Tag Archive for: CRISPR

A recent study carried out by researchers at Tulane University and published in the American Journal of Respiratory and Critical Care Medicine unveiled a novel diagnostic tool that can identify Mycobacterium avium complex (MAC) nontuberculous mycobacterial (NTM) infections in as little as two hours.

In late 2023, Vertex Pharmaceuticals and CRISPR Therapeutics made history by gaining the first FDA approval for a CRISPR-based drug, exagamglogene autotemcel (Casgevy) for the treatment of sickle cell disease with vaso-occlusive crisis. This historic achievement coincided with a record number of 14 review designations awarded by the FDA to CRISPR-based therapies in 2023, according to GlobalData.

The announcement comes just a month after Casgey was approved by the FDA in December 2023 to treat patients with sickle cell disease, when it became the first CRISPR gene editing therapy to reach the U.S. market.

Vertex still has an ongoing diabetes partnership with CRISPR, which gives it a non-exclusive license to the latter’s platform to develop a potential cure for type 1 diabetes.

Moderna-backed Metagenomi is a precision genetics medicines company with what it describes as a comprehensive genome editing toolbox consisting of programmable nucleases, base editors, RNA-mediated integration systems and CRISPR-based techniques.

The agency approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel Prize-winning CRISPR gene editing technology.

Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020, Britain’s Medicines and Healthcare products Regulatory Agency (MHRA) said.

The agency’s staff reviewers said it was not clear if the limited donor cells used for assessment were adequate to evaluate the potential safety risks of the therapy.

The New England Journal of Medicine study suggests that the 2022 death of a Duchenne muscular dystrophy patient was due to an adverse effect of recombinant AAV used to deliver the gene therapy.

The Institute for Clinical and Economic Review raised the cost estimate for two experimental gene therapies, stating that the new price can be cost effective to treat sickle cell disease.