Viz.ai has submitted a de novo classification request to the FDA for a new AI algorithm intended to identify and triage patients suspected with HCMSAN FRANCISCO (BUSINESS WIRE) #ai Viz.ai, a leader in AI-powered disease detection and care coordination, today announced a multi-year agreement with Bristol Myers Squ.
CRISPR Gene Editing Delivers Promise for Sickle-Cell Disease, Beta Thalassemia
CRISPR Therapeutics CEO Samarth Kulkarni, PhD
CRISPR Therapeutics and Vertex Pharmaceuticals have reported a consistent and sustained positive response in 10 patients treated for a pair of blood disorders sickle-cell disease (SCD) and beta thalassemia with their CRISPR-Cas9 gene-edited therapy CTX001 in a pair of Phase I/II trials. These are the first clinical studies of a CRISPR gene-editing candidate sponsored by U.S. companies.
According to data published last Saturday in the
New England Journal of Medicine (NEJM) and presented Sunday at the annual American Society of Hematology (ASH) Meeting and Exposition, all seven patients with transfusion-dependent beta thalassemia (TDT) including three who have either a severe or b0/b0 genotype were transfusion independent at the last follow-up in the TDT trial, which is known as CLIMB-111 (NCT03655678).