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gene therapy

Novartis replaced two top executives from its AveXis unit following data manipulation during early testing of its new gene therapy, Reuters reports.

Data, Manipulated

US Food and Drug Administration says some data from early testing of Novartis' Zolgensma was manipulated, according to Reuters.

Slow Adoption

Reuters reports that slow adoption of CAR-T therapies might be tied to their high prices.

The company, which spun out of the Netherlands Cancer Institute, has developed technology to identify promoters and enhancers.

For Coverage

Parents of children with spinal muscular atrophy tell the Washington Post they are pushing to get insurance coverage of Novartis's Zolgensma.

Priciest Yet

Novartis's newly approved Zolgensma, a gene therapy for spinal muscular atrophy, is to cost $2.1 million, the AP says.

Looking Into It

A second death in gene therapy trial for type 1 spinal muscular atrophy is under investigation, according to Reuters.

St. Jude Children's Research Hospital scientists have treated infants with X-linked severe combined immunodeficiency using gene therapy in an early phase study.

The New York Times reports on new gene therapy efforts to treat sickle-cell disease.

Positive Review

A Canadian panel recommends public coverage of the gene therapy Kymriah if its cost comes down, the Globe and Mail reports.

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The US National Institutes of Health's All of Us project awarded $4.6 million to the company Color to develop a genetic counseling resource for the program.

The Times of India reports on a pilot study that used genomic testing to determine whether patients had drug-resistant tuberculosis.

New guidelines say that more women may benefit from genetic testing for hereditary breast or ovarian cancer, according to the Los Angeles Times.

In Cell this week: small proteins identified among human microbiome, role for tumor microbes in pancreatic cancer survival, and more.