genetic diseases

News

A scheme to hasten the diagnosis of children with genetic disorders is achieving strong results and could be a template for national health systems.

News

Rocket Pharma has won FDA approval for the first gene therapy for LAD-I, a devastating genetic disorder causing severe vulnerability to infections.

R&D

Michelle Werner, Director of Alltrna, discusses her company’s approach of leveraging transfer RNA (tRNA) to shift the paradigm in genetic medicine.

News

Investor panic has poleaxed Quince Therapeutics share price, after a regulatory filing revealed the biotech's cash is running out.

News

The FDA said it needs more information before it can approve Regenxbio's gene therapy for Hunter syndrome, in a devastating decision for patients.

News

Servier has pushed its emerging neurology pipeline into the clinical trials stage by licensing a drug for Fragile X syndrome from the UK's Kaerus.