Medical Xpress

NOVEMBER 7, 2022

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 (..)

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STAT News

JULY 12, 2022

Somewhere in New Zealand, the first patient ever has been dosed with a kind of gene-editing treatment known as a base editor, a newer way of utilizing CRISPR for gene editing. The company studying the treatment, Verve Therapeutics, announced the news Tuesday. Continue to STAT+ to read the full story…

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Medical Xpress

JANUARY 20, 2023

A team of researchers at the University of Texas Southwestern Medical Center has found that it is possible in mice to protect the heart from ischemia-reperfusion using the CRISPR-Cas9 gene editing system.

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Gene Editing Genomics Genome Gene 81

Scienmag

DECEMBER 3, 2020

Those two key problems – safety and efficacy – are what continue to hold CRISPR-Cas9 gene targeting back from its full clinical potential, explains co-senior author Y. Eugene Chen, M.D., […].

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Gene Editing Gene DNA Genetics 41

XTalks

SEPTEMBER 4, 2024

The data, presented at the European Society of Cardiology Congress 2024, suggest that vutrisiran could significantly reduce the risk of death and heart-related issues, marking a potential treatment breakthrough. Vutrisiran works by silencing the gene responsible for producing TTR, reducing both mutant and wild-type TTR proteins.

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XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. 2022 was the first year on market for cell-based gene therapies Abecma (Idecabtagene vicleucel) and Breyanzi (lisocabtagene maraleucel) , which generated $388 million and $182 million, respectively. Both are anti-CD20 agents.

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