News brief­ing: In­tel­lia hits clin­ic with CRISPR ther­a­py; Vi­for con­tin­ues to spend, pay­ing up to $340M for DGF pro­gram

An­oth­er CRISPR ther­a­py has en­tered the clin­ic. In­tel­lia Ther­a­peu­tics, the Cam­bridge gene edit­ing biotech, an­nounced to­day that they dosed their first pa­tient with a ther­a­py meant to treat transthyretin amy­loi­do­sis, a rare ge­net­ic dis­ease caused by a mu­ta­tion in a liv­er pro­tein.

Al­though a hand­ful of CRISPR ther­a­pies have al­ready en­tered the clin­ic at In­tel­lia and else­where, the new study is no­table for be­ing just the sec­ond study to in­ject CRISPR di­rect­ly in­to a pa­tient. Oth­er ap­proach­es, such as those meant to treat sick­le cell ane­mia, in­volve tak­ing cells out of a pa­tient, edit­ing them, and then in­fus­ing them back in.

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Life Sciences Senior Associate

General Catalyst

San Francisco, CA, USA