Earlier this week, Krystal Biotech reported positive interim data from the highest-dose cohort of its Phase 1 CORAL-1 trial, ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

A retired HGV driver was the first person in the world to take part in a clinical trial to extend the lifespan of blood ...

A new treatment might just be hope for many people living with chronic pain. In a recent preclinical study, researchers ...

An update from the phase 2 LEGEND trial shows that the investigational nonviral gene therapy detalimogene voraplasmid has achieved a 62% complete response rate at six months in patients with high-risk ...

Children with hereditary deafness regained their hearing thanks to a type of gene therapy, a new study published on Wednesday found. In a clinical trial, co-led by investigators from Mass Eye and Ear, ...

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

Potentially more than 90% of Alzheimer's disease cases would not occur without the contribution of a single gene (APOE), ...

A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...