Clinical Trials and Research
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…
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today announced that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi…
The results of the phase 1 study open-label trial showed the therapy was safe, raising the possibility of a more effective and more appealing treatment for the…
The FDA has approved daratumumab and hyaluronidase-fihj (Darzalex Faspro), a subcutaneous formulation of daratumumab, for use in combination with bortezomib,…