Clinical Trials and Research
Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, today…
The first team to disable a disease gene directly in a person through an infusion of the genome editor CRISPR reported yesterday that levels of the toxic protein…
Intellia Therapeutics, Inc. and Regeneron Pharmaceuticals, Inc. announced positive interim data from an ongoing Phase 1 clinical study of their lead in vivo genome…
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics leveraging CRISPR-based…
Cardiac amyloidosis is not nearly as rare as one might think, and with effective treatments finally emerging in recent years, there's a need to increase awareness…
The treatment uses CRISPR/Cas9 editing technology, a technique derived from bacteria that allows researchers to edit the genome of a cell by specifying regions of…
Researchers from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners have published the first-ever clinical data that support the safety and…
People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually…
The gene editor CRISPR excels at fixing disease mutations in lab-grown cells. But using CRISPR to treat most people with genetic disorders requires clearing an…
Preliminary results from a landmark clinical trial suggest that CRISPR-Cas9 gene editing can be deployed directly into the body to treat disease.
The study is the…