Cell and gene sector needs regulatory clarity says Andelyn

Cell and gene therapy developers are still uncertain of regulator expectations according to Andelyn Biosciences, which says industry would benefit from more balanced oversight.

Various agencies have invested a lot of effort in the development of regulatory frameworks for cell and gene therapy development and manufacture.

Recent examples include the US FDA’s draft guidance on trialing multiple versions of a cell therapy and the EMA’s document on quality expectations for products containing genetically modified cells.

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While these efforts are a move in the right direction, there are some areas where clarification is needed according to Andelyn Biosciences’ CEO Mayo Pujols, who told us some developers have experienced difficulties as their programs mature from early phase to late phase.

“The central issue is uncertainty on the approach to comparability, specifications, and overall data required to commercialize their programs. With a large and diverse number of customers and product indications, each seems to have their own set of regulatory challenges as many of them have not been explored in the past to build upon. This increases the complexity and the need for unique data sets at times.”

Balanced expectations

The problem is not that regulatory agencies do not understand the scientific challenges involved in developing cell and gene therapies, rather it is that they are used to assessing processes used to make biopharmaceutical products, Pujols said.

“Most regulators we interact with are well informed of the complex nature and limitations of the current cell and gene therapy production and analytical platforms. However, their expectation is that processes and assays used to produce cGMP grade product need to meet the same level of reliability and robustness as other complex biologics and vaccines.

“This is not an unreasonable expectation, but the industry could greatly benefit if the approach was more balanced and harmonized as much as possible. There’s certainly a need for continued discussions on what is possible with today’s production and analytical technology and on finding the right balance to allow these breakthrough treatments to reach patients much faster.”


One positive is that those regulations that are in place to govern the production of cell and gene therapies do not vary significantly between regions, according to Pujols.

“We have seen some minor differences between the regulatory bodies across the large markets in terms of areas of areas focus and speed. However, our conclusion is that there’s no ‘easier’ market from a clinical trial or commercial approval perspective, since it is very program specific and industry continues to collaborate with regulatory bodies to routinely improve processes, safety, and performance expectations.”