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gene therapy

In Two

Bluebird Bio is splitting into two companies, the Wall Street Journal reports.

In Both

A gene therapy for Leber hereditary optic neuropathy given to one eye affected vision of the other, according to New Scientist.

Spectrum reports that a clinical trial of a gene therapy for Angelman syndrome is on hold.

Asks for More

Reuters reports that the Food and Drug Administration has rejected BioMarin's gene therapy for hemophilia A and has asked for longer-term data.

Mission Bio will use a recent $70 million funding round to develop quality control assays using its Tapestri single-cell analysis platform.

A High Price

A gene therapy for hemophilia could cost millions of dollars, NPR writes.

The companies are applying Personalis' technology to explore immune response to genetic therapeutics in Sarepta's pipeline.

GenomeWeb asked a number of prominent researchers in the field via email about their top picks for notable developments or achievements in genomics during the 2010s.

Walking Around

A Minnesota toddler given a gene therapy to treat her spinal muscular atrophy is now walking, according to Newsweek.

Therapy Lottery

The Guardian reports that parents of children with spinal muscular atrophy are concerned about a plan to have a lottery for free gene therapy doses.

Pages

The Wall Street Journal reports on gaps in COVID-19 testing affecting less affluent urban areas and rural locations.

According to NBC News, new SARS-CoV-2 variants are making it harder for researchers to model the course of the pandemic.

The New York Times reports that experts say President Joe Biden's goal of vaccinating 1 million people a day in the US in the next 100 days is too low a bar.

In Science this week: single-cell lineage tracing technique applied to study lung cancer metastasis, and more.