Patient groups push abandoned rare disease gene therapy toward clinical trial

Two rare disease groups have produced the key funding needed to make a gene therapy...

Published in Nature Medicine: Breakthrough gene therapy trial for Tay-Sachs Disease

Blu Genes Foundation supported: Duel-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase I/II trial...

Baby Is Healed With World’s First Personalized Gene-Editing Treatment

Article: By Gina Kolata, The New York Times – May 15, 2025 The technique used...

Study identifies a potential treatment for Sandhoff and Tay-Sachs diseases

In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff...

Blu Genes supports gene therapy at University of Wisconsin-Madison. Gene therapy protects against motor neuron disease.

Anjon Audhya The mutation is in the Trk-fused gene, which typically facilitates the transportation of...

Taylor Swift tickets being auctioned off to help fund for Ottawa boy with rare disease

A pair of Taylor Swift tickets are being auctioned off in Ottawa to help fund...

Saskatchewan artist fundraising to help family whose son has genetic disease

A Regina based artist is using his platform to fundraise in hope of helping a...

Family with child affected by SPG4 visits UMass Chan researchers developing gene therapy

A family from Ottawa, Canada, seeking a cure for spastic paraplegia type 4 (SPG4), a...

Ottawa community helping fundraise for boy with rare disease

Ottawa came together in Stittsville on Saturday, raising more than $15,000 to help fund a...

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

Today, the U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene...