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The first treatment based on the Nobel Prize-winning Crispr technology is poised to win FDA approval next month, offering hope to patients and opportunities to investors.
New research seems to show the potential of a CRISPR-based gene therapy meant to neuter certain forms of cardiovascular disease. In the world’s first human trial of the therapy, people given the highest doses experienced significant and long-lasting reductions in their low-density lipoprotein cholesterol (LDL-C). The treatment could be used to help people genetically predisposed to high LDL-C and its accompanying health risks.
Dublin, Oct. 31, 2023 (GLOBE NEWSWIRE) -- The
If the therapy is approved, Vertex has proposed a 15-year follow up of patients to evaluate the safety outcomes of the therapy. A vaso-occlusive crisis occurs when sickled red blood cells block blood flow to the point that tissues become deprived of oxygen and causes pain. The panel members said the 15-year follow up will help generate data from real-time monitoring of the therapy, which uses the new gene editing CRISPR technology.
(Reuters) -The U.S. Food and Drug Administration's staff reviewers on Friday asked the regulator's advisory panel to consider the need for additional studies for Vertex Pharmaceuticals and CRISPR Therapeutics' sickle cell disease gene therapy. The therapy, called exagamglogene autotemcel or exa-cel, uses new gene editing CRISPR technology, and is the first-of-its-kind product to reach the FDA for an approval decision.