STAT News

SEPTEMBER 16, 2022

Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed inside the body — found the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low- and high-dose cohort, respectively.

Gene Editing 94

Gene Editing Genetic Disease Genetics Protein 94

Camargo

JULY 27, 2021

Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

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In-Vivo Gene Editing Gene Therapy Gene 52

XTalks

DECEMBER 13, 2023

The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US.

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Gene Therapy FDA Approval Gene In-Vivo 98

pharmaphorum

JANUARY 10, 2022

The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases. This not only allowed Pfizer to establish a COVID-19 vaccine but the company also recently announced that it had begun a study testing an mRNA flu vaccine.

RNA 109

RNA Vaccination Vaccine Pharma Companies 109

XTalks

MAY 4, 2021

The CRISPR gene editing system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing. Epigenetic Editing with CRISPR.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

MAY 4, 2021

The CRISPR gene editing system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing. Epigenome Editing with CRISPR.

DNA 52

DNA Gene Gene Silencing Genome 52