Axovant Presents First Evidence of Clinical Stabilization in Tay-Sachs Disease at the European Society of Gene and Cell Therapy 27th Annual Congress

Initial data with AXO-AAV-GM2 suggests stabilization of disease course, attainment of normal developmental milestones, and...

Genome editing with precision

Prime editing system offers wide range of versatility in human cells, correcting disease-causing genetic variations...

$28 million grant supports Cincinnati Children’s role as coordinating center for Rare Diseases Clinical Research Network

The federal agency that oversees a large network of research centers seeking cures for hundreds...

New hope for Tay-Sachs disease at UMass Medical School

LISTEN Produced by UMass Medical School · New hope for Tay-Sachs disease A new Voices...

Blu Genes Afternoon Tea

Blu Genes is grateful to our Sponsors, supporters, friends and family for making our Afternoon...

AXOVANT announces clinical update from first Tay-Sachs disease patient dosed with AXO-AAV-GM2 gene therapy

Generally well-tolerated with no serious adverse events in a 30-month-old patient with advanced infantile Tay-Sachs...

New blood test detects genetic disorders in fetuses

Tel Aviv University researchers have developed a new blood test for genetic disorders that may...

UMMS licenses clinical-stage gene therapies for Tay-Sachs, similar diseases to Axovant

Axovant Sciences, a Swiss company developing innovative gene therapies for serious neurological diseases, has licensed...

New Approaches to Tay-Sachs Disease Therapy

Tay-Sachs disease belongs to the group of autosomal-recessive lysosomal storage metabolic disorders. This disease is...

Tay Sachs Sees Hope in First Gene Therapy Clinical Trial

Blu Genes Foundation Gives UMMS $1.4M(USD) to Bring Tay-Sachs Gene Therapy Approach to Clinical Trial...