said that with the development of the virus as a carrier technology, genetic therapy continues to improve, which also shows that “product innovation leads to a clinical revolution.” In addition, the complexity of many gene therapies is product-related and not clinically relevant. However, in the normal drug review, 80% is clinically relevant and the remaining 20% ​​is related to Chemical Manufacturing and Controls (CMC) and products. Because gene therapy and cell regeneration medicine are the exact opposite, the FDA must think differently about these regulatory issues. In response, the FDA expects to release six new gene therapy guidelines in July to provide a “modern architecture” for the therapy, most of which will focus on product-related issues, with the remaining focus on accelerating clinical endpoints.

Gottlieb also announced a new project called “Interact” at the Center for Biologics Evaluation and Research for preclinical testing and early involvement in cell and regenerative therapies. That is, prior to the Investigational New Drug (IND), the FDA will establish a formal structure for early engagement that addresses how to bring products into the IND phase, address CMC issues, and other clinical development issues.

Gottlieb concluded that the biotech industry should move in the direction of continuous production of cell and gene therapy to help seamlessly integrate research and commercial manufacturing. In 2019, the FDA will set a large budget to strengthen cooperation between the government and private companies related to continuous production.


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