Clinical Trials and Research
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,…
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi…
The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food…
Researchers at Boston Medical Center (BMC) and Boston University School of Medicine (BUSM), in collaboration with Columbia University Irving Medical Center (CUIMC…