Combining first-in-class delivery and engineering tools, Ensoma is developing one-time in vivo treatments that can ... short palindromic repeats (CRISPR)–associated protein 12a (Cas12a)-based ...

So far, human trial data for gene-editing drugs have been generated for ex vivo therapies, meaning the CRISPR technology is used to modify cells outside the body, which are then reinfused into the ...

The two companies claim AGN-151587 (EDIT-101) for Leber congenital amaurosis type 10 (LCA10) – an inherited form of blindness – is the first ‘in vivo’ CRISPR medicine to be administered to ...