The ATTR pipeline: Data Byte
Competition continues to heat up in the rare disease transthyretin amyloidosis (ATTR), with at least a dozen preclinical and clinical programs coming up behind the three marketed agents, according to BioCentury’s BCIQ database.
The indication also continues to serve as a testing ground for new modalities, with the latest development coming last month when Intellia Therapeutics Inc. (NASDAQ:NTLA) presented the first clinical proof-of-concept data for a systemically delivered CRISPR-based gene editing therapy, showing a single dose of NTLA-2001 reduced serum TTR levels in patients with hereditary transthyretin amyloidosis (ATTR) with polyneuropathy. ...