Health ❯ Genetics

Gene Therapy

CRISPR Technology Induced Pluripotent Stem Cells Hereditary Deafness PCSK9 Gene Embryonic Editing GBA1 Gene Experimental Treatment Mucopolysaccharidosis Type II Gaucher Disease Viral Therapies Cell-Based Therapies Genetic Editing CCR5 Gene

One Infusion Cuts LDL by Up to 62% in Early Gene‑Editing Trial

Interim NEJM data show dose‑dependent PCSK9 knockdown with an initially favorable safety signal prompting Fast Track status.

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

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