A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin ...

A one-time gene therapy called AMT-130 helped slow the progression of Huntington's disease by 75%, paving the way for the ...

Stunning success in halting Huntington's disease with gene therapy offers new hope to those with the disorder—and potential ...

Breakthrough Therapy designation granted based on FDA’s review of available clinical evidence of safety and efficacy from all ...

Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.

One oral presentation and five poster presentations will highlight exciting progress in AskBio’s pipeline and advancements in ...

French biotech EG 427 has had success with its urinary incontinence DNA therapy, EG110A, showing a more than 80% reduction in ...

Treatment introduces an expansion of the scope of gene therapies that might lead to similar treatments for neurodegenerative ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the ...

D Molecular Therapeutics (FDMT) offers attractive growth potential with strong cash reserves and promising late-stage gene ...

Taysha Gene Therapies' investigational treatment for Rett syndrome has received breakthrough-therapy designation from the Food and Drug Administration.

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct ...