Aurora Therapeutics' first target is the rare inherited disease phenylketonuria, also known as PKU. Here at MIT Technology Review we’ve been writing about the gene-editing technology CRISPR since 2013 ...

Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA regulatory pathway to commercialize treatments for rare diseases. Last February ...

A new CRISPR breakthrough shows scientists can turn genes back on without cutting DNA, by removing chemical tags that act like molecular anchors. The work confirms these tags actively silence genes, ...

As he addressed an audience of virologists from China, Australia, and Singapore at October’s Pandemic Research Alliance Symposium, Wei Zhao introduced an eye-catching idea. The gene-editing technology ...

In the five years since Italy-based Chiesi Group established its rare disease division in Boston, the unit has landed regulatory approvals worldwide for 10 therapies — all small molecules and ...

Since the beginning of the HIV epidemic, scientists, doctors, and public health experts have spent decades trying to understand the virus and control its spread. Modern treatments now allow people ...

A Chinese scientist, He Jiankui, made a shocking announcement to the world in 2018: He had secretly engineered the birth of the first gene-edited... The tech behind genetically modified babies is ...

Scientists at St. Jude Children’s Research Hospital report that they have developed an unbiased, sensitive, and resource-efficient method to identify small, off-target sites that pose a safety risk ...

Scientists and physicians can better assess precision genome editing technology using a new method made public today by St. Jude Children's Research Hospital. Significant amounts of time and resources ...

When gene therapy was first proposed in the 1970s, the idea seemed straightforward but revolutionary: replacing defective genes with functional ones to cure a disease. But over the decades, this ...