Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

Story Feb. 13 - Encoded Therapeutics: The gene therapy biotech is laying off 29% of staffers as it focuses resources toward its Dravet syndrome program. Most of the affected roles will be in ...

US President Donald Trump kicked off his second term with a series of directives aimed at the US National Institutes of ...

Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...

Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of symptoms in children, including seizures, intellectual disability, and even ...

ETH researchers have developed a new gene switch that can be activated using a commercially available nitroglycerine patch ...

Regenxbio's gene therapy faces market challenges as Goldman Sachs lowers its rating and price target, citing competition and ...

Orchard Therapeutics has tasked Robin Kenselaar with accelerating growth of Lenmeldy, the world’s most expensive drug, ...

CSL and uniQure's Hemgenix shows sustained efficacy in hemophilia B, reducing bleeding rates by 90% and eliminating ...

ExpressionEdits' Genetic Syntax Engine platform is designed to enhance gene expression within gene therapies by restoring introns.

Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics and headquartered in Switzerland, ...