Chinese gene editing therapies are developing

China’s race to test ‘mutation-free’ gene-editing technology on cancer patients

BEIJING — China could be just over a year away from clinical trials of a new gene-editing therapy with an unprecedented high level of safety, according to a team of Chinese scientists involved in the research programme.

The scientists said the research, based on groundbreaking work published in the journal Science earlier this month, could help save the lives of many patients battling deadly diseases including cancer.

The existing genome-editing method works like a shotgun, breaking up a large numbers of genome strands and sometimes missing its intended target, causing unnecessary damage to cells.

The new tool under development in China targets and swaps individual “letters” in the DNA with extreme precision, avoiding cuts to the strands and significantly reducing the risk of unexpected mutations.

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Bloomberg covers important investor interest in gene editing therapyBloomberg covers important investor interest in gene editing therapy

Biotech Investors Zero In on Gene Therapy as Next Big Frontier

(Bloomberg) -- Biotech investors are bullish on the market for gene therapies, even though actual sales of a few existing drugs have so far failed to impress some Wall Street analysts. Fund managers and venture capitalists discussed their outlook for the technology in interviews in Boston, where investors gathered for the Cowen health-care conference. Investor interest in gene-therapy stocks is high, as investing has paid off this year. Roche Holding AG and Biogen Inc. recently snapped up a pair of drug developers, boosting speculation of more deals. Also, U.S. regulators say they expect to approve 10 to 20 new medicines by 2025, while insurers grapple with treatment costs that can run into the hundreds of thousands of dollars.

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CRISPR corrects Duchenne muscular dystrophy mutation

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

Abstract

Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete exon 44 of the dystrophin gene represent one of the most common causes of DMD and can be corrected in ~12% of patients by editing surrounding exons, which restores the dystrophin open reading frame. Here, we present a simple and efficient strategy for correction of exon 44 deletion mutations by CRISPR-Cas9 gene editing in cardiomyocytes obtained from patient-derived induced pluripotent stem cells and in a new mouse model harboring the same deletion mutation. Using AAV9 encoding Cas9 and single guide RNAs, we also demonstrate the importance of the dosages of these gene editing components for optimal gene correction in vivo. Our findings represent a significant step toward possible clinical application of gene editing for correction of DMD.

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