Amyloidosis
AstraZeneca Amyloidosis Drug Fails Phase 3 Trials but Shows Potential in Subgroup
AstraZeneca; amyloidosis; anselamimab; Phase 3 trial; clinical trial failure; prespecified subgroup; rare diseases; Alexion; AL amyloidosis; drug development
Intellia Therapeutics Slashes Workforce by 27% to Focus on Late-Stage CRISPR Candidates Amid Challenging Market Environment
Intellia Therapeutics, CRISPR, Workforce reduction, Late-stage drug candidates, NTLA-2002, Nexiguran ziclumeran (nex-z), Hereditary angioedema, Transthyretin (ATTR) amyloidosis, Commercialization strategy
Alnylam’s Vutrisiran Shows Promise in ATTR-CM Treatment, but Faces Competition
Alnylam, Vutrisiran, ATTR-CM, Phase 3 Trial, HELIOS-B, RNAi Therapeutics, Cardiomyopathy, Amyloidosis
CRISPR Gene Editing Therapy Can Be Administered Twice, Intellia Study Suggests
CRISPR, gene editing, Intellia Therapeutics, transthyretin amyloidosis, gene therapy, re-dosing
Alnylam Achieves Breakthrough in Heart Study with RNAi Therapy Vutrisiran
Alnylam, Vutrisiran, RNAi Therapy, Heart Study, ATTR Amyloidosis, Cardiomyopathy