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Exciting advancements in the realm of genome editing!

5th March 2024

The FDA recently released finalised guidance on Human Gene Therapy Products Incorporating Human Genome Editing, providing a comprehensive roadmap for innovators in this field. 

In recent years, genome editing technology has progressed rapidly, enabling the production of cell-based therapies for many intractable diseases. However, the unique nature of these therapies can pose challenges for regulation within traditional frameworks, especially as they continue to evolve at a rapid pace.  

This guidance, which follows extensive collaboration with scientific communities, provides crucial insights into the implementation and control of human genome editing in therapeutic product development. 

From ex vivo modified cell-based therapies to in vivo targeted gene therapies, the FDA’s recommendations span the entire development trajectory, offering clarity and direction for the industry.  

So, what’s new in this final guidance?  

While the structure and content of the final guidance are similar to the draft version, there are some interesting changes that impact the cell and gene therapy industry: 

• A distinction is drawn between routine gene editing in drug product manufacturing and one-time editing upstream of a master cell bank, allowing for reduced manufacturing requirements for the latter. 

• It emphasizes retaining test articles for future analysis, acknowledging the fast-paced evolution of gene editing technology.  

• Based on input from the scientific community, the final guidance also affirms that human gene editing products could qualify for accelerated approval, particularly when addressing serious or life-threatening diseases with limited alternative treatments. 

We recommend reading this article to fully understand the implications for developers and suppliers alike

Stay ahead of the curve and follow us for more science news and updates!  

#GenomeEditing #FDA #Biotech #CellBasedTherapies 

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