The FDA is unveiling a faster approval pathway for custom gene-editing therapies, aiming to spark major investment and accelerate cures for rare diseases through Crispr-based innovations.
Intellia Therapeutics halts MAGNITUDE trials of nex-z gene therapy after patient hospitalization with Grade 4 liver toxicity. Over 650 patients enrolled in ATTR amyloidosis studies.
Jefferies sets $41 price target for Beam Therapeutics, highlighting BEAM-302's potential as one-time treatment for rare genetic disease with $2.5B peak sales opportunity.
Beam Therapeutics rallied after Wave Life Sciences' WVE-006 trial data for alpha-1 antitrypsin deficiency (AATD) fell below expectations.
