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Cutting a new path

Where gene editing therapies are heading next

As gene editing starts to make headway, its first indications are informed by the path laid by gene therapies and other nucleic acid modalities. But drug developers think editing is poised to move far beyond those applications, and are eyeing infectious disease and regenerative medicine as areas where the technology could be best differentiated.

According to gene editing companies interviewed by BioCentury, the sector’s first disease areas were picked largely based on the ground broken for delivery vectors by the antisense and gene therapy fields.

“Where you see folks starting is where you can more readily adapt existing delivery approaches to get to cells: eye, blood, liver,” said Katrine Bosley, CEO of Editas Medicine Inc. Around mid-year, Editas plans to submit an IND for EDIT-101 to treat Leber congenital amaurosis type 10 (LCA10), a form of blindness. The compound would become the first in vivo CRISPR-based gene editing therapy in the clinic outside of China.

The first wave of gene editing programs covers 24 indications, 16 of which overlap with gene therapy or antisense programs in the clinic (See “Road Less Traveled”).


Figure: Road less traveled

While gene editing companies are going after many of the same standard indications that gene editing and antisense therapy developers have already brought to the clinic, they also want to branch out into distinct indications.

According to BioCentury’s survey of 14 different gene editing company websites, at least 24 different indications, excluding cancer, are already represented in preclinical pipelines. The indications

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