Tag Archive for: CRISPR Therapeutics

The regulator’s advisory committee on Tuesday found Vertex Pharmaceuticals and CRISPR Therapeutics’ off-target analysis for its sickle cell disease candidate exa-cel to be sufficient.

In a briefing document for Tuesday’s advisory committee meeting, the FDA raised concerns about the potential off-target effects of Vertex Pharmaceuticals and CRISPR Therapeutics’ investigational gene edited therapy.

Currently, there are no gene editing–based treatments on the market, but the technology continues its march toward potential FDA approval, with several products in mid- and late-stage trials. As these programs mature, 2023 could be a pivotal year for companies in the space.

Genome editing has been a hot-button topic in recent years. With the rise of CRISPR technology in the news, this is not surprising for either scientists or investors.

Multimillion-dollar gene therapies being tested for sickle cell disease will be cost-effective in the United States if health disparities tied in part to decreased access to healthcare are taken into account, researchers said at a meeting of blood-disease specialists.

The nonprofit biotech Cure Rare Disease (CRD) is headed to the clinic with a CRISPR therapeutic aimed at halting the progression of Duchenne muscular dystrophy in single-patient dosing.

Precision BioSciences entered into an exclusive global in vivo gene editing research and development collaboration and license deal with Novartis Pharma. The two companies will work to develop a potential cure for hemoglobinopathies such as sickle cell disease and beta-thalassemia.