BioCentury
ARTICLE | Clinical News

Adverum reviewing capsid after discontinuing AAT gene therapy

November 2, 2018 2:45 PM UTC

Adverum Biotechnologies Inc. (NASDAQ:ADVM) sank $1.21 (27%) to $3.34 on Friday after it said it will discontinue development of gene therapy ADVM-043 to treat α-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency. The company said late Thursday that although ADVM-043 was well tolerated among six patients in the Phase I/II ADVANCE trial, M-specific AAT protein measurements failed to reach clinically meaningful levels of expression.

Adverum said it will review the study's results to inform development of its other gene therapies for rare diseases, particularly those using the AAVrh.10 capsid used in ADVM-043 to deliver AAT. The company no longer intends to submit an IND this quarter for ADVM-053, which uses the same capsid to deliver human complement 1 (C1) esterase to treat hereditary angioedema (HAE). Adverum plans to provide a broader rare disease pipeline update next half...