FDA Approves First CRISPR-based Gene Therapies to Treat Patients with Sickle Cell Disease

The U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an

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VERVE-101: CRISPR-Based Gene Editing Therapy Shows Promise in Reducing LDL-C and PCSK9 Levels in Patients With HeFH

A single infusion of a CRISPR-based gene editing therapy significantly reduced LDL-C and PCSK9 levels in patients with heterozygous familial hypercholesterolemia (HeFH), based on findings from the VERVE-101 trial presented Nov. 12 at AHA 2023. In presenting the findings, Andrew Bellinger, MD, PhD, said they provide the first proof-of-concept for in vivo DNA base editing in humans.

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Novel CRISPR/Cas9-based gene-editing therapy shows promise for patients with transthyretin amyloid cardiomyopathy

A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association’s Scientific Sessions 2022. The meeting, held in person in Chicago and

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New University of California Irvine-led report Illustrates potential of precision genome editing in treating inherited retinal diseases

Improvements in technology and delivery techniques could revolutionize treatment of genetic disorders of vision In a new paper, University of California, Irvine researchers explain how precision genome editing agents have enabled precise gene correction and disease rescue in inherited retinal diseases (IRDs). The study, titled, “Precision genome editing in the eye,” was published this week

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Geneticists retract study suggesting first CRISPR babies might die early

Researchers rapidly corrected finding through discussions on social media and preprints. Source Nature A study that raised questions over the future health of the world’s first gene-edited babies has been retracted because of key errors that undermined its conclusion. The research, published in June 2019 in Nature Medicine1, had suggested that people with two copies of a natural genetic mutation

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Carnegie Mellon and Yale Universities Receive NIH Grant To Further innovative, synthetic nucleic acid-based gene editing technique

The peptide nucleic acid-based technique offers an alternative to CRISPR-Cas9 A research team from Carnegie Mellon University and Yale University will advance their innovative, synthetic nucleic acid-based gene editing technique under a new grant from the National Institutes of Health’s (NIH’s) Somatic Cell Genome Editing (SCGE) Program. “Our PNA technique offers a promising avenue for treating — and possibly curing —

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