Moderna on Friday evening became the second developer of a COVID-19 vaccine to be granted an emergency use authorization (EUA) by the FDA, allowing patients to be dosed with the company’s messenger RNA (mRNA)-based mRNA-1273 just a week after the agency granted the first.
The company said the first doses of mRNA-1273 will be injected into patients next week. Like Pfizer and BioNTech’s BNT162b2, which received an EUA on December 11, mRNA-1273 is a two-dose vaccine. But Moderna’s vaccine is indicated for the prevention of COVID-19 in individuals 18 years of age and older, rather than 16 and older as BNT162b2 is indicated.
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).