Intellia plans to file IND for AATD trial next year; Ascidian raises $40M

In­tel­lia aims to start AATD tri­al next year: The gene edit­ing com­pa­ny an­nounced Thurs­day that it plans to file an ap­pli­ca­tion to start clin­i­cal tri­als for an al­pha-1 an­tit­rypsin de­fi­cien­cy (AATD) lung dis­ease pro­gram in the first quar­ter of next year. This would be In­tel­lia’s third pro­gram to reach hu­man tri­als. Un­like its cur­rent two pro­grams, which knock out dis­ease-caus­ing genes, the AATD ther­a­py would in­volve in­sert­ing a healthy copy of a gene en­cod­ing al­pha-1 an­tit­rypsin pro­tein. In­tel­lia did not spec­i­fy whether it plans to be­gin tri­als for NT­LA-3001 in the US or abroad. The biotech al­so said it stopped work on NT­LA-2003, its oth­er pre­clin­i­cal AATD gene edit­ing treat­ment, which was meant for the liv­er as op­posed to lung dis­ease. NT­LA-2003 was meant to knock out the gene pro­duc­ing ab­nor­mal pro­teins in the liv­er. — Lei Lei Wu

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