While offering curative potential, much remains to be known about the long-term clinical activity and safety of gene editing therapies. We expect answers to these complex questions will continue to be a key investor focus as programs advance.
While the clinical and regulatory progress of gene therapies may inform the road ahead for gene editing, permanent genomic changes will likely yield distinct advantages and challenges. Advancements in editing efficiency, the characterization of on – and off -targeted editing, and pre -conditioning regimens are likely focus areas in the years ahead. We have identified nearly 190 gene editing programs across broad therapeutic areas.
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