Health ❯ Genetics

Gene Therapy

CRISPR Technology Induced Pluripotent Stem Cells Hereditary Deafness GBA1 Gene Experimental Treatment Mucopolysaccharidosis Type II Gaucher Disease Cell-Based Therapies CCR5 Gene Embryonic Editing

LEO Pharma to Acquire Replay in $50 Million Gene-Therapy Deal

The move underscores LEO’s effort to show a growth path ahead of a potential stock-market listing.

Norwegian Man Appears Cured of HIV After Brother’s Bone Marrow Transplant

AI Buildout Meets Growing Backlash as Maine Moves to Ban New Data Centers

FDA Maps New Paths for Rare-Disease Drug Evidence, Keeps Bar High

Ambroxol Trial Confirms Safety, Brain Delivery and Early Cognitive Benefits in Parkinson’s Dementia

Ambroxol Demonstrates Safety and Neuroprotection Signals in Parkinson’s Dementia Trial

First Personalized CRISPR Therapy Corrects Rare Genetic Disorder in Infant

Yale Researchers Identify Potential Treatment for Rare 'Smooth Brain' Disorders

Pioneering Gene Therapy Restores Hearing in Deaf Toddler