Vertex Pharmaceuticals expanded the company’s collaborative partnership with CRISPR Therapeutics to develop and commercialize a possible cure for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The focus is on the development of CTX001, an autologous, ex vivo CRISPR-CAS9 gene-edited therapy.
The Nobel Prize in Chemistry was awarded to Emmanuelle Charpentier and Jennifer A. Doudna for their discovery and development of CRISPR gene editing.
Bay Area-based Graphite Bio launched with $45 million in Series A financing and a goal to use gene editing to correct defective genes through high-efficiency site-specific integration of new genetic sequences.
Light-activated CRISPR technology edits genes more precisely and faster than the more standard chemical methods, and enables “super-fast” DNA repair, according to research from Johns Hopkins University School of Medicine.
Intellia Therapeutics is focused on implementing a full-spectrum genome editing approach, as the company has a pipeline spanning both in vivo and ex vivo therapies to address severe and life-threatening diseases.
Researchers with the Abramson Cancer Center of the University of Pennsylvania, led by Carl June, published results from the first U.S. Phase I trial of CRISPR-Cas9-edited T-cells in humans with advanced cancer.
CRISPR Therapeutics and Vertex Pharmaceuticals announced positive interim data from the first patients in the companies’ Phase I/II clinical trials of the CRISPR/Cas9 gene-editing therapy CTX001.