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gene therapy

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.

Nature News reports that gene therapy approaches are tackling sickle cell disease, but that the cost of treatment is a concern.

Pause Button Hit

Novartis has paused a trial of its Zolgensma gene therapy that treats spinal muscular atrophy, the Wall Street Journal reports.

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

Pages

Novavax has begun a phase III trial of its SARS-CoV-2 vaccine, according to the New York Times.

Vox reports that the Trump Administration may limit student visas for individuals from some countries to two years.

The governor of New York says the state will conduct its own review of any SARS-CoV-2 vaccine, NPR reports.

This week in Science: Neanderthal Y chromosomes replaced by Homo sapiens Y chromosomes, and more.