genetic diseases

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.

News

PTC Therapeutics' treatment for the rare disease PKU, Sephience, will launch in its first market next month after getting EU approval.

News

In a medical breakthrough, a child diagnosed with a rare genetic disorder has received a bespoke CRISPR gene editing therapy to correct the mutation.

News

Beam says its base-editing drug has met its goals in a phase 1/2 trial in genetic disease AATD, and it wants to raise $500m to take it forward.