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Aw, A Success

A Forbes article entitled, “Genomics: No Longer A Failure,” says that Human Genome Sciences and Vertex Pharmaceuticals are finally living up to the hype promised during Human Genome Project. Human Genome saw positive results in a second study of its lupus drug, Benlysta, and Vertex presented mid-stage results of its hepatitis C drug, telaprevir, that indicated it could work as well if taken twice a day as three times a day, which is how it is currently being tested. Forbes notes that Benlysta would be “one of the first [drugs] to emerge from newfangled efforts to decipher the human genome that started in the early 1990s, amid enormous amounts of hype that it would lead to a huge cornucopia of new drugs.”

The Scan

Study Finds Few FDA Post-Market Regulatory Actions Backed by Research, Public Assessments

A Yale University-led team examines in The BMJ safety signals from the US FDA Adverse Event Reporting System and whether they led to regulatory action.

Duke University Team Develops Programmable RNA Tool for Cell Editing

Researchers have developed an RNA-based editing tool that can target specific cells, as they describe in Nature.

Novel Gene Editing Approach for Treating Cystic Fibrosis

Researchers in Science Advances report on their development of a non-nuclease-based gene editing approach they hope to apply to treat cystic fibrosis.

Study Tracks Responses in Patients Pursuing Polygenic Risk Score Profiling

Using interviews, researchers in the European Journal of Human Genetics qualitatively assess individuals' motivations for, and experiences with, direct-to-consumer polygenic risk score testing.