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gene therapy

Health insurers in the US are devising new approaches to handle the high costs of gene therapies, the Wall Street Journal reports.

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.

Pages

Russian CRISPR researcher moves along with plans to ultimately alter the genes of embryos of deaf couples, though awaits regulatory approval, Nature News reports.

University of California, San Francisco, researchers have uncovered a gene mutations that appears to make a father-son duo more efficient sleepers.

NPR reports a large health insurer has begun to cover some pharmacogenetic tests for psychiatric drugs.

In PLOS this week: genome-wide association study of non-syndromic orofacial cleft subtypes, epigenetic and transcriptomic analysis of pancreatic ductal adenocarcinoma, and more.