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

09 Nov 2023

Gene TherapyClinical StudyIND

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 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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Organizations

Intellia Therapeutics, Inc.

Ascidian Therapeutics, Inc.

Indications

Alpha 1-Antitrypsin DeficiencyLung Diseases

Targets

A1AT

Drugs

NTLA-3001NTLA-2003

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